Academic journal article Bulletin of the World Health Organization

Modern Treatment of Haemophilia

Academic journal article Bulletin of the World Health Organization

Modern Treatment of Haemophilia

Article excerpt

Primary prophylaxis in haemophilia


Although many persons with haemophilia who received blood products over the period 1979-84 have died of acquired immunodeficiency syndrome (AIDS), many are still alive but have human immunodeficiency virus (HIV) infection and declining cellular immunity, with or without AIDS-defining illness. Many of these individuals are also positive for hepatitis C virus (HCV) antibodies. There is no cure for either of these bloodbome viral illinesses; finding more effective modes of treatment therefore remains a necessity.

Fortunately, with only a few exceptions, there have been no reports of new cases of HIV infection resulting from transfusion of blood or clotting factor concentrates for several years. Thus more attention can be focused on other problems and questions caused by haemophilia, e.g., the advantages and disadvantages of prophylaxis in the prevention of joint disease, the choice of an expanding array of products, what can be done to prevent or eradicate inhibitors, prospects for a cure via gene therapy, and how persons with haemophilia who live in developing countries can best be managed with the available resources.

Rationale and experience to date

What is the role of primary prophylaxis in haemophilia care? In Sweden, the prevention of joint and musculoskeletal disease in severe haemophiliacs has been accomplished by starting prophylaxis early in life (age, 1-2 years), with dosages and dosage intervals aimed at keeping factor (F) VIII or F IX trough levels >1%. The rationale behind this approach is that persons with trough levels >1% F VIII or F IX have far fewer bleeding episodes and a much milder clinical course than those with severe haemophilia (trough levels < 1% F VIII or F IX).

While several groups of investigators have reported small series of patients utilizing various prophylactic regimens, the most comprehensive study to date was carried out in Malmo, Sweden; Nilson et al. have published 25-years' experience with prophylaxis involving 60 patients with severe haemophilia A and B(1). Over the 25-year study period the prophylaxis has been intensified by starting it at an earlier age and by increasing the dosage of F VIII or F IX. As a result, the most recent subset of 15 boys (now aged 4-12 years), who began prophylaxis at 1-2 years of age, have experienced no episodes of joint bleeding and have excellent orthopaedic and radiological joint scores (all zero)(1).

The situation in the USA and Canada

After an in-depth review of the Swedish experience with primary prophylaxis, in early 1994 the National Hemophilia Foundation's Medical and Scientific Advisory Council (MASAC) recommended that in the USA primary prophylaxis be considered optimal therapy for persons with severe haemophilia (F VIII or F IX levels <1%), beginning at an early age(1-2) years). MASAC also recommended that a mechanism be developed to evaluate periodically joint status, to document any complications, and to record all costs associated with each child's prophylaxis. A further recommendation was that the risks versus the benefits and all possible problems associated with prophylaxis be discussed with each family, including venous access problems and complications that could result from indwelling central venous catheters(2).

Also, based mainly on the excellent results in Sweden, the Canadian Hemophilia Society recommended in May 1993 that prophylactic treatment be offered to all young children with severe haemophilia.

Cost versus benefits

Although there has been concern that primary prophylaxis may be considerably more costly than episodic ("on demand") treatment, this is not necessarily the case. For example, Carlsson et al. investigated the use of individual pharmacokinetics as an aid to determining the optimal dosage of F VIII for each patient, with emphasis on the impact of different dosage regimens on cost. …

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