The PNH RegistryYour opportunity to enhance global understanding of PNH

The expected survival rate of an age- and gender-matched control group is shown for comparison.

Survival of patients with PNH compared to controls4

Pat

ient

s su

rviv

ing

(%)

Patients with PNH

Age- and gender-matched controls

100

80

60

40

20

00 5 10 15 20 25

Years after diagnosis

35%

of PNH patients die within 5 years

of diagnosis4

What is PNH?• Paroxysmal nocturnal hemoglobinuria (PNH) is a progressive,

life-threatening disease of chronic hemolysis that causes thrombosis, end organ damage, and impaired quality of life1,2

• In this acquired hematopoietic stem cell disorder, clonal expansion of hematopoietic stem cells with complete or marked loss of the terminal complement inhibitors CD55 and CD59 renders blood cells susceptible to chronic complement-mediated hemolysis1,3

• In a patient population in which half the patients have <30% clone, 1 in 7 patients died within 5 years5

• PNH may be diagnosed at any age, with median age in the early 30s6; diagnosis is typically delayed from 1 to more than 10 years4

• The following patient populations warrant testing as they are at higher risk for PNH7,8:

What is the PNH Registry? The PNH Registry is an international, observational, noninterventional study collecting data on patients with PNH, regardless of clone size or treatment approach.

The PNH Registry:

• Provides an invaluable opportunity to increase understanding of the natural history of PNH and the safety and efficacy of treatments

• Offers the international community greater insight into an uncommon disease with potentially devastating consequences1

“The PNH Registry is a robust, effective, and critical tool to collect data to support the further understanding of the pathophysiology, clinical manifestations, and long-term outcomes of patients living with PNH.” — Robert A. Brodsky, MD

Director, Division of Hematology Professor of Medicine and Oncology Johns Hopkins University

How is the PNH Registry governed?• The PNH Registry is overseen by an independent,

collaborative executive committee of physicians who are highly experienced in managing PNH patients and who provide scientific advice and advocacy

• Individual physicians are key to data integrity and success of the PNH Registry

• All patient data are de-identified for confidentiality

– Coombs-negative hemolytic anemia

– Hemoglobinuria – Aplastic anemia

– Refractory anemia-myelodysplastic syndromes

– Unexplained cytopenias – Unexplained thrombosis

(venous or arterial)

What are the objectives of the PNH Registry?• Collect and publish data to characterize the progression of

PNH, clinical and patient-reported outcomes, and morbidities and mortality of patients with PNH

• Expand knowledge of PNH natural history and demographics

• Analyze and assess the safety and efficacy of approaches to PNH management

• Raise PNH awareness in the medical community and patient population

• Establish a robust, international database collected in a real-world setting

How extensive is the PNH Registry?The PNH Registry includes more than 1650* patients worldwide.

Who can contribute?• All physicians managing patients with PNH, regardless of

treatment approach

Who is eligible to be enrolled?• All patients who have been newly or previously diagnosed

with PNH or have evidence of positive PNH cells

How are data collected? The PNH Registry adheres to a uniform process of data collection.

• Data are easily entered by means of a secure Web portal at enrollment and approximately every 6 months thereafter

• Data can be collected during routine visits or entered from patient medical records

• Data entry includes: demographics, medical history, PNH diagnosis, flow cytometry results, symptomatology, safety events of interest, PNH progression, clinical outcomes, quality of life, and pregnancy

• The PNH Registry data are analyzed by a collaborative global scientific board chaired by Peter Hillmen, MD, FRCP, FRCPath, PhD, in England, and are managed by an independent group with expertise in observational research

• United States• Canada• Argentina• Denmark• The Netherlands • UK• Japan• South Korea

• Belgium • France • Germany • Spain • Switzerland • Luxembourg • Russia • Mexico

• Finland• Sweden• Austria• Norway• Australia• New Zealand • Taiwan

Countries include*:

*As of August 2012.

Data generated by the PNH Registry advance understanding of the disease

Contact your local representative of the PNH Registry to learn more

Phone: 1.800.913.4893

E-mail: pnhregistry@iconplc.com

ASH 2011: “Clinical Characteristics of Classic Paroxysmal Nocturnal Hemoglobinuria (PNH) in Pediatric Patients: A Comparison With Classic PNH in Adults. An International PNH Registry Study”9

—Urbano-Ispizua et al.

EHA 2011: “Pediatric Diagnosis of Paroxysmal Nocturnal Hemoglobinuria in the International PNH Registry”10

—Urbano-Ispizua et al.

ASH 2010: “Use of Blood Transfusions in Paroxysmal Nocturnal Hemoglobinuria Patients With and Without Aplastic Anemia Enrolled in the Global PNH Registry”11 —Schrezenmeier et al.

