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ISSUE No 60December 2011
Published by the Thalassaemia International Federation | www.thalassaemia.org.cy
TIF MAGAZINE
KNOWLEDGE IS OUR POWER UNITY IS OUR STRENGTH
ThalassaemiaInternationalFederation
Limassol
Cyprus
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Dear friends,
The start of a new year offers a chance to reflect on recent
achievements, while gathering ourselves for the chal-
lenges that lie ahead.
After two and a half decades of painstaking work, TIF
activities have entered a new phase. Having established
itself as a leading patient-driven, activist organisation, TIF
is now in a position to push for major improvements in
the lives of patients. But that is not enough. TIF must also
ensure that our efforts have a measurable impact, on
policy-makers and on patients, their families and the
medical staff that care for them.
Guidelines for the treatment and prevention of thalas-
saemia are now available from every national associa-
tion, and sustained attempts to increase the number of
reference centres around the world are ongoing.
However, a major challenge is to give greater voice to
patients by educating them about their disease, facilitat-
ing their ability to better manage the demands of living
with thalassaemia—to be involved in the decisions that
affect them and to lobby for their rights to services, to
education, to employment. The guiding principle is, as the
slogan has it: "Nothing for us, without us."
In many ways, TIF has been ahead of the curve here.
Patient-centred healthcare, for example—a concept that
has recently assumed centre stage in Europe—is a long-
standing TIF demand, as are the issues of patient safety
and patient rights. The 2nd pan-European Conference,
held in Berlin in March 2010, had as its motto "Knowledge
is Power". TIF has always said the same.
TIF's Expert Patients Programme aims to further edu-
cate patients about thalassaemia and broader public
health issues, empowering them to act as valuable part-
ners in policy-making. A core group of five expert
patients met in London in November 2011, and the pro-
gramme will soon expand to include a wider group
spread around the world.
TIF is also realising the fruits of efforts to leverage its
access to the European Union, taking a lead role in a
number of international projects aimed at improving the
management of chronic conditions. By establishing tha-
lassaemia in the broader class of haemoglo-
binopathies—diseases of the blood—TIF has ensured
that thalassaemia joins a widely-discussed category of
diseases that affect millions of people. The need now is to
integrate the management of thalassaemia in still more
national healthcare systems.
The big story, though, is about thalassaemia itself. As a
result of TIF's work with national thalassaemia associa-
tions, scientists, patients and their families, the natural
history of thalassaemia has changed forever.
Thalassaemia is no longer a fatal disease, it is a chronic
disease—one that is both preventable and treatable, with
high rates of survival where there is free access to qual-
ity care. This is a truly exceptional development.
The second major challenge, then, is to persuade policy-
makers everywhere that the most cost-effective way of
dealing with thalassaemia and other haemoglo-
binopathies is to recognise such diseases as significant
public health issues, and treat them accordingly.
TIF is proud of the great strides made by all our member
associations in 2011, encouraged by the fighting spirit of
thalassaemia patients and their families. Their remark-
able energy and determination is an inspiration to us all.
Best wishes for a happy, healthy 2012.
Panos Englezos
TIF President
“...Thalassaemia is no longer a fatal disease,
it is a chronic disease—one that is both preventable
and treatable, with high rates of survival where
there is free access to quality care.
This is a truly exceptional development.”
PRESIDENT’S Address
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CHIEF EDITOR’S Address
As we enter 2012, it is time to reflect on the past year andplan ahead for the future.
The last months have been extremely busy, exciting andproductive, with numerous activities taking place all overthe world. Indeed we have undertaken, participated in andsupported meetings and conferences in many parts of theworld including Algeria, Morocco, Philippines, Myanmar,Jordan, Bulgaria, Albania, and the Russian Federation.
We have made significant progress in consolidating ourcollaborative relationships with important organisationsin Europe and across the world. Particularly withpatient/parent associations that we had the opportunityto see during the International Conferences onThalassaemia and Other Haemoglobinopathies that tookplace last May in Antalya, Turkey.
Τhe Antalya International Conferences were undoubtedlythe highlight of the year for the global thalassaemia com-munity. They provided, as always, a delight to witness inperson the gathering of such numbers of patients, parentsand dedicated medical professionals.
This zest was also evident in the celebrations of WorldThalassaemia Day that took place on the 8th of May. Thetheme this year was ‘Equal Chance to Life’ and wasselected to highlight the health inequalities experiencedby hundreds of thousands of patients across the world.This issue of TIF Magazine contains a selection ofreports and activities that took place internationally,embracing this theme: fighting against the violation ofpatient rights.
2011 marked also the establishment of a new presti-gious and highly significant international award – theSultan Bin Khalifa International Award. This importantaward was born by the generosity of His Highness SheikhDr. Sultan Bin Khalifa Al Nahyan, son of the President ofthe United Arab Emirates. It is a great honour and privi-lege for TIF and all of us who work for the cause of tha-lassaemia to have the recognition of such eminent per-sons. The global award constitutes a milestone in thepromotion of TIF’s mission in raising awareness and pro-
moting national prevention programs and most impor-tantly, strengthening research.
An overview of all the significant events of the previousyear can be found in this issue but also a sneak preview ofthe exciting events to come. In 2012 two major regionalconferences will be the driving force of TIF’s activities inthe Asian and European regions.
The postponement of the 1st Pan-Asian Conference onHaemoglobinopathies in November has meant that thiswill take place now on 8 – 10 February 2012 in Bangkok,Thailand. Those who will attend what is expected to be animpact-making meeting will benefit from its targeted pro-gramme which with the support and contribution of keypolicy markers and medical specialists aims to map thesituation and progress of more than 15 South – East Asianand Western Pacific countries.
Furthermore, TIF has already set wheels in motion for theorganisation of the 3rd Pan-European Conference onHaemoglobinopathies and Rare Anaemias. This regionalconference will take place on 24 – 26 October 2012 inLimassol, Cyprus thus taking advantage not only of thewonderful sunny weather but also of the geographicalcrossroads at which Cyprus rests upon – joining the eastwith the west. This is an event that I am sure we all awaitwith excitement, will be the undisputed high point of theyear. This grand educational event has been placed underthe auspices of the Cyprus Government during itsEuropean Presidency and will feature a wealth of topicsboth for medical professionals as well as for patients,policy-makers and the wider health community.
TIF’s activities in 2012 will focus on further strengtheningour collaboration with member associations and medicalnetworks and deepening our impact at the regional level,building on a wide-ranging portfolio of projects developedover recent years.
On behalf of the TIF office, I wish all our readers a healthyand productive 2012.
Dr Androulla Eleftheriou
Executive Director
In 2012 two major regional conferences
will be the driving force of TIF’s activities
in the Asian and European regions.
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EDITORIAL Team
TIF MAGAZINE
Thalassaemia International Federation
PO Box 28807, 2083 Nicosia, Cyprus
Tel: +357 22319129 / 22319134
Fax: +357 22314552
E-mail: [email protected]
www.thalassaemia.org.cy
Designed by Chromasyn
Printed in Nicosia
Reproduction of material published in TIF Magazine for educational purposes isencouraged, provided it is accompanied by the following attribution “... according toTIF Magazine, the official newsletter of the Thalassaemia International Federation”.The contents of any scientific article or presentation of any material by manufactur-ers does not imply the expression of any specific companies or products does notimply that they are endorsed or recommended by TIF in preference to others. The con-tents express the opinions of the authors who alone are responsible for the viewsexpressed. TIF does not accept any legal responsibility for their contents.
Chief Editor
Androulla Eleftheriou, PhD
Editor
Helen Perry
Contributors
Dr Saeed Jaafar Al Awadhi
Dr Ali Taher
Dr Khaled Musallam
Dr Chi Kong Li
Dr Mehran Karim
Dr Michael Angastiniotis
Mrs Phoebe Katsouris
Ms Lily Cannon
Board of Directors
Panos EnglezosPresident
Shobha TuliVice President
Loizos PericleousSecretary
Riyad ElbardTreasurer
Her HighnessSheikha Sheikha BintSeif Al-Nahyan
George Constantinou
Loris Angelo Brunetta
Anton Skafi
Michael Michael
Robert (Bob) Ficarra
Saeed Jafaar Al-Awadhi
Fatemeh Hashemi
Mouna Haraoui
Ivan Dimitrov Ivanov
Duru Malyali
Ramli Mohd Yunus
Christina Stephanidou
Nailya Guliyeva
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6 Board Members Corner
8 TIF Projects
12 TIF Activities
14 TIF International Conferences12th International Conference on Thalassaemia and the Haemoglobinopathies and
14th TIF International Conference for Patients and Parents / Antalya, Turkey
17 TIF Delegation Visits
19 News from our Members Around the World
24 TIF Regional Conferences3rd Pan-European Conference on Haemoglobinopathies and Rare Anaemias
24-26 October 2012 / Limassol, Cyprus
27 World Thalassaemia Day
32 Featured ArticlesFOCUS: Thalassaemia & Employment
37 Medical FocusSynthetic blood: An end to blood drives?
A closer look at β-thalassaemia intermedia
How to select iron chelators in 2012
Hydroxyurea management in β-thalassaemia intermedia
44 TIF Regional Conferences1st Pan-Asian Conference on Haemoglobinopathies, Bangkok, Thailand
(8-10 February 2012)
48 Upcoming Events
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TABLE of Contents
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BOARD MEMBERS’ Corner
A major new award for significant contributions to
thalassaemia was announced in April 2011, backed by
His Highness Sheikh Sultan Bin Khalifa Al Nahyan, son
of the President of the United Arab Emirates (UAE),
with the support of the Thalassaemia International
Federation (TIF).
The Sheikh Sultan International Thalassaemia Award,
worth around $1 million, is sponsored by His Highness
Sheikh Al Nahyan, with TIF acting as expert adviser. TIF
President Panos Englezos will serve as Vice President
of the Award’s administrative Board of Trustees.
The prize aims to raise awareness about thalassaemia
and other haemoglobin disorders by celebrating
outstanding achievements in the field, and will be
awarded every two years. Winning individuals and/or
organisations will be announced during the biennial TIF
international thalassaemia conferences, followed by
an award ceremony in Abu Dhabi.
The UAE is proud to take the lead in establishing such
a prestigious prize, following its own significant
advances in the management of thalassaemia and in
the broader medical arena. It is hoped that this award
will play a major part in strengthening efforts to
educate the public about thalassaemia, as well as
providing further motivation and encouragement to all
those working to improve the lives of thalassaemia
patients.
Prestigious new international award for thalassaemia
Her Highness Sheikha Sheikha Bint Saif Al Nahyan
Her Highness Sheikha Sheikha Bint Saif Al Nahyan has been a driving force in raising awareness and
promoting excellence in the treatment of haemoglobinopathies in the United Arab Emirates (UAE).
With a particular interest in the well-being of children, Her Highness is also Chairman of Make-A-
Wish UAE, and a leading supporter of other local and international charities.
Saeed Jaafar Al Awadhi
has been a Board Member of the Emirates Thalassemia Society since 2003, where he
also serves as Financial Manager, in addition to his role as an officer of the Dubai Health
Authority Thalassaemia Centre. Saeed is a member of the Sultan Bin Khalifa
International Thalassaemia Award Executive Committee, and attended the recent 12th
International Conference on Thalassaemia & Haemoglobinopathies and the 14th TIF
International Conference for Patients and Parents. Saeed has been elected to the TIF
Board of Directors for a term of four years.
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TIF Projects
TIF is involved in a number of major projects across a range of areas relating to
thalassaemia, from ensuring good blood donor management to promoting the further
education of specialists in the haemoglobinopathies and developing systems for the
electronic exchange of patient data.
Donor Management
in Europe—DOMAINE
DOMAINE is a European Commission-funded project to
identify and promote good blood donor management in
practice. TIF was the only patient-led organisation
selected to take part in this important research.
The DOMAINE project aimed to devise ways of ensuring
the safe and sufficient supply of blood, by comparing
national experiences across Europe to establish good
donor management practice. The research concentrated
on donor recruitment and retention strategies, and blood
bank policy regarding patients requiring long-term
transfusion.
The first phase of the project involved a survey of donor
management practice in a number of countries. In the
second phase a manual was compiled, setting out
guidelines for good donor management, includes tools and
examples of incentives to motivate future and current
donors to become regular donors.
The final phase of the project entailed training
professionals in the use of the manual.
Following a preliminary training workshop in Slovenia in
April 2011, the DOMAINE training committee organised a
two-day, interactive training workshop in Lisbon, Portugal,
on 17-19 June 2011. The workshop involved 25
participants from across Europe, all of whom had advised
on the compilation of the manual. Participants were then
asked to complete a questionnaire evaluating the
material. The results of the questionnaire are currently
being analysed and are expected to be made public soon.
Further details regarding the DOMAINE project can be
found at www.domaine-europe.eu.
Epidemiology project
approaches completion
TIF’s epidemiology project is coming to a close after
months of data-gathering. A final analysis of the
information collected was presented to the TIF Board
of Directors in November 2011, covering more than 80
countries affected by β-thalassaemia and HbE. Data
included details of carrier rates and the number of
anticipated births each year, as well as information
regarding national health systems and correlations
between GDP, per capita health expenditure and
disease prevalence, highlighting focal points in the
global effort to manage haemoglobin disorders.
The second phase of the project will get underway in
2012, completing the collection of data on sickle cell
disease and HbH. The data compiled will play a critical
role in shaping future TIF work, as well as offering
essential information to leading United Nations agencies
such as the WHO, in their efforts to establish these
disorders on national health agendas.
MSc in Haemoglobinopathy faces
challenges
TIF was instrumental in the establishment of an MSc in
Haemoglobinopathy at University College London (UCL),
which has been recognised by the authorities in Cyprus as
a long-distance learning Masters degree. TIF is now
working to obtain similar recognition for the course from
the Ministries of Health of a number of Middle Eastern
countries, including Syria, Oman, Qatar, Kuwait, Saudi
Arabia and the UAE.
Ranked as one of the world's top four universities, UCL
is an outstanding institution of further education. The
MSc has been extremely successful and the course
directors have been awarded the Provost's Teaching
Award for excellence in teaching. It is therefore very
important that countries with a high rate of
thalassaemia and sickle cell disease recognise this
leading programme as a crucial tool in the further
education of their health care professionals.
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TIF Projects
ENERCA- the European Network
for Rare and Congenital Anaemias
TIF has also played a leading role as a partner in the very
important European Commission-funded project ENERCA
(European Network for Rare and Congenital Anaemias).
