Distribution and treatment of haemophiliacs

Speaker: Dr PAUL GIANGRANDE

Consultant Haematologist and Director, Oxford Haemophilia Centre

Dr Giangrande set the scene for the conference bydescribing the distribution and treatment of patientswith haemophilia.

Haemophilia is present in 1 in 16 000 people. It isa very serious condition in the absence of treatment.For this reason the World Federation of Hemophilia(WFH) seeks to stimulate services and increase accessto care world-wide for people with haemophilia andrelated disorders. The success of such an approach isillustrated by the fact that in 1985 (i.e. before theimpact of HIV infection and AIDS) the life expec-tancy of adults with haemophilia was 69.5 years. It isa condition that will not go away. Rather, numberswill increase.

In 1997 there was a total of 6480 haemophiliapatients in the UK. Of these, 5326 had haemophiliaA, with 1789 (34%) severe and 268 (5%) withinhibitors. There were 1154 haemophilia B cases,with 355 (31%) severe and 11 (1%) with inhibitors.For haemophilia A, most of the severe cases are aged20±50 years (Fig. 1). Consumption of coagulationfactor concentrate has risen markedly in recent years(Fig. 2).

AIDS remains the principal cause of death forhaemophiliacs. For example, in the UK in 1994, 73haemophiliacs died from AIDS. Cancer was thesecond most common cause of death (Table 1).Many deaths are iatrogenic, which has led to thedemand for total safety in treatment. This, however,is impossible to achieve.

The UK Centre Directors see recombinant factorVIII (rVIII) as the treatment of choice:

`Recombinant factor VIII is the treatment of choicefor all patients. If the introduction of recombinantfactor VIII has to be prioritized then those who maybene®t most should receive it ®rst. Priority shouldtherefore be given to those who have been leastexposed to blood products in the past. These willmost commonly be children.' [1]

However, as adults die, the funds allocated to theirtreatment could be transferred to children. The

bene®ts of the prophylactic use of rVIII in childrencan be seen from Fig. 3. (More detailed data on thiswere presented later by Dr Jan Astermark.)

In addition to the cost of prophylactic treatment,there is also the problem of inhibitors. For example,for a child weighing 20 kg, immune tolerance treat-ment of 100 units kg)1 day)1 would be needed for6 months. At 30p per unit, the total cost would be£120 000.

Treatment is based in 85 haemophilia centres;however, there are only 22 comprehensive carecentres, working to the guidance in HSG(93)30 [2].This sets out 15 functions of a comprehensive carecentre, including a 24-h clinical service and labora-tory facilities for diagnosing and monitoring treat-ment; access to specialist consultancy for surgery

Fig. 1. Age range of 1882 patients with severe haemophilia A in

the UK (United Kingdom Haemophilia Centre Directors' Organi-

sation, 1995).

Table 1. Principal causes of death amongst UK haemophiliacs:

1994 (United Kingdom Haemophilia Centre Directors' Organisa-

tion, 1995)

Cause Number of deaths

AIDS 73

Cancer (including 4 liver cancers) 15

Liver disease 14

Intracerebral haemorrhage 9

Bleeding from other sites 4

Infections 8

Ischaemic heart disease 3

Correspondence: Dr Paul Giangrande, Oxford HaemophiliaCentre, Churchill Hospital, Oxford OX3 7LJ, UK.

Haemophilia (1998), 4, (Suppl. 1), 1±2

Ó 1998 Blackwell Science Ltd 1

such as orthopaedic, infectious diseases (e.g. HIV)and dental surgery.

Other haemophilia centres provide the ®rst nine ofthe 15 functions detailed in HSG(93)30. Taking allhaemophilia centres, 42 treat fewer than 10 severelyaffected patients and only one centre treats morethan 100 severely affected patients (Fig. 4).

The ultimate hope is to be able to treat haemo-philia by gene therapy, so ending the need for rVIIItreatment. Various approaches are being explored,but there are risks of complications including ex-tended cellular infection and malignancy. It is tooearly to predict the outcome of this work [3].

References

1 United Kingdom Haemophilia Centre Directors' Orga-nisation Executive Committee. Guidelines on therapeu-tic products to treat haemophilia and other hereditarycoagulation disorders. Haemophilia 1997; 3: 63±77.

2 NHS Executive. HSG(93)30. Provision of HaemophiliaTreatment and Care. 1993.

3 Pasi KJ. Gene therapy for haemophilia. BaillieÁre's ClinHaematol Int Practice Research 1996; 2: 305±18.

4 Nilsson IM, et al. Twenty-®ve years' experience ofprophylactic treatment in severe haemophilia A and B. JInt Med 1992; 232: 25±32.

5 Miners AH, et al. Financing the rising cost of haemo-philia care at a large comprehensive care centre. J R CollPhysicians Lond 1997; 31: 640±4.

Fig. 2. Factor VIII units used by

UK Haemophilia Centre Directors

in 1981±1994 to treat haemophilia

patients.

Fig. 3. Effect of the prophylactic use of rVIII on orthopaedic joint

score [4].

Fig. 4. Distribution of severely affected patients amongst 85

haemophilia centres [5].

2 P. GIANGRANDE

Haemophilia (1998), 4, (Suppl. 1), 1±2 Ó 1998 Blackwell Science Ltd