ASH 2010: “Evaluation of Paroxysmal Nocturnal Hemoglobinuria Disease Burden: The Patient’s Perspective. A Report From the Global PNH Registry”12 —Muus et al.

EHA 2010: “Evaluation of Paroxysmal Nocturnal Hemoglobinuria Disease Burden in Patients Enrolled in the International PNH Registry”13

—Urbano-Ispizua et al.

ASH 2009: “A Global Registry of Patients With Paroxysmal Nocturnal Hemoglobinuria”14 —Brodsky et al.

What are the key benefits of PNH Registry participation?• Enhance understanding of PNH

• Capture the long-term outcomes of patients in order to better guide and assess treatments

• Expand a robust, international database on PNH

• Contribute to scientific exchange and publications

• Help to define practice patterns

• Promote evidence-based medicine

• Share data and treatment experiences through participation in periodic Registry meetings

Enroll your patients in the PNH Registry todayUnite with a global community of physicians in contributing to the largest, most comprehensive PNH patient registry.

Contribute to treatment objectives, practice patterns, and best practices.

Support scientific collaboration in the PNH community.

To learn more, contact the PNH Registry at 1.800.913.4893 or pnhregistry@iconplc.com.

The PNH Registry is sponsored by Alexion Pharmaceuticals.

References: 1. Rother RP, Bell L, Hillmen P, Gladwin MT. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin: a novel mechanism of human disease. JAMA. 2005;293:1653-1662. 2. Brodsky RA. Paroxysmal nocturnal hemoglobinuria. In: Hoffman R, Benz EJ Jr, Shattil SJ, et al, eds. Hematology: Basic Principles and Practice. 4th ed. Philadelphia, PA: Elsevier Churchill Livingstone; 2005:419-427. 3. Brodsky RA. Advances in the diagnosis and therapy of paroxysmal nocturnal hemoglobinuria. Blood Rev. 2008;22:65-74. 4. Hillmen P, Lewis SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal nocturnal hemoglobinuria. N Engl J Med. 1995;333:1253-1258. 5. Peffault de Latour R, Mary J-Y, Salanoubat C, et al; on behalf of the French Society of Hematology and the French Association of Young Hematologists. Paroxysmal nocturnal hemoglobinuria: natural history of disease subcategories. Blood. 2008;112:3099-3106. 6. Socié G, Mary J-Y, de Gramont A, et al; for the French Society of Haematology. Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. Lancet. 1996;348:573-577. 7. Borowitz MJ, Craig FE, DiGiuseppe JA, et al; for Clinical Cytometry Society. Guidelines for the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria and related disorders by flow cytometry. Cytometry Part B. 2010;78:211-230. 8. Parker C, Omine M, Richards S, et al; for International PNH Interest Group. Diagnosis and management of paroxysmal nocturnal Hemoglobinuria. Blood. 2005;106:3699-3709. 9. Urbano-Ispizua A, Schrezenmeier H, Muus P, et al. Clinical characteristics of classic paroxysmal nocturnal hemoglobinuria (PNH) in pediatric patients: a comparison with classic PNH in adults. An International PNH Registry Study. In: Abstracts of the 53rd ASH Annual Meeting and Exposition; December 10-13, 2011; San Diego, CA. Abstract 2102. 10. Urbano-Ispizua A, Schrezenmeier H, Muus P, et al. Pediatric diagnosis of paroxysmal nocturnal hemoglobinuria in the International PNH Registry. In: Abstracts of the 16th Congress of the European Hematology Association; June 9-12, 2011; London, United Kingdom. Abstract 833. 11. Schrezenmeier H, Brodsky R, Muus P, et al. Use of blood transfusions in paroxysmal nocturnal hemoglobinuria patients with and without aplastic anemia enrolled in the Global PNH Registry. In: Abstracts of the 52nd ASH Annual Meeting and Exposition; December 4-7, 2010; Orlando, FL. Abstract 2241. 12. Muus P, Szer J, Schrezenmeier H, et al. Evaluation of paroxysmal nocturnal hemoglobinuria disease burden: The patient’s perspective. A report from the Global PNH Registry. In: Abstracts of the 52nd ASH Annual Meeting and Exposition; December 4-7, 2010; Orlando, FL. Abstract 1525. 13. Urbano-Ispizua A, Schrezenmeier H, Brodsky R, et al. Evaluation of paroxysmal nocturnal hemoglobinuria disease burden in patients enrolled in the International PNH Registry. In: Abstracts of the 15th Congress of the European Hematology Association; June 10-13, 2010; Barcelona, Spain. Abstract 1022. 14. Brodsky R, Schrezenmeier H, Muus P, et al. A global registry of patients with paroxysmal nocturnal hemoglobinuria. In: Abstracts of the 51st ASH Annual Meeting and Exposition; December 5-8, 2009; New Orleans, LA. Abstract 3007.

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