The project aims to bring experts together to improve
public health services in the field of rare anaemias and
more particularly, to identify criteria and support the
national and European efforts towards establishing or
strengthening existing Centres of Expertise in rare
anaemias, including haemoglobinopaties across the EU.
TIF has been a participant collaborator in this project for
the last three years. Thalassaemia is a rare anaemia in
most of in Europe and ENERCA is a project which aims to
create reference networks in Europe to share expertise,
disseminate knowledge for both professionals and
patients and to maintain epidemiological surveillance. The
project is ongoing but already has produced interesting
results such as a report on the legal framework and
ramifications for the establishment of expert centres,
including matters of confidentiality of patients’ data and
samples. It has produced and published recommendations
on the clinical management of sickle cell disease, while
recommendations for thalassaemia are being prepared.
Also completed are recommendations for the prevention
of haemoglobin disorders which are awaiting publication.
For more information about this and related activities,
visit the project website: www.enerca.org. In collaboration
with the Bulgarian Thalassaemia Organisation, ENERCA
has successfully organised a workshop on rare anaemias
in Sofia on the 19th - 20th of November 2011.
The final result of this phase of the project will be a white
paper on the requirements of an expert or reference centre
for rare anaemias. In formulating the recommendations the
expectations of patients of such a centre will be considered
according to patient responses to a questionnaire prepared
by TIF. This report is expected to be ready in 2012.
Thalassaemia Specific
Electronics Communications
The effective follow up of disease progression and
documentation of the impact of treatment in
thalassaemia as for every other chronic disease
has always depended on accurate clinical records.
As technology moves more and more into health,
the value of the use of electronic formats has been
widely recognised. Electronic records are ideally
suited to the long-term collection and evaluation
of such data. In an effort to support the Cyprus
Thalassaemia Centre's efforts to transfer paper-
based records to an electronic format, TIF
launched the Thalassaemia Specific Electronics
Communications project—a pilot study using a
specially designed software to upload more than
40 years’ patients data. The results of the study
were presented at a workshop held in Cyprus on 20
October 2011 and raised considerable interest and
confirmed the immense value of this project.
TIF is now working on expanding this project
across Cyprus, with the eventual aim of sharing
this software with other haemoglobinopathy
centres around the world, facilitating data
transfer and storage, as well as boosting
opportunities to network.
New TIF Publications
TIF is two publish two new books in 2012, as well as revised
editions of textbooks on the prevention of
haemoglobinopathies, updated to include advances in
laboratory techniques.
The first of the new books is Thalassaemia Guidelines for the
Accident and Emergency Department—a manual covering the
management of thalassaemia in emergency room situations,
compiled by world experts in the fields of thalassaemia and
emergency medicine. The manual offers medical staff a
convenient, easy-to-use guide to diagnosis and practical action
in the event of a thalassaemia patient requiring emergency
care. The handbook lists common presenting symptoms, such
as dyspnoea, chest pain, syncope and oedema, using flow charts
and concise text to convey essential points. It will be made
available to emergency hospital units and primary care clinics.
The second book, Guidelines for Nurses in a Haemoglobinopathy
Centre, is a textbook for nurses working in specialised
haemoglobinopathy centres compiled by a group of nurses
working in Lebanon, Cyprus and the UK. The book reflects
recognition of the special role of nurses in centres dealing with
thalassaemia and sickle cell disease, where they offer a
combination of expert knowledge and long-term patient support.
As such, the book aims not only to improve nursing care but also
to attract nurses to work in such centres over long periods, to
ensure continuity of care.
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Chain of Trust
‘Chain of Trust’ is the code name of a European funded
project, developed by the European Patients Forum (EPF) in
which TIF participates. The project focuses on building
confidence and acceptance of telehealth solutions among
patients and health professionals. Involved in the project
are professional and patient organisations, The programme
has investigated attitudes and practices but has also
conducted workshops in several countries including Norway,
Holland, Latvia and soon in Greece, to sensitise and inform
the various groups on the importance of telehealth. The
project is expected to be completed by the end of 2011.
Expert Patients Programme
This project is one of the most important projects TIF
has ever undertaken, aiming to empower patients to
take meaningful and timely decisions about issues
affecting their healthcare and quality of life. The main
component of the project has been the preparation of
educational material, which has been reviewed by an
international expert patient group as well as by a panel
of scientists. The final draft is expected to be presented
at the first official meeting of the International Core
Group of Experts in London on 25 November 2011,
where the second phase of the project will be completed.
The vision of TIF is to be able to extend in the coming
years widely across the world accurate and up-to-date
information and knowledge to its global patient family.
Endocrinology Network
The Endocrinology Network, which was established
during the international conference in Antalya in May
2011, held a second meeting in Doha, Qatar on 2-3
October 2011, during a workshop on the endocrinological
complications of thalassaemia. The objectives of the
group include the promotion of endocrinological follow-
up of multi-transfused patients, the education and
training of more endocrinologists in this specialised field,
and the promotion of collaborative research. One project
discussed in Qatar is the development of guidelines for
the management of endocrine complications in
thalassaemia. A third meeting of the group took place in
Catanzaro, Italy on 11-13 November 2011. During this
meeting, a final plan of action for the group, known as I-
CET (International Complicanze Talassemia), was agreed
and a proposal presented to TIF. The group consists
mainly of endocrinologists, as well as thalassaemia
specialists from Italy, Egypt, Greece, Cyprus, Qatar, Iran,
India, Turkey and the USA.
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TIF Projects
Thalapump 20
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Understaffingrisks WHO work onNon-Communicable Diseases
The European Genetic Alliance Network (EGAN) has joined
TIF in submitting a statement to the WHO, outlining con-
cerns over understaffing of the Human Genetics
Programme.
It is the belief of both organisations that the delay in
appointing a replacement for the Programme's previous
coordinator, Dr Victor Boulyjenkov, risks undermining
WHO work in the field of chronic genetic diseases such as
the haemoglobinopathies and other blood disorders.
The joint statement of concern, addressed to the Director
General of the WHO, also requested that the haemoglo-
binopathies receive prominence on the WHO list of Non-
Communicable Diseases (NCD). A high-level meeting of
the UN General Assembly on NCD was convened in
September 2011.
Cyprus Alliance of RareDisorders
The Cyprus Alliance of Rare Disorders (CARD) took part in
a workshop organised by the Steering Committee for Rare
Diseases of the Cyprus Ministry of Health, held on 23-24
June 2011. TIF is a founding member of CARD, which was
represented at the meeting by TIF Executive Director Dr
Androulla Eleftheriou and CARD Coordinator Lily Cannon.
The workshop aimed to map the current situation regard-
ing rare diseases in Cyprus, and included roundtable dis-
cussions on the research, prevention, treatment and diag-
nosis of rare diseases. A discussion on the support servic-
es offered to patients with rare diseases was led by TIF,
covering rehabilitation, palliative and respite care.
The workshop was the first of a series of stakeholder
events that will contribute to the drafting of a strategic
national plan for rare diseases, in accordance with
European Council Recommendation 2009/C 151/02 and
EUROPLAN guidelines.
Addressing participants, Cyprus Minister of Health Dr
Christos Patsalides stated that the creation of such a
plan was essential, enabling the state to meet its obliga-
tion to support patients by improving and expanding
services as necessary.
The workshop concluded that a significant amount of
work remains to be done, particularly in the areas of
prevention, diagnosis and treatment. The working
groups are expected to meet again before the end of the
year, for further deliberations.
21st Regional Congress of theInternational Societyof Blood Transfusion
TIF took part in the 21st Regional Congress of the
International Society of Blood Transfusion (ISBT), held in
Lisbon, Portugal, on 18-22 June 2011.
A key area of interest was the ISBT Working Party on Global
Blood Safety, formed after the International Congress of
the ISBT held in Berlin in July 2010. The objective of this
group is to foster improvements in blood safety around the
world, through the networking of individuals and organisa-
tions with expertise in transfusion medicine. Blood safety
constitutes a major concern for multiply-transfused
patients, particularly as transmission of pathogens through
blood is still occurring in many regions of the world with
high prevalence of thalassaemia.
Joint TIF/EPHA/ASPHERConference
A conference aimed at preparing healthcare professionals
in Cyprus for the island's Presidency of the European
Council, which begins in the second half of 2012, was
organised by TIF, the European Public Health Alliance
(EPHA) and the Association of Schools of Public Health of
the European Region (ASPHER), held in Nicosia, Cyprus on
3 September 2011.
Entitled Preparing the Health Community for the Cypriot
Presidency of the EU, the conference aimed to empower
public health actors to engage in policy-making before and
during the six months that Cyprus will hold the Presidency.
Topics included the need for EU consensus over the
role of public health, and the public health capacity needed
to meet identified targets. Participants included
representatives of the governmental and non-governmental
sectors—patients, policy makers and public and private
healthcare managers.
TIF Activities
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TIF Activities
TIF Executive Director Dr Androulla Eleftheriou opened
proceedings with an overview of ASPHER’s public health
competencies programme in relation to the Cyprus
Presidency, while ASPHER Board Member and EPHA Vice
President Chris Birt provided a particularly inspiring
address on the drive needed to take part in the policy-
making process.
EHA pushes innovation inhaematological research
TIF’s Executive Director was invited to represent
patients with non-malignant haematological diseases
at the 16th EHA Congress, held in London on 9 June
2011. This marked a step forward for the European
Haematology Association (EHA) in bridging the gap
between haematologists and patients, as the
Association attempts to establish a Patient Advisory
Committee. The Committee hopes to bring together
patient organisations which represent patients with
both malignant and non-malignant haematological dis-
eases throughout Europe.
In continuation of this first meeting in London of patient
organisations, a follow-up meeting was organised by
EHA in collaboration with the European Coalition of
Cancer Patients, in Brussels, Belgium on 30 -31 August
2011. TIF’s Executive Director attended this stakehold-
ers meeting entitled ‘Haematology and the Next Decade’
and participated in the meeting with a presentation
about the success story of thalassaemia in the
Mediterrnean region, focusing specifically on the story of
Cyprus. The meeting specifically focused on discussing
the need to strengthen research funding in haematology,
the revision of the clinical trials directive and the recog-
nition of professional qualifications as a means of pro-
viding quality healthcare across Europe.
Opening the conference, Member of the European
Parliament (MEP) Pawel Kowal joined fellow MEP
Elzibieta Lukacijewska to highlight public ignorance of
haematology as a key issue of concern. EHA continues to
focus considerable effort on ensuring that haematologi-
cal diseases are at the forefront of areas of interest to
the European Union's research and development pro-
gramme.
TIF joined by the other haematological patient organisa-
tions present at the meeting are preparing a joint position
paper on the outcome of the discussion, which is to be
presented to the EU Commissioner for Research and
Innovation Sciences, Mairie Geoghegan-Quinn and EU
Commissioner for Health and Consumers, John Dalli, urg-
ing them to take action.
Rare Diseases andthe Russian FederationThe 2nd All-Russian Conference for Rare Diseases andRarely Used Medical Technologies took place in StPetersburg, Russia on 21-22 April 2011. TIF was represent-ed by two experts on the haemoglobinopathies, Dr JohnPorter and Dr Mary Petrou. The conference was organisedunder the auspices of the EUROPLAN project, which sup-ports national health authorities to develop and implementstrategies for the management of rare diseases.The conference was followed by WHO's First GlobalMinisterial Conference on Healthy Lifestyles and Non-Communicable Disease Control, held in Moscow on 28-29April 2011. The aim of the conference was to support mem-ber states in the development of programmes on healthy liv-ing and disease prevention.A report on the resolutions of the All-Russian Conference forRare Diseases was prepared by Svetlana Karimova,President of the National Association of Organisations ofPatients with Rare Diseases. The report concluded that acommitment to establish national plans for rare diseaseshas gained momentum across Europe, reinforcing work bythe Russian Ministry of Health to introduce a draft federallaw. Recommendations in her report include the harmonisa-tion of Russian terminology with that of the EU, and theestablishment of an Expert Council for Rare Diseases andOrphan Techniques at the Russian Ministry of Health.For its part, TIF is maintaining its efforts to make sure thathaemoglobinopathies, and in particular thalassaemia, areincluded in the formulation of Russia's national plan for rarediseases and in the Federation's broader public health pro-gramme. We are confident that with further cooperation, thecountry will soon establish a register of thalassaemiapatients along with screening and treatment programmes,as well as prevention strategies.
Eastern Mediterranean Region(EMRO) Guidelines forHaemoglobin DisordersOn the 27-30 June 2011, TIF participated in a meetingorganised by the Regional WHO-EMRO and the BloodDisorders unit of the CDC of Atlanta, USA, held in Amman,Jordan. The meeting focused on Haemoglobinopathies andGenetic Diseases, bringing together experts and publichealth representatives from across the region. TIF was rep-resented by Executive Director Dr Androulla Eleftheriou,who was invited to cover the topic of haemoglobinopathies indeveloping countries. The purpose of the meeting was to ini-tiate a project for the formulation of guidelines for haemo-globin disorders in the Eastern Mediterranean, a process inwhich TIF will play a significant role. During the visit, TIF also gathered epidemiological and otherdata, as part of its work to assess national health servicesfor patients with thalassaemia. (For news on TIF's Delegation Visit to Jordan, see inside.)
This year's twin conferences on thalassaemia andthe haemoglobinopathies, held in Antalya, Turkey,were the most successful yet, attracting more than1,300 participants from over 50 countries acrossfive continents. The four-day event, held at the WOWKremlin Palace Hotel, was co-organised by TIF and theThalassaemia Federation of Turkey, with the support ofthe Turkish Ministry of Health. A notable feature of this year's conferences was the evenlymatched numbers of patients and medical specialists, helpingto encourage particularly lively discussion between the two. Anotherwas the awe-inspiring beauty of Antalya and the exceptional hospitality of the conferences'Turkish hosts.
Abstracts of the scientific programme can be found at
http://pagepressjournals.org/index.php/thal/article/view/tr.2011.s1
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12th International Conference on
Thalassaemia and the Haemoglobinopathies
14th TIF International Conference
for Patients and Parents Antalya, Turkey / 11-14 May 2011
TIF INTERNATIONAL Conferences
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Panos Englezos andGeorge Englezos Awards
The Antalya event served as the ideal forum for the award
of two prestigious prizes—the Panos Englezos Award for
exceptional role models in the fight against thalassaemia,
and the George Englezos Award for outstanding scientific
contributions to the field.
The 2011 Panos Englezos Award was presented to two
outstanding individuals, Costas Kontourou and Anastasios
Leventis.
The posthumous recognition of Costas Kontourou
highlighted the exemplary courage and energy of a man
who refused to give up the fight against thalassaemia. As
a patient, Costas was a committed and tireless supporter
of TIF's work, never missing an international conference or
regional workshop. But perhaps more importantly, Costas
had an infinite capacity for compassion and friendship,
sharing a joy and zest for life with everyone he met.
Costas's much-deserved award was accepted by his
beloved sister and mother, who travelled to Antalya for
this very special occasion. He is much missed by the many
people touched by his inspirational life.
As Chairman of the Leventis Foundation, Anastasios
Leventis has shown great generosity in his support for the
struggle against thalassaemia, including the
establishment of an e-MSc in Haemoglobinopathies at
University College London, launched in 2009. TIF and the
wider thalassaemia community are greatly indebted to Mr
Leventis for his unstinting encouragement of efforts to
improve the knowledge and treatment of patients
suffering from haemoglobinopathies. TIF is honoured to
have had the opportunity to mark its appreciation of such
an admirable and accomplished individual.
The 2011 George Englezos Award for an outstanding
scientific contribution to the field of thalassaemia was
awarded to Dr Hans Peter Schnebli, in recognition of his
groundbreaking work on iron chelation. Dr Schnebli's
research resulted in the development of the oral chelator
Deferasirox, an FDA/EMA approved drug that has
transformed the lives of so many patients around the world.
But this award recognised not just Dr Schnebli's scientific
prowess but his humanity. Dr Schnebli's sensitivity to the
pain and suffering of thalassaemia patients enabled him to
understand just how much an oral chelator would improve
their quality of life. It is thus as much for his personal
qualities that TIF is proud to have presented Dr Schnebli with
the 2011 George Englezos Award.
The vital l
ink in
optim
isin
g iron c
helatio
n
The only standardised, quality-controlled, MRI-based
measurement of liver iron concentration (LIC) with
international regulatory clearances and approvals.
(CE Mark, FDA, Health Canada, TGA)
FerriScan® R2-MRI provides an LIC measurement that is trusted not only by doctors managing patients in 30 countries, but also by pharmaceutical companies in clinical trials of chelation therapies.
FerriScan® o�ers an accuracy and reliability that is unrivalled by other MRI-based methods and now represents the gold standard in LIC measurement.
The FerriScan® report is available within two working days, providing an LIC result, not an indirect measurement. This, together with a report of the patient’s LIC history enables clinicians to easily and accurately assess their progress.
A measurement of cardiac T2* can also be provided with a FerriScan® at selected facilities internationally, providing a comprehensive picture of body iron loading.
Please visit www.ferriscan.com or email [email protected] for further information.
R2-MRI
17
Jordan 27-28 June 2011
TIF's participation in a WHO-EMRO meeting in Jordan in
June 2011 offered an excellent opportunity for TIF
Executive Director Dr Androulla Eleftheriou and a
member of staff to visit thalassaemia centres and blood
banks in Amman and Irbid. TIF also enjoyed a very
productive meeting with members of the Jordanian
Thalassaemia and Haemophilia Association—a long-
standing member of TIF, and its president, Dr Basem
Kiswani.
The TIF delegates, accompanied by Dr Kiswani, also met
the General Secretary of Jordan's Ministry of Health, Dr
Daifallah Al Louzi, with whom they discussed the current
status of control strategies and management services for
thalassaemia, and considered ways of strengthening TIF’s
cooperation and support for further improvements. The
meeting was extremely productive and TIF was very
pleased to have seen evidence of significant
improvements.
Morocco 4-5 May 2011
During the course of a Delegation visit to Morocco on 4-5
May 2011, Minister of Health Yasmina Baddou and TIF
Executive Director Dr Androulla Eleftheriou signed a
three-year joint plan of action, aimed at establishing a
national thalassaemia control programme. The move
marks a major step forward in recognising the importance
of haemoglobin disorders, both in terms of Morocco's
national health agenda and that of the national
haematology association.
The plan covers the 2011-2013 period, during which a
number of services to improve the lives of thalassaemia
patients are expected to be designed and implemented.
TIF was also invited to participate in a haematological
conference to be held in November 2011—a valuable
opportunity to further strengthen TIF's relationship with
the Ministry of Health, as well as to meet patients and
extend its network of health professionals in the country.
Philippines 2-4 September 2011
TIF's delegation visit to the Philippines in September
2011 marked a number of important firsts. This was the
first such visit to the country by TIF, and was organised to
coincide with the 1st National Thalassaemia Workshop
for Patients and Parents, and the 1st Health Professional
Workshop on Thalassaemia and Haemoglobinopathies.
The Philippines is highly affected by β-thalassaemia HbE
and HbH. Aside from offering an invaluable opportunity to
meet patients, parents and health professionals, the visit
also represented a chance to discuss and assess the
management, prevention and treatment of thalassaemia
on the ground, facilitating TIF's efforts to tailor its
support as needed. An important start has already been
made, with the launch of a national patients’ register to
establish patient numbers and carrier rates.
The visit kicked off with a meeting of representatives from
the national health authority, blood banks and patient
groups. TIF representative Dr Michael Angastiniotis joined
Dr Anthony Calibo, Supervising Health Programme Officer
at the Family Health Office of the National Centre for
Disease Prevention and Control, and members of the
Thalassaemia Working Group and the Philippine Society of
Haematology and Blood Transfusion for broad-ranging
discussions on key issues. The following day, the
delegation met the Head of the National Blood Centre, Dr
Bonifacio, and the Director of the Red Cross National
Blood Service, Dr Christie Monina Nalupta.
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TIF Delegation Visits
Day three saw the start of the 1st National Thalassaemia
Workshop for Patients and Parents, bringing together
patients and parents from across the country to share
their concerns and network, with the final day dedicated
to the 1st Health Professional Workshop on
Thalassaemia and Haemoglobinopathies.
Egypt 26 September 2011
A Delegation visit to Egypt on 26 September 2011
focused on supporting efforts by the country's medical
community to establish a national control programme. TIF
has enjoyed a long history of cooperating with
thalassaemia groups in Egypt, and welcomed the chance
to visit again following a period of political instability.
While Egypt has seen many significant advances in the
management of thalassaemia over recent years, carrier
rates remain high and the lack of a coherent prevention
strategy means that the already large number of
thalassaemia patients is on the increase. The visit
therefore offered a valuable opportunity to assess overall
progress in the fight against thalassaemia in Egypt, with a
view to further strengthening TIF's support.
Israel 5-6 October 2011
Despite an exemplary health system offering patients free
and full access to the highest standards of care, there are
indications of weakening compliance amongst adult tha-
lassaemia patients in Israel. TIF's delegation visit to the
country on 5-6 October 2011 therefore aimed to focus on
this issue, with a view to exploring aspects relating to psy-
chosocial needs and social integration, including opportu-
nities for employment and higher education.
The TIF Delegation had the opportunity to meet with
internationally-renowned medical experts such as
Professor Rachmilevitch – a long standing collaborator of
TIF. The Delegation also visited the EMEK Medical Centre
in the city of Afula medical staff and had discussions with
representatives of the Galilee Society for Thalassaemia
and Sickle Cell Anaemia.
The following day, the Delegation visited the Wolfson
Medical Centre, the Rabin (Beilinson) Medical Centre and
the Schneider Children’s Medical Centre of Israel.
Meetings were also held with representatives of the
Israeli Association for Thalassaemia and Sickle Cell
Disease.
Discussion centred on a number of issues, including current
research on thalassaemia in Israel, the extension of MRI
T2* to more countries for the monitoring of cardiac iron
overload, and ways to move forward successfully transfer-
ring from paediatrics to adult haematology medicine.
The visit offered TIF the opportunity to further strengthen
its commitment to supporting the thalassaemia commu-
nity in Israel in a number of ways, including the develop-
ment of cooperative networks of medical specialists and
national thalassaemia associations, the provision of edu-
cational material and the organisation of a national tha-
lassaemia workshop in the near future.
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TIF Delegation Visits
Send us your newsIf you have important news
you want to share
with the world thalassaemia community,
let us know!
4th Pan-AmericanThalassaemia Conference
By Dr Vasili Berdoukas, USA
and Dr Aderson Araujo, Brazil
The 4th Pan-American Thalassaemia Conference tookplace in São Paulo, Brazil at the Maksoud Plaza Hoteland Conference Centre on 25-26 March, 2011. The eventattracted around 60 participants, mainly physicians, fromthe Americas and Europe for two days of expertworkshops and intensive debate on current treatment andthe latest advances in thalassaemia. Presentations covered transfusion therapy, chelationtherapy, emerging challenges for paediatric thalassaemiapatients, the kidney in thalassaemia, transfusion andchelation in thalassaemia intermedia, and bone marrowtransplantation in thalassaemia. Three breakout workshopsfocused on heart iron overload, the treatment of childrenand endocrine disorders. The Conference also provided an opportunity forparticipants to learn more about ABRASTA and theexperience of patients in Brazil, including presentations onthe work of the Thalassaemia Working Group at Brazil'sMinistry of Health and a case study of pancreatic iron load inBrazilian patients. An overview of ABRASTA's work highlighted theorganisation's close cooperation with national healthcareprofessionals and the Ministry of Health. As in many othercountries, all three iron chelating medications arecurrently available in Brazil. In addition, patients enjoy ahigh standard of transfusion therapy, with safetyexpected to be further improved with the introduction ofmolecular testing of individual units of blood for virusesbefore each transfusion. Viral inactivation will also beintroduced when available.
The country also boasts state-of-the-art facilities forthalassaemia patients, such as those at the AlbertEinstein Hospital for the assessment of iron load on theheart, liver and pancreas—essential procedures for alltransfusion-dependent patients, including those withthalassaemia intermedia and sickle cell syndrome. The Conference organisers are extremely grateful toeveryone that helped make the event such a success.
Westminsterhosts receptionThe UK All-Party Parliamentary Group (APPG) on Sickle
Cell and Thalassaemia
held a reception at the
House of Commons on
16 November 2010,
entitled Sickle Cell
Disease and
Thalassaemia: What
Next?
The event was hosted by APPG Chair Diane Abbot, MP,
with around 100 guests attending, including patients, cli-
nicians, nurses and MPs. The aim of the event was to
ensure that the National Health Service (NHS) maintained
a focus on thalassaemia and sickle cell patients in the
midst of fundamental changes to the NHS. Guests were
invited to sign a petition calling for ethnic minorities
affected by these diseases continue to receive equal
access to NHS services and that funding for the spe-
cialised services they require receive adequate funding.
The petition was delivered to the Prime Minister's resi-
dence, 10 Downing Street, by representatives of the UK
Thalassaemia Society and the Sickle Cell Society, accom-
panied by MP David Burrowes, on 2 February 2011.
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Cyprus Institute ofNeurology and Geneticscelebrates 20 years
‘The Cyprus Institute of Neurology and Genetics stands
out like a beacon in the Mediterranean Sea attracting the
admiration of Europe and the world.’ So said Richard
Hughes, President of the European Federation of
Neurological Societies, on the occasion of the Institute's
anniversary.
Known by its acronym CING, the Institute was founded by
Dr Lefkos Middleton in Nicosia in 1990. The scientific and
academic communities of the island were extremely
underdeveloped at the time. However, given the immense
number of neurological and genetic disorders that occur in
the Mediterranean region, it was thought essential to build
up local capacity to carry out research on such disorders.
CING has established national programmes for genetic
testing, as well as developing means of preventing
thalassaemia, Down’s Syndrome, hereditary breast cancer
and meningitis, among others. It has collaborated with
over 100 academic institutes around the world and has
published countless scientific articles in internationally
recognised journals. New diagnostic, preventative and
national programs are currently being introduced, while
the academic character of the institute has been
augmented with the establishment of a Masters degree in
Genetics at the University of Cyprus, from September
2010, with the result that the Institute is now recognised
as one of the leading scientific foundations in Europe.
CING's Department of Molecular Genetics, Thalassaemia,
has produced outstanding work in developing a new
technique for the diagnosis of thalassaemia and other
haemoglobinopathies, using Preimplantation Genetic
Diagnosis (PGD) and the microarray ‘ThalassoChip’. The
development of another innovative technique is currently
underway, for the non-invasive pre-natal diagnosis of
haemoglobinopathies, via testing of the maternal plasma.
Despite the current economic crisis, Dr Middleton insisted
that the Institute’s research must continue to be funded,
as a relatively small investment that promises to continue
making major contributions to science.
Foundation AgainstThalassaemia Faridabad, India
The Foundation Against Thalassaemia organised a huge
awareness-raising event on 5 June 2011 at Gopal Garden,
Faridabad, attracting around 5,000 people for a fun-
packed occasion that was broadcast live by Divya TV.
The day could not have been the success it was without
the generous support of a number of individuals. The
Foundation Against Thalassaemia wishes to extend spe-
cial thanks to singer Narinder Chanchal, who made a
donation of Rs 50,000 in support of local children with
thalassaemia. The event venue was provided by Sant
Gopal Gupta and family, who also provided refreshments
and dinner for all. The Member of the State Legislative
Assembly (MLA) for Faridabad, Anand Kaushik, and Mayor
Ashok Arora also pledged to lend political support to
patients with thalassaemia.
There is currently no government funding provided for the
treatment of children with thalassaemia in India, and local
patients rely on our organisation for essential drugs. An
additional aim of the occasion was therefore to raise
funds for this purpose, as well as to appeal for regular
blood donors. Contributions towards the purchase of
medicines were gratefully received from H K Batra,
Mukesh Aggarwal, Sanjay Kakkar, Manohar Puniyani, I J
Kalia and Rachna Sharma.
Kerala patients protestagainst empty promises
The Blood Patients Protection Council (BPPC) organised a
march on 8 April 2011, in protest at the continued failure
of political parties in the southern Indian state of Kerala
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to honour commitments to patients suffering from blood
disorders. Around 30 protesters were detained by police
and subsequently released on bail.
Patients in Kerala have been promised that a properly
equipped haematology-oncology department would be
established at the Kozhikkode Medical College Hospital
and the facility upgraded to a centre of excellence, and that
patients would be provided with free treatment—pledges
that had persuaded BPPC to withdraw its candidate for
state Assembly elections, Girija Krishnadas. Following the
protest, the state government agreed to provide thalas-
saemia patients with leuckocyte filters, free of charge.
The march was led by BPPC's Kareem Karassery, along
with K S Praseeda, K K Nizar and Sathyan, and included
patients with thalassaemia, haemophilia, sickle cell
anaemia and leukaemia and their families.
A second day of action was organised to mark World
Sickle Disease Day, 19 June 2011, when BPPC observed a
one-day fast to draw attention to the needs of patients
suffering from blood-related diseases such as sickle cell
anaemia, thalassaemia, haemophilia and leukaemia. The
fast was led by BPPC General Convener Kareem
Karassery and M Muhammed Ershad, the parent of a child
with sickle cell disease.
BPPC is demanding that patients with acute blood-related
diseases receive a monthly pension of Rs 3,000, as well as
compensation for medical negligence. The Council also
submitted a list of 11 adolescent sickle cell patients who
have died over recent months as a result of inadequate
expert treatment. A 20-year-old who died on World Sickle
Cell Disease Day was also included in the list. A copy of the
memorandum was submitted to Kerala's Chief Minister, the
Health Minister and the leader of the opposition.
Thalassaemia JagrutiFoundation
An audience of some 250 parents and patients with thalas-
saemia and cerebral palsy gathered at the Polio Foundation
in Ahmedambad on 19 June 2011, for a fun-filled day co-
organised with Prathama Blood Centre, the Civil Hospital,
Ananya and the Thalassaemia Jagruti Foundation.
The event, hosted by TV personality Hridaynath
Garekhan, opened with a performance by Ganesh Stuti of
Manali Dance Academy, followed by a musical pro-
gramme led by Bipinbhai Solanki and a skating perform-
ance by Sahil Piyushbhai Shah, a young—and very tal-
ented—thalassaemia patient. There was also a fascinat-
ing presentation by Dr Anil Khatri on the Practical
Management of Thalassaemia.
The event was marked by a number of examples of excep-
tional generosity, for which organisers, patients and their
families would all wish to give their warmest thanks.
One highlight was the announcement by Dr K M Maheria,
Head of Paediatrics at Civil Hospital, that all thalassaemia
patients would receive free iron chelation therapy, repre-
senting a monthly outlay in the region of Rs 3,000 per
patient.
Local chartered accountant Shri Rajnibhai and Hardik
Book Store announced plans to provide free text books to
children with thalassaemia, while an anonymous donor
offered to pay their school fees.
A number of donations were also received: the
Thalassaemia Jagruti Foundation received Rs 10,000 from
Doliben, while the Polio Foundation and Prathama Blood
Centre also received generous financial contributions.
After light refreshments, provided by Ananya, children
and their parents were treated to the services of a pro-
fessional photographer, also courtesy of Ananya, with
framed pictures to be presented free of charge to each
family.
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‘The New Generation’ 16th Thalassaemia CampMalaysia 10-12 June 2011
The 16th Thalassaemia Camp was held in Taiping, Perak,
Malaysia, on 10-12 June 2011, attracting more than 100
participants including patients and their families and
medical practitioners.
The theme of the event was ‘The New Generation’.
Presentations by patients on living with thalassaemia
were extremely well received, as well as talks by medical
professionals on issues such as blood transfusion and
iron chelation therapy.
Compliance was another important area discussed.
Charendee Soon highlighted the tragic fact that despite
free treatment, thalassaemia patients continued to die
from complications associated with iron overload because
of the challenges of fully adhering to prescribed
treatment—a point reinforced by Abu Mansor Bin
Muhamad, who also stressed the importance of patients
attending regular medical appointments. And he
emphasised the extent to which thalassaemics can lead
long and productive lives, successfully pursuing further
education, fulfilling careers, marrying and having a family.
Another speaker, Benita Aryyani, expressed her gratitude
for the moral support she receives, helping her enjoy daily
activities just like any other teenager.
The last day of the camp focused on areas such as the
role of nutrition, as well as the personal experiences of a
number of patients.
More Blood. More life
World Blood Donor Day, celebrated on 14 June 2011, is an
integral part of national blood donor programmes, serving
as an opportunity to thank blood donors and to promote
voluntary blood donation as a means of securing a safe
supply of blood and blood products.
The organisers of the annual event (the WHO, the
International Federation of Red Cross, Red Crescent
Societies, the International Federation of Blood Donor
Organisations and the International Society of Blood
Transfusion), invited participant countries to ‘Paint the
world red’ by covering or lighting monuments, buildings
and popular landmarks with the colour red, and by staging
cultural events with a red-coloured theme.
This year’s host for World Blood Donor Day was Buenos
Aires, Argentina, which organised events around the
theme ‘More Blood. More Life'—highlighting the urgent
need for more blood donors everywhere.
AustraliaSydney IVF (now renamed Genea) hosted an event on
Preimplantation Genetic Diagnosis (PGD) on 22 March
2011. The evening served to inform patients about PGD,
explaining how IVF is used in combination with PGD to help
people with genetic disorders have healthy children.
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Ali Taher receives honorary distinction
Dr Ali Taher, Professor of Internal Medicine at the American University of Beirut (AUB),
has been elected a Fellow of the Royal College of Physicians (FRCP) of London. The title
of FRCP serves as recognition by one of the most prestigious associations in medicine,
and is a great honour for Dr Taher and the Faculty of Medicine at AUB. The announcement
is a very timely acknowledgment of Dr Taher’s significant scholarly work in the world of
haematology research, particularly in the field of thalassaemia intermedia.
First Lady of Iraq receives awardIraq’s First Lady, Hero Talabani, was one of the individuals
recognised by the Kurdistan Thalassaemia Care Centre for
her work helping thalassaemia patients in the Kurdistan
region. The award ceremony took place at the Sulaimani
office of the Kurdistan Thalassaemia Care Centre.
Pan-Hellenic Thalassaemia
ConferenceThe annual Pan-Hellenic Thalassaemia Conference took
place on 30 September-2 October 2011 in Ioannina, Greece.
The conference, organised by the Hellenic Thalassaemia
Federation, attracted over 650 participants, including
patients, medical professionals and medical students. The
main objective of the Conference is to provide updates on
recent scientific research and other important
developments in thalassaemia.
Baku hosts the 61st WHO Regional
Committee for EuropeMinisters and representatives from the 53 countries of the
WHO European Region met in Baku, Azerbaijan, on 12-15
September 2011, for the 61st session of the WHO Regional
Committee for Europe. TIF representative Dr Victor
Boulynjekov participated as an observer at the Regional
Committee meeting, contributing a presentation on the
health agenda for 2012-2020.
Dr Boulynjekov also made a statement entitled ‘Management
of Thalassaemia: A multi-organ disease – A public health
issue’, calling on the WHO European Regional Office to adopt
a more active role in supporting the fight against thalas-
saemia and other haemoglobinopathies within the WHO's
non-communicable and rare diseases programmes.
TIF has stressed the importance of more effective disease
control management in European countries, in light of the
increasing migration to Europe of populations from affect-
ed countries. A further issue highlighted is the difficulties
European health systems face in communicating with
members of such populations due to language, cultural and
social constraints.
Most European countries have no national prevention pro-
grammes in place, risking an increase in the number of affect-
ed births. TIF has therefore suggested that the WHO
European Regional Office assists in the promotion of such
national control programmes, as well as in the collection of
accurate, up-to-date epidemiological data on the haemoglo-
binopathies, and in the adoption of guidelines for the consis-
tent management of such disorders across Europe. TIF has
also called for the creation of European Reference Centres to
support cooperation between patient communities and the
medical profession.
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Pakistan Thalassaemia
Welfare Society
The PakistanThalassaemiaW e l f a r eSociety has hada busy fewmonths. The Society wasvery grateful toL o n d o n ' sB i s m i l l a hC h a r i t yFoundation for organising a visit by Dr Farrukh TasnimShah, Consultant Haematologist at the WhittingtonHospital and University College Hospital, London whocame spent the day with us on 27 April 2011. TheFoundation also donated five pumps and 600 vials ofDesferal. During her visit Dr Shah held a free clinic for thalas-saemia patients, seeing around 50 patients sufferingfrom retarded growth, endocrine deficiencies, and car-diac and hepatic complications. Each was given adetailed examination and treatment advice. The opportunity to receive such expert assessment wasgreatly appreciated by patients and parents, as well asby observing medical staff. The Society also organised a visit to Sozo AdventurePark in Murree for over 40 patients and parents. The visitaimed to raise awareness about thalassaemia, throughthe distribu-tion of pam-phlets and aseries of moti-vational lec-tures byShabnum Ijazand MurtazaAli Burhani.On 3 August 2011, the Pakistan Crescent Society held aceremony to mark the donation of 50 pints of blood tothe Pakistan Thalassaemia Welfare Society. Lt-Gen MKamal Akbar, Vice President of the Society, received thegenerous donation with gratitude.
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TIF is delighted to announce the 3rd Pan-European
Conference on Haemoglobinopathies and Rare Anaemias,
to be held in Limassol, Cyprus on 24-26 October 2012.
The Conference will be held under the auspices of the
Cyprus Presidency of the European Union (EU) and the
Cyprus Ministry of Health, and is expected to attract
participants from all 27 EU-member states.
The overall theme of the event is the growing public
health burden of chronic and rare diseases in Europe, with
a particular focus on patient rights and policy-making.
Topics for discussion include: health inequalities
across the EU; cardiac, endocrine and liver
complications; and advances in the care and cure
of haemoglobin disorders and rare anaemias. The
programme includes a day of policy-related
discussion on control, prevention and clinical
management, the conclusions of which will be
submitted to the European Commissioner for
Health and Consumers Affairs.
Patients, organisations, healthcare professionals,
academics and policy-makers from all levels
(national, regional and EU) are encouraged to attend.
Thalassaemia sets the pace
This Conference aims to highlight a critical link between
research and policy that has led to major breakthroughs in
the treatment and prevention of thalassaemia—a trend
that TIF strongly believes can be replicated in tackling
other haemoglobinopathies. Once a fatal disease,
thalassaemia is now a treatable condition;with appropriate
national control programmes, it is also preventable.
After years on the fringes, haemoglobin disorders have
finally been recognised as a major policy focus; and as an
immense burden on national heath systems, requiring
policy-makers' sustained attention. The World Health
Organisation (WHO) has adopted two specific resolutions
on sickle cell anaemia, thalassaemia and other
haemoglobinopathies, calling on member states to
implement national control programmes as a matter of
priority, including training health professionals, promoting
community education and cooperating with the WHO to
establish expert groups in each region. Other resolutions
urge member states to ensure the sustainability of blood
supplies, to raise awareness of birth defects (including
haemoglobin disorders) and to improve epidemiological
surveillance systems. These should serve as the
foundation for further improvements in the prevention and
clinical care of haemoglobinopathies.
The EU boasts some of the world's highest quality public
health services. However, there is considerable variation
TIF REGIONAL Conferences
3rd Pan-European Conferenceon Haemoglobinopathies and Rare Anaemias24-26 October 2012 / Limassol, Cyprus
The Cyprus Presidency of the EU
Cyprus assumes the Presidency of the Council of the
European Union in the second half of 2012, offering an
invaluable opportunity to highlight issues of concern
across the Union. At a time when societies across
Europe face considerable challenges, Cyprus is
determined to use its Presidency to promote principles
of solidarity, humanity and the vision of a healthier
Europe by 2020. TIF is delighted to enjoy the full
support of the government of Cyprus in playing its part,
ensuring that the 3rd Pan-European Conference on
Haemoglobinopathies and Rare Anaemias is a
success—not just for patients with thalassaemia, but
for all those fighting the haemoglobinopathies and
other rare diseases.
24
25
in standards of management of haemoglobin disorders. It
is therefore essential that the EU adopts WHO
resolutions in this area, with a view to standardising the
control, management and treatment of haemoglobin
disorders across the Union. It is also essential that
interested parties work together to ensure that the
haemoglobinopathies—both malignant and non-
malignant—are presented as a united and distinct group
of diseases, worthy of top-class research. To this end, the
Conference aims to highlight the immense contribution
research into thalassaemia has made to the broader field,
particularly in the areas of gene therapy, pre-natal
screening and the development of more effective drugs.
The Conference will also focus on the challenges national
health services face in the treatment and monitoring of
chronic conditions, as well the challenges faced by
patients in negotiating highly heterogeneous health
systems with great variations in knowledge and expertise
between (and sometimes within) countries and regions.
Discussions will include the revision of the Clinical Trials
Directive, counterfeit medicines, cross-border healthcare
and pharmacovigilance.
Participants
The Conference is being organised in cooperation with the
Cyprus Ministry of Health, under the auspices of President
of Cyprus Mr Demetris Christofias, the Cyprus and
European Haematology Societies, and the office of the
Cyprus Presidency of the EU. It will be co-chaired by the
Minister of Health, Dr Stavros Malas, and TIF President
Panos Englezos.
President Christofias has been invited to open the
Conference, joined by representatives from the European
Commission, the European Parliament, national health
authorities and European and international patient
organisations. The event is also being supported by the
Cyprus Alliance for Rare Disorders (CARD), of which TIF is
a founding member, and the EU's ENERCA project.
Over 800 individuals from more than 35 countries are
expected to attend, including patients and their families,
academics, researchers, health professionals, carers, and
public health officials.
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TIF REGIONAL Conferences
The town of Limassol is a leading tourist
resort with state-of-the-art conference
facilities, set along a 16-km beach front
overlooking the Mediterranean Sea. The
town is a traveller's delight of historical
monuments, from ancient Amathus to a
Venetian castle, with soul-stirring stories
to match (the marriage of King Richard
the Lionheart to Princess Berengaria is
just one). All of which promise to make
the 3rd Pan-European Conference on
Haemoglobinopathies and Rare Anaemias
an unforgettable experience.
Join us in sunny Limassol!
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8th of May
This year's World Thalassaemia Day was celebrated
with all the joy and enthusiasm we have to expect. The
slogan for the 2011 event was Equal Chance to Life,
inspiring patients everywhere to keep up the fight for
fair access to healthcare, education, employment—in
short, to fulfil their dreams.
Bangladesh
Bangladesh Thalassaemia Foundation (BTF)
The Bangladesh Thalassaemia Foundation (BTF) was proud
to welcome Prof Dr Syed Modasser Ali, Health Adviser to
Prime Minister Sheikh Hasina, as chief guest at a seminar
held to mark World Thalassaemia Day 2011. The event was
also attended by the Secretary-General of the Bangladesh
Medical Association, Prof Sarfuddin Ahmed, the General
Secretary of Shadhinota Chikitsak Porisod, Prof Iqbal
Arsnal, and the Director of the Bangladesh Medical
Research Council, Prof Habib-e-Millat.
The seminar focused on health policy and governance, as
well as marking the official launch of TIF's 'Guidelines for
the Clinical Management of Thalassaemia', published by
BTF for distribution in Bangladesh.
BTF Secretary-General Dr Md Abdur Rahim provided an
overview of thalassaemia in Bangladesh, which has about
350,000 thalassaemia patients. Around 7% of the total
population are carriers of thalassaemia and more than
7,000 affected babies are born each year. A major
challenge is therefore posed by the inter-marriage of
carriers. Thalassaemia has been included in the 2011-16
Health, Population and Nutrition Section Strategic Plan
(HPNSSP), however, Dr Abdur Rahim urged officials to
ensure that the disease remains high on the policy agenda.
Dr Ali offered his assurance that he would appraise the
prime minister of the continued need for diligence in the
prevention and treatment of thalassaemia, encouraging
the Health Education Bureau to increase awareness of
the disease.
But Shabab Haider Siddique, a thalassaemia patient, had
a wider message, telling the audience that “An equal
chance to life does not mean merely living with
treatment, but equal rights in education, work and other
services.”
Bangladesh Thalassaemia Samity (Society)
The Bangladesh Thalassaemia Samity (BTS) marked the
8th of May with an event at the National Press Club,
addressed by the Minister for Social Welfare, Enamul
Haque Mostafa Shaheed.
A discussion panel brought thalassaemia patients and their
families together with paediatricians, haematologists and
other medical specialists, while BTS Vice-President Nazrul
Islam made a presentation on the prevention and
treatment of thalassaemia.
A number of speakers called for urgent action to improve
the care of thalassaemia patients, including:
World Thalassaemia Day
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World Thalassaemia Day
• establishing facilities for the treatment of thalassaemia
in all government hospitals
• ensuring the availability of blood for transfusions
• improving/increasing facilities for the screening of
blood before transfusions
The Minister responded by noting his deep concern at the
increased number of thalassaemia patients diagnosed in
Bangladesh each year, acknowledging that government
authorities needed to do more to contain the disease and
to ensure appropriate patient care. And he invited the
submission of proposals for projects to that end, to be
funded by the Ministry of Social Welfare.
World Thalassaemia Day was also observed in all 12
medical colleges across the country, following
cooperation between BTS and the Medicine Club, a social
welfare organisation.
Cyprus
The Cyprus Thalassaemia Association in cooperation with
TIF organised a workshop entitled ‘Blood Transfusion
Therapy—Standards, Quality and GCP (Good Clinical
Practice)’, held at the Cyprus Institute of Neurology and
Genetics in Nicosia, under the auspices of the Cyprus
Ministry of Health. The aim of the workshop was to edu-
cate patients and health professionals on European stan-
dards in transfusion therapy.
Guests included world-renowned specialists Prof
Umberto Rossi, President of the European School of
Transfusion Medicine (ESTM), and Niels Mikkelsen,
Honorable President of the International Federation of
Blood Donor Organisations (FBDO/FIODS).
Speakers included Dr Shubha Allard, Consultant
Haematologist at Barts & The London NHS Trust/NHS
Blood and Transplant; Dr Alan Kitchen, Head of NTMRL at
NHS Blood and Transplant, London; Carolina Stylianou,
Laboratory Officer at the Nicosia General Hospital Blood
Bank; and TIF Executive Director Dr Androulla
Eleftheriou.
India
Foundation Against Thalassaemia The Foundation Against Thalassaemia organised a
grand function at DAV Centenary College, Faridabad,
hosted by Foundation President RD Sharma and inaugu-
rated by DAV Principal Dr Satish Ahuja. The event was
dedicated to the late Gopal Sharma, founder of MVN
Institutions and a leading figure in the establishment of
the Foundation.
Faridabad Mayor Ashok Arora announced his intention
to meet the Chief Minister of the state of Haryana, to
request a grant towards the treatment of thalassaemia,
as part of efforts to improve patient quality of life.
Guests were treated to a much-appreciated song and
dance performance by children with thalassaemia—a
credit to the work of music teacher Anju Munjal and
Ganesha Dance Academy.
Thanks to Mukesh and Shashi Aggarwarl, Mr Kushal and
Mrs Palak for their generous gifts, which were greatly
appreciated by the performers, as well as to TV person-
ality Nikuj Malik. The event was generously supported
by Venus Industries and Sanjay Kakkar, including a din-
ner for patients, families and friends.
Blood Patients Protection Council
The Blood Patients Protection Council (BPPC), based in
Kozhikode, Kerala, distributed pamphlets, as well as
organising a series of discussions for patients and their
families. Topics included the prevention of thalassaemia,
and the prevalence of depression in adolescent thalas-
saemia patients.
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Dr S P Foundation
Dr S P Foundation focused its event on the problem of
stigmatisation of patients and the importance of social
support for patients. Free blood-tests were available,
along with informative talks on prenatal diagnosis.
Awareness-raising pamphlets were also distributed.
Iran
Khouzestan Thalassaemia SocietyThe celebration of International Thalassaemia Day in
Khouzestan province, Iran, was marked by an event at
Aftab Hall, attended by around 500 patients, families, and
medical specialists. The event was also attended by mem-
bers of the Sports Committee of Special Diseases, which
works to increase the participation of patients with tha-
lassaemia, diabetes and haemophilia, and organ trans-
plant patients.
The head of the Khouzestan Thalassaemia Society, Dr
Forouzan Sadeghian, opened proceedings with a discus-
sion of the Society's activities for 2011, which include
seminars on thalassaemia and alloimmunisation treat-
ment, a patient workshop, and free bone densitometry
assessments for patients in the province. The society has
also provided assistance in setting up thalassaemia asso-
ciation websites and holding Nowruz (Persian New Year)
exhibitions, as well as cooperating with the ministry of
health to distribute information regarding thalassaemia.
Another project highlighted was the purchase of land for
the construction of a thalassaemia clinic. But perhaps the
most important development of all is the distribution of
free oral chelating drugs, which has had a great impact on
patients' quality of life.
The event included live music and theatre performances,
along with face-painting for children. A particularly spe-
cial moment was taken to recognise the achievements of
university graduates and athletes with thalassaemia, as
well as those who have overcome the odds to find a happy
marriage.
Lebanon
Middle East Medical Assembly
In honour of World Thalassaemia Day, this year's 44th
annual Middle East Medical Assembly (MEMA) included a
special day of lectures on the haemoglobinopathies. The
Assembly, held at the American University of Beirut
Medical Centre on 5-8 May 2011, was jointly sponsored by
the Cleveland Clinic Foundation and was attended by
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World Thalassaemia Day
around 140 participants with an interest in cardiology,
childhood oncology, haematology, surgery and molecular
medicine.
There were five lectures on issues related to thalas-
saemia, including the Khalil Abou Faysal Memorial
Lecture, presented by Dr Paul Hassoun of Johns Hopkins
Medical Centre, and presentations by Prof Maria Domenica
Cappellini of the University of Milan, Dr John Wood of the
Children’s Hospital of Los Angeles, Prof Ali Taher and Prof
George Atweh of the University of Cincinnati. The sympo-
sium was followed by a Q&A session.
Myanmar
Myanmar celebrated World Thalassaemia Day for the first
time this year, in an event marked by more than 200
patients and parents.
The event offered a combination of fun and practical
advice. Free assessments of bone density were on offer—
as well as an abundance of ice-cream and cake. Attractive
gifts of a bag printed with the words 'World Thalassaemia
Day 2011' were also well received.
There was also time for a highly informative talk on the
"Optimal Management of beta-Thalassaemia intermedia"
(based on British Journal of Haematology article by
Taher, Musallam, Cappellini and Weatherall, 152, 512-
523), leading to a lively discussion amongst the haematol-
ogists present.
Pakistan
Thalassaemia Society Of Pakistan
The Thalassaemia Society of Pakistan (TSP) celebrated the
8th of May with a colourful variety programme, held at a
children's library. The event marked the culmination of a
month of activities, including seminars, blood donor drives,
awareness-raising lectures and the distribution of inform-
ative literature.
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World Thalassaemia Day
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TSP President Dr Jovaria Mannan highlighted the chal-
lenges posed by thalassaemia in Pakistan, where 9,000
afflicted children are born each year and 6% of the popu-
lation are carriers—a situation aggravated by the custom
of marriage between cousins.
TSP General-Secretary Prof Yasmin Raashid stressed the
role of prevention, calling for couples to be tested before
marriage and for prenatal diagnosis for carriers in the
12th week of pregnancy.
TSP Chief Administrator Abdul Munim Khan appealed for a
nationwide push to increase blood donation and to ensure
blood safety, warning that patient health was being jeop-
ardised by blood from unauthorised sources. He also said
that a great many patients still lacked regular iron chela-
tion therapy.
Zakia Shahnawz, adviser to Punjab Chief Minister and
guest of honour, pledged the support of the Chief
Minister's office and applauded the continued efforts of
patients and Society staff. She then joined the audience
for a warmly-received performance by the children.
Palestine
Palestine Avenir FoundationThe Palestine Avenir Foundation/Hippocrates Centre
organised a wonderful event to celebrate two important
occasion—Thalassaemia and Haemophilia International
Days. The event was attended by some 450 patients, par-
ents, official guests and supporters.
Philippines
Balikatang Thalassaemia Foundation Balikatang Thalassaemia Foundation marked its 6th
annual celebration of World Thalassaemia Day with a
conference on the Economics of Thalassaemia, held on 14
May 2011 at Dr Fe del Mundo Medical Centre. The event
brought together patients, friends and families, along
with staff from Philhealth, the Philippine Charity
Sweepstakes Office and the Philippine National Red
Cross.
Guests were invited to wear red, and enjoy a day of learn-
ing, fun and fellowship, as well as raffle prizes and other
surprises.
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World Thalassaemia Day
Palestine Avenir Foundation
Never allow thalassaemia to hinder your road to success. I take it as a challenge and
not a problem, a different ability rather than a disability—one that comes with deeper
insights into the possibilities of what life may bring to us. Disease is inevitable. But
whether or not it causes ‘dis-ease’ is entirely optional, just as pain is inevitable but
suffering is altogether optional.Sukhsohit Singh (patient with β-thalassaemia major, India)
32
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FEATURED Articles
Introduction:
The preparation of this statement was inspired by the
experience of Sukhsohit Singh, a thalassaemia patient in
India who was rejected for employment by the Indian Civil
Service (ICS) on the basis of a Medical Board decision
focused exclusively on the fact that he had thalassaemia
major—despite his presenting no debilitating
complications associated with the condition. Mr Singh has
an excellent academic record and performed brilliantly on
the ICS exam. The case follows that of Dr Anjali Sardana,
also in India, who was rejected for an academic post on
the same grounds.
Current Global Situation:
The problem of employment discrimination against
patients with thalassaemia and other chronic disorders is
by no means confined to India. Indeed, patients in India are
in many ways better supported than those in many other
countries. As a result of sustained advocacy work by local
patients’ associations and specialist physicians, both of
the above-mentioned cases have been reconsidered: Dr
Sardana is now an Assistant Professor, and the ICS is
reviewing the ruling regarding Mr Singh.
Consider the startling results of a recent survey
conducted by TIF as part of the ENERCA project on rare
”
‘‘
TIF Statement on the Employment Rightsof Thalassaemia Patients
FOCUS: THALASSAEMIA & EMPLOYMENT
anaemias, funded by the European Commission and
involving more than 300 patients over the age of 20 with
thalassaemia (90%), sickle cell syndromes (8.5%) and
other congenital anaemias. The survey found that a total
of 30.7% were unemployed (13.9% through choice), with a
further 19.3% working part-time. Only half of patients
surveyed were fully employed,.
Europe has robust health and social services resulting in
the best survival rates for thalassaemia, and takes a lead
role in declarations and conventions on patients’ rights,
the rights of the disabled and the chronically sick. But the
continent can only employ 50% of the adult affected
population. What, then, is to be expected of lower
resourced parts of the world?
Another TIF survey, conducted in the Middle
East in 2009 and involving 96 patients, found
that over 80% felt healthy and strong enough
to undertake a full-time job. Despite their
desire to work, however, many reported
obstacles—from prospective employers and
society in general. Less than 20% of
respondents felt they faced 'no problem' in
getting a job. Just over 30% of patients
stated that employers were reluctant to
employ people with thalassaemia, while
11.4% stated that they faced outright refusal.
While a few patients (9.4%) cited job
shortages as a contributing factor, others
referred to prospective employers' own
biases. The following are examples of the
reactions some job-seekers faced
‘They think it (thalassaemia) is catching’
‘Repeated absences’
‘Fear that something is going to happen
because of the disease’
‘They think that we can’t do the job’
‘Short height’
‘Difficult to get a job if features, facial or skin
colour are obvious’
‘They see us as parasites’
‘There is pity which is not a good feeling’
Modern treatment has dramatically
increased the life expectancy of patients
with thalassaemia major, especially those
who adhere to treatment regimes. However,
a longer life is not a gift unless accompanied
by the fulfilment of one's expectations and
abilities, in terms of education, employment
and marriage.
The TIF/ENERCA survey of patients in Europe found that
27.5% of 273 respondents were married, with another
7.7% cohabiting and 2.9% divorced. A total of 35 children
have been born to those surveyed and 48.6% of 150
respondents were university graduates. All of which only
reiterates what we already know: it is possible for
thalassaemia patients to fulfil their aspirations to be
productive members of society, supporting themselves
and their families.
Responsibility of Employers and Governments toEnsure Patients’ Employment:
Article 23 of the Universal Declaration of Human Rights,
1984, states, 'Everyone has the right to work, to free
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FEATURED Articles
The dream of a lifetime came true for Mohammad Faizan, when he
was enrolled to serve as a police inspector for a day (and paid!).
Rawalpindi Highway and Motorway Police offered the position to 18-
year-old Mohammad, who has thalassaemia, as part on an
awareness-raising campaign by the Pakistan Thalassaemia Welfare
Society to commemorate World Thalassaemia Day.
After a day of patrolling, monitoring speed limits and briefing
officials, Mohammad said the experience had given him great hope
for what life had to offer.
"I ask all thalassaemics to live happily and fight the disease
courageously," he said.
The occasion was no less moving for Mohammad's mother, who
confessed that she cried with joy at the sight of her son in a police
uniform.
"All I can say is may he live long and become a real officer one day,"
she said.
Police Academy: Dreams do come true!
choice of employment, to just and favourable conditions
of work and to protection against unemployment.'
This document has been further bolstered by the 2006
United Nations Convention on the Rights of Persons with
Disabilities, which has been ratified by a number of
countries, including India in 2007 and Cyprus in 2011. The
Convention binds signatories to meet the needs of
persons with disabilities, guaranteeing their full human
rights without discrimination and with equal opportunity,
including to work and employment. Article 27 states that
‘State parties recognise the right of persons with
disabilities to work on an equal basis with others,'
prohibiting discrimination on the basis of disability,
assuring equal remuneration for work of equal value, and
safe and healthy working conditions. Furthermore, state
parties must ‘Promote employment opportunities and
career advancement for persons with disabilities’.
The question, however, is whether a thalassaemia patient
is a person with a disability. In the case of Mr Singh, the
Prime Minister of India responded to initiatives by the
National Thalassaemia Association and the wider medical
community by proposing to ‘include thalassaemia as one of
the disabilities covered by the Disability Law, which
presumably arises from India’s ratification of the
Convention on the Rights of
persons with disabilities. The
definition of disability in the
Convention is stated in Article
1 as follows: ‘Persons with
disabilities include those who
have long-term physical,
mental, intellectual or sensory
impairments which in
interaction with various
barriers (attitudinal or
environmental) may hinder
their full and effective
participation in society on an
equal basis with others’.
If the Indian Medical Board, which rejected Mr Singh on the
grounds of thalassaemia, did not regard him as disabled,
i.e. as not having a long-term physical impairment, then it
should not have rejected him. If, on the other hand, they did
regard him as having an impairment, then their rejection
was illegal according to the Disability Law of the country.
The fact is that Mr Singh's application to the ICS was
rejected out of prejudice and ignorance, guided by the
belief that his health might pose a liability to the Service.
This is an illogical argument based on the idea that
thalassaemia is a fatal condition that prevents patients
leading a normal life. And such as argument then raises the
following questions:
• Does not the adult Mr Singh prove that modern
treatment has changed the disease outcome?
• Given his many years of consistent academic
achievement, in the course of which he has bettered the
majority of his peers, not prove him eminently suited to
performing the duties of a civil servant?
Employers and governments alike should carefully
consider the issue of health as a condition of employment.
In addition, medical boards should include specialists on
the conditions under consideration. Indeed, the medical
communities of many countries include a good number of
thalassaemia patients who are themselves medical
practitioners.
The Role of Thalassaemia Expert Centres andMedical Centres:
Traditionally, a doctor's duty to a patient ends with the
provision of appropriate medical treatment. In the case
of chronic conditions, psychosocial issues have
normally been left to others—or to patients
themselves. However, with education, employment and
other social issues increasingly recognised as
important factors in overall patient
well-being, this must change. After
all, it is the medical experts who are
most aware of how their treatment
can change patient survival and
quality of life. It is therefore also
their duty to raise awareness in
broader society. It is also their duty
to advocate their patients' interests
in whatever forums may affect
patient well being, including
educational authorities, potential
employers and government
services.
Above all, however, expert centres should ensure that
the treatment they recommend does not itself hinder
patients’ social integration. A key issue here is time of
transfusion. Even the best care providers in Europe
tend to offer transfusions in the morning (62%) or the
afternoon (32%), with only 5.3% arranged in the
evenings or at weekends. This means that thalassaemia
patients attending school or those who work are more
frequently absent, leaving a negative impression on
teachers or employers. In this way, health services are
at least partly responsible for the social prejudice
patients face.
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FEATURED Articles
The fact that people with
thalassaemia are living well
into adulthood means that
older patients now face
additional responsibilities in
terms of taking the initiative
to act as advocates for their
right to fulfil their dreams.
35
The question of how to bring service provision in line with
broader patient needs is a long -standing challenge that
will require concerted pressure from adult patient
populations and their support associations. TIF's recently
established Expert Patients Group should prove a
valuable source of help in spreading the message.
The Role of Patients in Changing AttitudesConcerning Employment:
The fact that people with thalassaemia are living well into
adulthood means that older patients now face additional
responsibilities in terms of taking the initiative to act as
advocates for their right to fulfil their dreams. However,
patients are not alone in this fight: associations and health
providers stand ready to offer support and encouragement,
just as their families have done in the past.
Perhaps this is provocative. Every individual with
thalassaemia spends each day fighting. Who has the right
to say, do more?
But I know we can do more. Everyone—TIF, national
thalassaemia associations, friends and families—can
learn more about the legal rights of thalassaemics and
work to ensure that these are enforced. Most of all, it is
the duty of us as thalassaemics to recognise our duty to
ourselves and to society.
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My name is Somdutta, and I am a 22-year-old with tha-
lassaemia intermedia. But that is not my identity.
I am from Kolkata, working in Delhi as Deputy Manager
in Corporate Communication at one of India's leading
foreign trade enterprises. I have been a first-class stu-
dent all my life, always balancing work and play to the
best of my ability, and I share the common desire to lead
an independent, productive life by dint of merit and hard
work. I expect to go places.
But my experience in finding employment has not all
been plain sailing. Despite having had the good fortune
to receive a top-class education, I almost missed the
chance of a job that I am ideally suited for, because I
have thalassaemia.
After graduating from Presidency College, Kolkata with
Honours in English Literature, followed by a post-grad-
uate diploma in Advertising and Public Relations from
the Indian Institute of Mass Communication, New Delhi,
I received several job offers. The one I wanted—a pres-
tigious public sector job—seemed the ticket to every-
thing I hoped to achieve. But there was a catch I had not
foreseen.
In the course of a medical assessment, I declared myself
to have thalassaemia intermedia. I did so with no reser-
vations, knowing that the condition was of no conse-
quence to my fulfilling my life as a professional. After a
long wait, I began calling the office every few days,
always receiving the same non-committal answer.
My frustration grew and I found myself putting my life
on hold, crawling from one day to the next. The media
was full of claims and counter-claims in the case of
Sukhsohit Singh (see above), who was fighting for his
right to a place in the Uttar Pradesh civil service. I began
to despair.
After several months without progress, my parents and
I decided to visit the Thalassaemics India office in Delhi.
Suddenly, a much needed support system opened up.
Shobha Tuli immediately took up my case, and her
encouragement did much to dispel my sense of hope-
lessness.
Mrs Tuli contacted my prospective employer, request a
face-to-face meeting with the HR Manager. By the end
of the week she had had her meeting, followed up by a
letter in which she cited the Universal Declaration of
Human Rights, emphasising the fact that thalassaemics
had the same ability—and right—to work.
The involvement of a credible and heavyweight non-prof-
it third-party acted as a catalyst, resulting in a percepti-
ble change in the attitudes of my prospective employers.
I was asked to meet the company’s Chief Medical Officer,
with whom I had a friendly chat, followed by another
health check, this time with a specialist a haemato-
oncologist. I was pronounced medically fit, with a recom-
mendation that I receive regular check-ups, and was
finally presented with a formal job offer two days after
India celebrated 65 years of independence.
With help from the right people, I won my battle. But we
are a long way from winning the war. For every person
who refuses to give in to defective bureaucracy, there
are many without the resources to go on. There is scant
knowledge about thalassaemia in Indian society and, as
they say, a little knowledge is a dangerous thing.
Discrimination against the condition is rampant, partic-
ularly amongst employers. The fact that people with
thalassaemia can and should lead normal lives still
eludes many. That is is a problem that deserves the
immediate attention of policy-makers.
My Identity as a Thalassaemic
We provide valuable medicines to rare disease patients
SOIUKP1110 - Date of Preparation: March 2011
37
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A new study by researchers in France has demonstrated
the clinical feasibility of a stem cell-based alternative to
donor blood-derived red blood cells, raising the hope that
patients in need of a blood transfusion might one day
become their own donors.
The authors of the research, Marie-Catherine Giarratana,
Hélène Rouard et al, recently published their findings in
Blood, the Journal of the American Society of Hematology.
According to the article, the team successfully generated
red blood cells (RBCs) from stem cells, injecting the RBCs
back into the same individual. The transfusion,
administered to a volunteer, showed that after five days
the survival rate of the cultured red blood cells (cRBCs)
was 94-100 per cent. At 26 days, the survival rate stood at
41-63 per cent—consistent with a conventional half-life
of native red blood cells of 28 days.
This research marks the first time researchers have
successfully injected a human donor with cRBCs created
from human haematopoietic stem cells—the cells from
which all blood cell types are generated, including RBCs.
Annual global demand for donor blood currently stands at
90 million units of RBCs—a figure that rises every year,
just as the number of blood donors is falling. At the same
time, the risks of transfusion-transmitted disease
remains extremely high. These findings are therefore
doubly welcome, showing the way towards increasing
sources of haemoglobin while dramatically reducing the
associated dangers of transfusion.
The reported findings of this research have demonstrated
proof of principle. It is now possible to envisage a time
when thalassaemia patients could receive ‘younger’ red
blood cells with a high HbF content, thus lengthening the
time between transfusions and reducing iron overload. In
addition, patients would enjoy the benefits of receiving
RBCs from a single, ‘clean’, donor, thus possibly avoiding
the formation of antibodies, as well as the risk of disease.
Although a great deal more research is needed, this is
good news indeed.
Synthetic blood: An end to blood drives?
38
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Ali Taher is a Professor of Medicine with a special interest in haemoglobinopathies. A Consultant Haematologist at
the Chronic Care Centre, Beirut since 1993, Prof Taher is a leading TIF partner and recently received the Medical
Alumni Chapter Gold Award for outstanding contributions to his field. Prof Taher has published hundreds of articles
in leading scientific journals, as well as two books and three book chapters. He is co-author of TIF's Guidelines for
the Clinical Management of Thalassaemia.
Department of Internal Medicine, American University of
Beirut Medical Centre, Beirut; and the Chronic Care
Centre, Hazmieh, Lebanon
Abstract: Our understanding of the processes underlying
the disease process in patients with β-thalassaemia
intermedia (TI) has substantially increased over the past
couple of decades. There are a number of options avail-
able for managing patients with TI, with several studies
highlighting the benefits and risks of each approach.
However, until solid evidence-based guidelines are avail-
able, individualised treatment should be maintained.
β-thalassaemia exhibits across a wide spectrum of clinical
severity. At one extreme are patients with a clinically silent,
mild anaemia, known as β-thalassaemia minor. At the other
are patients with β-thalassaemia major, who present
severe anaemia from an early age and require regular
blood transfusions and iron chela-
tion therapy throughout their lives.
The term β-thalassaemia interme-
dia (TI) refers to those cases that
fall between these two extremes,
and who usually present the need
for medical attention later in life.
The clinical characteristics of TI are
very heterogeneous, making the
understanding and management of
the disease a challenging task. This
article aims to assess significant advances made to that
end over the past 20 years.
It is now known that in the case of TI, several genetic and
environmental factors play a part in determining the
severity of anaemia and clinical complications of the dis-
ease (phenotype). Examples of genetic factors modulating
the severity of TI are:
• a broad diversity of mutations affecting the β-globin
genes;
• co-inheritance of α-thalassaemia;
• several newly discovered genes involved in modifying
the γ-chain response (which alters the production of
foetal haemoglobin);
• and genes directly involved in the expression of clinical
complications (1).
Significant environmental factors may include malaria infec-
tion. The mechanism of disease in TI patients is attributed to
three main factors: inability of the bone marrow to produce
normal red blood cells (ineffective erythropoiesis); chronic
anaemia and destruction of red blood cells (haemolysis); and
increased absorption of iron from the gut. Recent work has
identified several genetic mutations and internal factors
that play a part in regulating these processes, which could
be future targets for therapy (2). The combination of these
mechanisms leads to several clinical complications in TI,
which may not be as frequently observed in patients with β-
thalassaemia major and which could cause significant mor-
bidity, therefore warranting immediate attention.
Ineffective erythropoiesis is associated with skeletal
deformities and osteoporosis, as
well as compensatory formation of
masses elsewhere in the body
(extramedullary haematopoiesis)
that can cause mechanical prob-
lems. Haemolysis has mainly been
associated with enlargement of the
spleen; however, recent evidence
suggests that haemolysis, along
with other factors, causes a high
frequency of thrombosis in patients
with TI and may explain other com-
plications such as pulmonary hypertension (increased
pressure in lung vessels) with secondary right heart fail-
ure. The extra iron absorbed from the gut can accumulate
in the liver and (less so) in the heart, as recently observed
through the application of MRI technology, and may even-
tually lead to significant liver disease (3).
The management of patients with TI remains a challenge.
There are currently no solid, evidence-based guidelines
for management, and treatment relies mainly on constant,
expert observation of each individual patient.
Once a diagnosis of TI is established, the patient should be
assessed over a period of several months before beginning
A closer look at β-thalassaemia intermedia Ali Taher, MD and Khaled Musallam, MD
Once a diagnosis of TI is
established, the patient
should be assessed over a
period of several months
before beginning any form
of treatment.
39
any form of treatment. No treatment modality, especially
transfusion therapy, should be embarked upon too hastily.
Many patients with TI are unnecessarily subjected to a life-
time of regular blood transfusions, particularly those that
present an unusually low level of haemoglobin during a
period of infection. In such situations, it is often appropri-
ate to administer transfusions only in the acute case, and
not to immediately commit to a longer-term transfusion
programme. Moreover, the need for transfusion is depend-
ent not only on the severity of the anaemia but also on the
patient’s general well being, particularly with respect to
activity, growth, development and the early appearance of
skeletal changes or other disease-related complications.
A recent study of 584 patients with TI from the Middle
East region and Italy (the Optimal Care study) demon-
strated the beneficial role of transfusions in decreasing
various complications in TI, such as thrombosis, pul-
monary hypertension, heart failure, leg ulcers and
extramedullary haematopoiesis (4). The same study also
highlighted a higher risk of many disease complications
after splenectomy, suggesting that the recommendation
of such a procedure in TI requires careful consideration.
The study also showed a beneficial role for iron chelation
therapy in TI. However, the efficacy and safety of this
treatment is currently being evaluated in a large study
using the oral iron chelator, deferasirox. Finally, the
promising role played by agents that increase the produc-
tion of foetal haemoglobin (such as hydroxyurea) has been
documented in several studies, however further research
in this area is essential.
Although TI is considered a milder form of the disease at
initial presentation and diagnosis, TI patients are at risk
of developing serious complications, especially as they
grow older. Further clinical studies are therefore
required, to assess the optimal type and timing of treat-
ment to be offered to this group of patients, to avoid dis-
ease-related morbidity.
Key References
1. Sankaran VG, Lettre G, Orkin SH, Hirschhorn JN.
Modifier genes in Mendelian disorders: the example of
hemoglobin disorders. Ann N Y Acad Sci,
2010;1214(1):47-56.
2. Gardenghi S, Grady RW, Rivella S. Anemia, ineffective
erythropoiesis, and hepcidin: interacting factors in
abnormal iron metabolism leading to iron overload in
β-thalassemia. Hematol Oncol Clin North Am,
2010;24(6):1089-107.
3. Taher A, Hershko C, Cappellini MD. Iron overload in tha-
lassaemia intermedia: reassessment of iron chelation
strategies. Br J Haematol, 2009;147(5):634-40.
4. Taher AT, Musallam KM, Karimi M, El-Beshlawy A et al.
Overview on practices in thalassemia intermedia man-
agement aiming for lowering complication rates across
a region of endemicity: the Optimal Care Study. Blood,
2010;115(10):1886-92.
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Dr Chi Kong Li specialised in Paediatric Haematology/Oncology/BMT at the University of Hong Kong, followed by
further training at John Radcliffe Hospital, Oxford, Great Ormond Street Hospital, London, and Fred Hutchison
Cancer Research Centre, Seattle. As Vice-chairman of China's Paediatric Haematology Committee, he has a partic-
ular interest in childhood leukaemia, haematopoietic stem cell transplantation and thalassaemia. He is Editor of the
medical journals Paediatric Blood Cancer and the Chinese Journal of Paediatric Haematology and Oncology, and is
actively involved in the development of paediatric haematology in mainland China.
How to select iron chelators in 2012By Chi Kong Li
Vice-chairman, Paediatric Haematology Committee, China
Abstract: With three iron chelators available in many
countries, a great number of thalassaemia patients now
have a selection of therapies to choose from. For many,
the long-sought after option is oral, doing away with the
need for invasive injections. However, other aspects of
treatment are also important, including socio-economic
factors, patient age, severity of iron overload and the
organs affected—not to mention availability. New oral
iron chelators currently under study offer the hope that
such therapy will one day be a reality for all thalassaemia
major patients, irrespective of age or income.
The three iron chelators currently available are deferriox-
amine, deferiprone and deferasirox. Deferrioxamine, which
is administered sub-cutaneously, has the longest history
of clinical use, and its efficacy and side effects are well
known to clinicians. The oral chelator deferiprone has
gained in popularity following its approval by the
European Medicines Agency (EMA) and boasts a superior
ability to protect the heart, which has been shown to
improve survival in many studies. The combined use of
deferrioxamine and deferiprone is now recommended by
many clinicians for patients who have severe iron over-
40
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load, especially cardiac overload as demonstrated by MRI.
The third chelator, deferasirox, is also oral, providing
patients and their doctors with a further alternative for
the removal of excess body iron.
In most western countries, the number of new cases of
thalassaemia major is decreasing or is maintained at very
low incidence. However, in countries lacking comprehen-
sive prenatal screening programmes, the number of new
cases born each year remains significant. These transfu-
sion-dependent children usually develop excess body iron
and begin chelation therapy at 2-3 years of age.
The standard treatment is the subcutaneous administra-
tion of deferrioxamine in the region of the abdomen,
demanding a great deal of young patients and of the par-
ents who must come to terms with the difficult task of
inserting a needle into their child.
The oral chelator deferasirox can be given to children as
young as 2. However, the drug remains prohibitively
expensive for most families in developing countries,
despite the manufacturer's efforts to reduce costs.
The other oral chelator, deferiprone, is not suitable for
children under the age of 5. Previously only available in
tablets, deferiprone has recently been introduced in solu-
tion form. A six-month clinical study of deferiprone solu-
tion administered to 59 children under the age of 6, con-
ducted in Egypt, Malaysia and Indonesia, demonstrated
short-term efficacy in reducing serum ferritin. Assuming
the same biological efficacy as the tablet form,
deferiprone solution certainly benefits young children
most, and they may not require injectable iron chelation in
the future. However, the long-term effects on young chil-
dren of deferiprone, including side effects, are still
unknown. Two of the 59 children receiving deferiprone
solution developed agranulocytosis within the six month
period, while 6% developed mild neutropenia. It is not
known whether the incidence of agranulocytosis increas-
es with longer duration of deferiprone treatment.1 Joint
pain or arthralgia occurred in 4% of patients; one patient
experienced severe joint pain that required discontinua-
tion of deferiprone treatment. Whether young children
with growing skeletal system will be more prone to joint
toxicity is a question that requires further study. The accu-
mulation of more clinical data over a longer period are
therefore required before recommending deferiprone as
first-line treatment in young children.
A number of studies of older patients over the past 10 years
have concentrated on the prevention of cardiac toxicity and
thus improved survival. The administration of deferiprone
alone or in combination with deferrioxamine has been
shown to be more effective in removing excess iron from
the heart. Deferasirox has also been the subject of careful
study regarding cardiac protective effect. A one-year study
showed that mild to moderate cardiac iron overload
patients benefit more from deferasirox as the T2* meas-
ured on cardiac magnetic resonance with better improve-
ment of T2*. Follow-up studies indicated further improve-
ment in cardiac iron status at two and three years.2,3
The process of removing iron from heart cells is slow and
it may take several years to achieve very good clearance
of heart iron. In patients with severe heart iron overload,
as demonstrated by T2* < 6ms, the chance of developing
heart failure is 47% in one year.4 Such patients should
receive intensive iron chelation to prevent heart failure,
with most experts recommending a combination of
deferiprone and deferrioxamine. However, studies have
shown that about 25% of patients on combination treat-
ment have to stop combined treatment, with agranulocy-
tosis cited as one of the main reasons. The combined uses
of deferrioxamine and deferasirox, or deferasirox and
deferiprone, have not been subject to full investigation,
with the exception of a few individual cases. However,
patients are more likely to comply with a treatment of
combined oral iron chelators, making it an option that may
thus improve survival. Deferiprone has better cardiac pro-
tective effect, while deferasirox appears to provide good
clearance of liver iron. The combined use of both agents
may therefore achieve better overall control of total body
iron. Lower doses of either agent used in combination may
be possible. However, careful study is required into the
optimal recommended dosage of each, and the toxicity
profile of combined chelators.
References:
El Alfy M, Sari TT, Chan LL; Tricta F, El-Beshlawy A. The
Safety, Tolerability, and Efficacy of a Liquid Formulation
of Deferiprone in Young Children With Transfusional Iron
Overload. Journal of Pediatric Hematology/Oncology.
32(8):601-605, November 2010.
Pennell DJ, Porter JB, Cappellini MD, et al. Continued
improvement in myocardial T2* over two years of
deferasirox therapy in β-thalassemia major patients with
cardiac iron overload. Haematologica 2011; 96: 48-54.
Continued Improvement and Normalization of
Myocardial T2* in Patients with β-thalassemia Major
Treated with Deferasirox (Exjade®) for up to 3 Years. 2010
Annual Meeting of American Society of Hematology,
Poster #: III-1055.
Kirk P, Roughton M, Porter JB, et al. Cardiac T2*
Magnetic Resonance for Prediction of Cardiac
Complications in Thalassemia Major. Circulation
2009;120;1961-1968.
41
Abstract: The reported success of hydroxyurea in treating
patients with sickle cell disease has led to a focus on its
possible benefits for patients with β-thalassaemia major,
with studies finding reduced blood transfusion
dependency. There are also indications of its beneficial
use in the treatment of thalassaemia intermedia.
However, further research is needed into the possible
side-effects of the long-term use of hydroxyurea.
Hydroxyurea (HU) is a virtually tasteless, white crystalline
powder with the chemical formula of CH4N2O2 (Figure 1).
It is an antimetabolite that can promote foetal
haemoglobin (HbF), and is approved by the US FDA for
treatment of patients with cancers such as chronic
myelocytic leukemia (1). It can also form part of an anti-
HIV regimen and is sometimes used to treat psoriasis
(2,3). Available as a capsule for oral administration, HU is
also known as hydroxycarbamide and is marketed under
the registered trademarks Hydrea and Droxia.
A dependency on regular blood transfusions and resulting
iron overload are key complications of β-thalassaemia
major (TM), and reducing the need for transfusions can
play an important part in improving the safety and quality
of life of TM patients. It has been suggested that the
administration of HU in patients with β-thalassaemia
major (TM) has beneficial effects to this end, via the
mechanism of neutralizing excess α-chains through the
production of γ-chains, resulting in partial correction of
ineffective erythropoiesis.
Studies of the efficacy of HU in TM have revealed a
modest increase in Hb levels, reducing blood transfusion
dependency or even altogether eliminating the need for
transfusions. One study (2004) showed a good response
to HU in transfusion-dependent TM patients in Iran, in
whom the Hb level was kept above 9.5 g/dL without
transfusion (8). Studies have also shown polymorphism
for gG XmnI (gG ã 158 C > T), homozygous for β-globin
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Hydroxyurea management in β-thalassaemia intermediaDr Mehran Karim
Haematology Research Centre, Shiraz University of Medical Sciences, Shiraz, Iran
Email: [email protected]
Prof Mehran Karimi
Prof Karimi is Professor of Paediatric Haematology-Oncology and Director of the Haematology Research Centre
at Shiraz University of Medical Science (SUMS), working in the field of thalassaemia and coagulation. After com-
pleting his studies at SUMS in haematology with a focus on thalassaemia and haemophilia, he completed a fel-
lowship at the University of Milan under the supervision of Prof PM Mannucci. He is a member of the
International Society on Thrombosis and Haemostasis (ISTH) and has twice been awarded the Razi Festival prize
for best researcher, in 2001 and 2008. Prof Karimi has authored over 165 articles and 155 abstracts, and is a reg-
ular peer-reviewer for a number of international journals.
Results Length of therapyHU dose
(mg/kg/d)No of
patientsReferences
20(44.5%) goodresponse with 1.5 gr/dl
in Hb level1 year
16.3±2.3 Raised to17.4±2.4
45 Bradi et al.
9 good response with Hblevel of mean 8.2±0.7
gr/dl and transfusion free46±25 months 10.9±3 11 Korean et al.
Table 1: Results of two newly studies in the efficacy of HU in β-thalassemia major patients.
42
mutations [IVS-II 1 (G-A) or IVS-I 5 (G > C)],
and indicated that α-thalassaemia deletions
have a strong influence on the clinical
response to HU therapy (6,8). Table 1 shows
the results of two new studies regarding the
efficacy of HU in TM patients (6,9).
β-thalassaemia intermedia (TI)
The term β-thalassaemia intermedia (TI)
refers to patients whose clinical phenotype
is milder than that of thalassaemia major
(TM). The clinical course of TI is
characterised by several complications that
can be prevented by accurate follow-up.
Despite chronic anaemia, individuals with TI
do not require regular transfusion, except in
association with concurrent illness (7).
Furthermore, because the α/β-globin
imbalance in β-thalassaemia intermedia (TI)
is less than that in TM, the use of HU may be
expected to result in better clinical responses and
haematological improvements (5), as reported by a
number of studies (6-7).
Although many of these studies included only a small
number of patients, we reported clinically significant
responses to HU in a larger group of TI patients from Iran
(7). The study divided 163 TI patients into two groups,
according to blood transfusion dependency. Group I
consisted of 120 patients receiving regular blood
transfusion, and group II consisted of 43 patients with
long-interval transfusion or no history of transfusion. All
patients were treated with 8–12 mg/kg/d of HU
administered orally once a day.
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Results Length of therapyHU dose
(mg/kg/d)No of
patientsReferences
45.9% transfusion freeor ↑Hb>2gr/dl
24.3%↓transfusion to50% or ↑Hb1-2gr/dl
4-36months
10-20(mg/kg/d)
37 Dixit et al.
Mean Hb ↑ 1.5 gr/dl 1 year5-30
(mg/kg/d)
18 splenecto-mozed
untransfusedMancuso et al.
↓ the size of extramedullarly
hematopoiesis mass andcured leg ulcers
3 months1000
(mg/d)6 Gamberini et al.
Table 2: Some studies about the efficacy of HU treatment in β-thalassemia intermedia.
Figure 1: Chemical structure of HU
43
A total of 149 patients (91.4%) showed a positive
response to HU therapy, with the mean Hb level in Groups
I and II maintained at 9.5 and 9.6 g/dL, respectively. HU
was also associated with a marked increased in MCV and
MCH. After HU treatment, 97% of patients described an
increase in exercise tolerance and sense of well-being. No
significant facial changes were observed. Spleen size in
non-splenectomised patients remained unchanged in 83%
of patients. In addition, HU therapy was shown to
decrease complications in cardiac function and to reduce
pulmonary hypertension in TI patients (13). Table 2 shows
the results of a number of other studies into the effects of
HU therapy in TI (10-12).
Another recent study evaluated the adverse effects of
low-dose HU (8–12 mg/kg/d) in TI patients who had been
treated with HU for a period of 10 years. Most recorded
adverse effects were dermatological, neurological or
gastrointestinal, and none required the discontinuation of
therapy. There were no reports of haematologic toxicity,
bone marrow suppression or secondary malignancies
during HU treatment (14). These results indicated that
low-dose HU therapy in thalassaemia may be well
tolerated without serious side effects. However, further
research into the possible adverse effects of long-term
HU therapy is required.
The administration of HU has been demonstrated to be
effective in increasing Hb levels in TI patients, reducing
transfusion dependence as well as the incidence of
osteoporosis, extramedullary haematopoiesis, skeletal
deformities and splenomegaly, while increasing energy
levels. Some studies have also demonstrated positive
clinical and haematological responses to HU in TM
patients, making it a possible useful alternative to blood
transfusion for some patients—one that is inexpensive
and delivered orally.
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Key references:
Katzung B, Masters S, Trevor A. Basic and Clinical
Pharmacology, 11th Edition. 2009.
Kumar B, Saraswat A, Kaur I. Rediscovering hydroxyurea:
its role in recalcitrant psoriasis. Int J Dermatol.
2001;40(8):530–534.
Biron F, Ponceeau B, Bouhour D, Boibieux A, Verrier B,
Peyramond D. Long-term safety and antiretroviralactivity
of hydroxyurea and didanosine in HIV-infected patients. J
Acquir Immune Defic Syndr. 2000;25(4):329–336.
Charache S, Terrin ML, Moore RD, et al. Effect of
hydroxyurea on the frequency of painful crises in sickle
cell anemia. Investigators of the multicenter study of
hydroxyurea in sickle cell anemia. N Engl J Med.
1995;332(20):1317–1322.
Karimi M. Hydroxyurea in the management of thalassemia
intermedia. Hemoglobin. 2009;33 Suppl 1:S177-82.
Bradai M, Pissard S, Abad MT, Dechartres A, Ribeil JA,
Landais P, et al. Decreased transfusion needs associated
with hydroxyurea therapy in Algerian patients with
thalassemia major or intermedia. Transfusion. 2007
Oct;47(10):1830-6.
Karimi M, Darzi H, Yavarian M. Hematologic and clinical
responses of thalassemia intermedia patients to
hydroxyurea during 6 years of therapy in Iran. J Pediatr
Hematol Oncol. 2005 Jul;27(7):380-5.
Yavarian M, Karimi M, Bakker E, Harteveld CL, Giordano
PC. Response to hydroxyurea treatment in Iranian
transfusion-dependent beta-thalassemia patients.
Haematologica. 2004;89(10):1172–1178.
Koren A, Levin C, Dgany O, Kransnov T, Elhasid R, Zalman
L, et al. Response to hydroxyurea therapy in beta-
thalassemia. Am J Hematol. 2008; 83(5):366-70.
Dixit A, Chatterjee TC, Mishra P, Choudhry DR, Mahapatra
M, Tyagi S, et al. Hydroxyurea in thalassemia intermedia--
a promising therapy. Ann Hematol. 2005;84(7):441–6.
Mancuso A, Maggio A, Renda D, Di Marzo R, Rigano P.
Treatment with hydroxycarbamide for intermedia
thalassaemia: decrease of efficacy in some patients during
long-term follow up. Br J Haematol. 2006;133(1):105–6.
Gamberini MR, Fortini M, De Sanctis V. Healing of leg
ulcers with hydroxyurea in thalassaemia intermedia
patients with associated endocrine complications. Pediatr
Endocrinol Rev. 2004;Suppl 2:319–22.
Karimi M, Borzouee M, Mehrabani A, Cohan N.
Echocardiographic finding in beta-thalassemia intermedia
and major: absence of pulmonary hypertension following
hydroxyurea treatment in beta-thalassemia intermedia.
Eur J Haematol. 2009;82(3):213–218.
Karimi M, Cohan N, Moosavizadeh K, Falahi MJ,
Haghpanah S. Adverse effects of hydroxyurea in beta-
thalassemia intermedia patients: 10 years' experience.
Pediatr Hematol Oncol. 2010 Apr;27(3):205-11.
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TIF REGIONAL Conferences
1st Pan-Asian Conferenceon Haemoglobinopathies
ROYAL ORCHID SHERATON HOTEL & TOWERS
Bangkok, Thailand / 8-10 February 2011
In collaboration with
Thalassaemia
International
Federation
Organised by
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ANNOUNCEMENT
The first TIF Pan-Asian Conference on Haemoglobinopathies has been postponed, due to catastrophic
flooding in Bangkok. Originally scheduled to be held on 3-5 November 2011 at the Royal Orchid Sheraton
Hotel and Towers, the Conference will now be held on 8-10 February 2012.
The Conference has been organised by TIF in cooperation with the Thalassaemia Foundation of Thailand,
Mahidol University, Thailand and the Thai Ministry of Health.
The first TIF Pan-Asian Conference on Haemoglobinopathies takes as its theme ‘Patients and Health
Professionals together for Optimal Care’. The event aims to bring together patients and their families,
medical professionals, policy-makers and other interested parties from the South-East Asia and
Western-Pacific regions, to exchange expertise regarding best practice in clinical care, patient
empowerment and disease prevention. The Conference will also provide an opportunity to assess the
status of current control strategies in Asia, and to discuss ways of ensuring that haemoglobin disorders
are prominent on national health agendas.
A ceremony in honour of Prof Khunying Soodsarkorn Tuchinda, who is to be presented with a Lifetime
Achievement Award, has been organised to coincide with Prof Tuchinda's 90th birthday.
Inaugural Ceremony: ROYAL ORCHID BALLROOM - 1
Chairpersons: Panos Englezos & Suthat Fucharoen
TIME: 16:00 – 19:00
16:00 - 16:10 Welcome Message from the President of the Thalassaemia Foundation of Thailand
16:10 - 16:20 Welcome Message from the President of Mahidol University
16:20 - 16.30 Welcome Message from the Ministry of Health of Thailand
16:30 - 16:40 Welcome Message from the WHO-SEARO/WPRO – Maureen Elisabeth Birmingham
16:40 - 17:00 Welcome Message from the President of the Thalassaemia International Federation – Panos
Englezos
COFFEE BREAK: 17:00 – 17:30
TIME:17:30 - 19:00
Chairpersons: Androulla Eleftheriou & Shobha Tuli
17:30 -18:00 Prof. Sir David Weatherall - Keynote speech ‘The inherited Diseases of Haemoglobin are an
emerging Global Health Burden’
18:10 - 18:30 Award Ceremony - Celebrating the 90th Birthday and Life Achievement in Thalassaemia of Prof
Khunying Soodsarkorn Tuchinda
18:30 –19:00 Introduction to Sultan Bin Khalifa International Thalassaemia Award
ROYAL ORCHID BALLROOM8 November 2011
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TIME: 08:00-09:00 Registration
TIME: 09:00-10:00
Session 1 – Country Reports – Current Strategies of Prevention and Management of Haemoglobin Disordersin Asian and South East Asian Country – Country reports
Chairpersons: Suthat Fucharoen & John Wood
09:00-09:20 Global Epidemiology of β-, HbE, α-thalassaemia and SCD – Specific Reference to Asia –Vip Viprakasit (Thailand)
09:20-09:40 Overview of Genotypes/Phenotypes of Thalassaemias in Asia – Suthat Fucharoen (Thailand)
09:40-10:00 Prevention programmes for haemoglobinopathies – Appropriate for ASIA – Existing and needed –Policy and Laboratory Perspective – Anuja Premawardhena (Sri Lanka)
TIME: 10:00 – 11:30
Session 2 – Current Situation in Control (Prevention & Management) Strategies and Health Systems in Asia
Chairpersons: Roshan Colah & Ne Win
10:00-10:10 Representative Ministry of Public Health of Thailand - Thailand
10:10-10:20 Jovarian Mannan - Pakistan
10:20-10:30 Roshan Colah – India
10:30-10:40 Ajit Rayamajhi - Nepal
10:40-10:50 Ransnayake Mudiyanse – Sri Lanka
10:50-11:00 Amalia P. Wahidiyat - Indonesia
11:00–11:10 Farzana Khatoon - Maldives
11:10-11:20 Ne Win – Myanmar
11:20-11:30 Ahmed Waqar Khan - Bangladesh
COFFEE BREAK: 11:30-12:00
TIME: 12:00 – 13:00
Session 3 – Current Situation in Control (Prevention & Management) Strategies and Health Systems in Asia
Chairpersons: Ahmed Waqar Khan & Hisham Shah Mohammed Ibrahim
12:00-12:10 Chen Ping – People’s Republic of China
12:10-12:20 Vincent Lee - People’s Republic of China – Hong Kong
12:20-12:30 Sourideth Sengchanh – Republic of Laos
12:30-12:40 Hisham Shah B. Mohammed Ibrahim - Malaysia
12:40-12:50 Ernesto d’Y Yuson - Philippines
12:50-13:00 Law Hai Yang - Singapore
LUNCH BREAK: 13:00 – 14:30
AFTERNOON SESSIONS:- TIME: 14:30– 17:30
Session 4 – Current situation in Control (Prevention & Management) strategies and Health Systems in Asia
14:30-14:40 Nguyen Anh Tri - Vietnam
14:40-14:50 Piseth Prak Raingsey – Kingdom of Cambodia
14:50-15:00 Sant-Rayn Parischa - Australia
15:00-15:10 Meng Yao Lu - Taiwan
15:10-15:30 Ching-Tien Peng - Genetic screening and prenatal diagnosis of thalassaemias andhaemoglobinopathies in Taiwan today
COFFEE BREAK: 15:30 – 16:00
Session 5 – OUTCOMES & CONCLUSION
Chairpersons: Suthat Fucharoen, Michael Angastiniotis, Androulla Eleftheriou, Panos Englezos, Ramli Yunus &Shobha Tuli
TIME: 16:00– 17:30
Interactive Discussion: Outcomes, Conclusions and presentation of strategic plan for Asia (2012-2016)
ROYAL ORCHID BALLROOM - 19 February 2012
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TIME: 09:00-17:45 CLINICAL MANAGEMENT
Session 6 – Management and policies: State-of-the-art in the Treatment of:
TIME: 09:40-10:40
Chairpersons: Miguel Abboud & Elisabeth George
09:00-09:30 β-thalassaemia major – John B. Porter (UK)
09:30-10:00 Non-transfusion dependent Thalassaemias- β-thalassaemia Intermedia and HbE/β-thal types –
Elisabeth George (Malaysia)
10:00-10:30 α-thalassaemias -HbH type – Vip Viprakasit (Thailand)
COFFEE BREAK: 10:30 – 11:00
Session 7 – Management and policies: State-of-the-art in - & WHO Policies:
TIME: 11:00-12.30
Chairpersons: Vip Viprakasit & John B. Porter
11:00-11:30 The Treatment of Sickle Cell Disease – Miguel Abboud (Lebanon)
11:30-12:00 A multi-disciplinary approach: Centres of Reference – Michael Angastiniotis TIF
12:00:12:30 WHO and Policies on NCDs and Hb Disorders – Androulla Eleftheriou – TIF
LUNCH BREAK: 12:30 – 13:30
Session 8 – Advances in Curative methods
TIME: 13:30-15:45
Chairpersons: Philip Leboulch & Khunying Soodsarkorn Tuchinda
13:30-13:45 Gene therapy – Philip Leboulch (USA)
13:45-14:00 Haematopoietic stem-cell transplantation in developing countries – Suradej Hongeng (Thailand)
14:15-14:30 Fetal globin induction in Haemoglobin Disorders – Suthat Fucharoen (Thailand)
14:30-14:45 Understanding the molecular mechanisms leading to depression of γ-globin gene expression –
Jim Vadolas (Australia)
14:45-15:00 Copy Drugs/Counterfeit Medicines – Carlo M. Nalin (USA)
15:00-15:15 MRI-based technology for developing countries – John Wood (USA)
15:15-15:30 MRI-based Monitoring tools for Iron Chelation – Pairash Saiviroonporn (Thailand)
15:30-15:45 Stem cell transplantation in Developing countries – BMT Experience in Pakistan – the cost-effective
model- Lawrence Faulkner (Italy)/ )/ Naila Yaqub (Pakistan)
COFFEE BREAK: 15:45 – 16:30
TIME: 16:00– 17:15
Session 9 – FUTURE PROSPECTS
Chairpersons: Suthat Fucharoen, Michael Angastiniotis & Panos Englezos
TIME: 16:30– 17:45
16:30-17:00 New advances and future outlook in the management and cure of Haemoglobin disorders –
Philippe Leboulch (France/USA)
17:00 – 17:30 Patients as active and valuable partners in achieving progress and advances – Panos Englezos (TIF)
17:30 – 17:45 Role and commitment of National Health Authorities in Asia towards Chronic Genetic diseases –
Representative from the Thai Ministry of Health
ROYAL ORCHID BALLROOM - 110 February 2012
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UPCOMING Events
Event Venue DateWorkshop for Development of National Guidelines for Sickle CellDisease
Nicosia, Cyprus January 12-13
EFGCP Annual Conference 2012 - Informed Consent:
How Less could be more: Effecting a paradigm shift so
we do inform patients
Brussels, Belgium January 24-25
EUCERD Winter Meeting Luxembourg,Luxembourg
January 26 – 27
ENERCA 6th Executive Meeting Paris, France February 2
ESH - ENERCA Training Course on ‘Diagnosis and Management of very
rare anaemias: a challenge’Paris, France February 3-4
1st Pan-Asian Conference on Haemoglobinopathies and Rare Anaemias Bangkok, Thailand February 8-10
SCS 3rd Patients Educational Seminar London, UK February 22
Eurordis Gala Dinner: Celebrating Rare Disease Day 2012 Brussels, Belgium February 29
IAPO 5th Global Patients Congress London, UK March 17-19
European Conference on Rare Diseases and Orphan Drugs Brussels, Belgium May 23-25
2nd World Congress on Controversies in Haematology (COHEM) Barcelona, Spain October 4-6
3rd Pan European Conference on Haemoglobinopathies Limassol, Cyprus October 24-26
EVENTS CALENDAR 2012
A new e-Msc course launched in 2009 byUniversity College London (UCL) and TIFUCL in one of the most prestigious uni-versities in the world and one of the fewto have the necessary infrastructure andexpertise to teach through e-learning.
This course represents a uniqueopportunity for health professionalsto specialise in haemoglobinopathiesonline with minimun disruption toprofessional and personal lives.
It is designed to meet the needs of awide range of medical professionals,including:• medical graduates interested inhaemoglobinopathy (general physi-cian, specialists such as pediatricians,haematologists, clinical geneticists,obstetricians/gynaecologists, behav-ioural scientists)
• Science graduates interested in med-ical research related to haemoglo-binopathy and genetics
• other healthcare professionals interest-ed in haemoglobinopathy (counsel-lors, clinical psychologists, nurse spe-cialists and midwives)
SUPPORTED BY:
For further information, please visitwww.instituteforwomenshealth.ucl.ac.uk or www.thalassaemia.org.cy/msc.html or contact TIF or UCL:
UNIVERSITY COLLEGE LONDON“MSc in Haemoglobinopathy”88-96 Chenies MewsLondon WC1E 6HX, United KingdomTel: +44 (0)20 7679 6060Fax: +44 (0)20 7380 9984Email:[email protected]
THALASSAEMIA INTERNATIONALFEDERATION“MSc in Haemoglobinopathy”31 Ifigenias Str., 2007 Strovolos, CyprusTel: +357 22 319 129Fax: +357 22 314 552Email: [email protected]
MSc COURSE IN HAEMOGLOBINOPATHYMake a difference in your professional life and in the lives of people
affected by haemoglobin disorders!
BOARD MEMBERS Corner
About Thalassaemia International Federation
ΤIF The Thalassaemia International Federation (TIF) is a non-profit, non-govermental
organization, founded in 1987 by small group of patients and parents representing mainly
National Thalassaemia Associations in Cyprus, Greece, UK, USA and Italy - countries where
Thalassaemia was first recognized as an important public health issue and where the first
programmes for its control, including prevention and clinical management had started to be
promoted and implemented.
MISSION “Promotion of control programmes and access to quality treatment for every patient with
Thalassaemia where ever he or she may live”
OBJECTIVES Τhe objectives of the Federation in addressing effectively the needs of the world Thalassaemia
family have since its establishment remained the same and include:
• The establishment of new and promotion of existing National Thalassaemia
Patients/Parents Associations
• Encouraging, motivatiing and supporting studies and research for furher improving
prevention strategies, clinical care and for achieving the long-awaited final cure
• Extending the knowledge and experiences gained from countries with successful control
programmes to those in need.
JOIN US, become a member of our world thalassaemia family!
Thalassaemia International Federation
P.O. Box 28807, Nicosia 2083, Cyprus
Tel.: +357 22 319 129
Fax: +357 22 314 552
Email: [email protected] • www.thalassaemia.org.cy
