Factor concentrates for haemophilia in the developing world

Abstract

Summary. The success in the management of haemophilia in the last two decades has been predominantly due to the availability of sufficient quantities of safe factor concentrates. Unfortunately, the prohibitive cost of these products has prevented this benefit from being available to the vast majority (∼80%) of haemophiliacs living in the developing world. A few developing countries have established facilities for the production of low- to intermediate-purity factor concentrates locally. The infrastructure required to achieve this can be very basic. The experience in South Africa, Thailand, Cuba and Brazil, described herein, shows that this approach provides factor concentrates which are very economical in comparison with more purified commercial products. This has had a major impact on the quality of haemophilia care in these countries. Wide availability of low-cost factor concentrates has made these products accessible to a large number of haemophiliacs and even made home therapy possible. The effort to provide these products results in improvement of the blood transfusion services. This, in turn, contributes to better facilities for patients with other transfusion-dependent diseases and society in general. Installation of small plasma fractionation plants is also a viable option. This not only allows processing of large pools of plasma for greater quantities of factor concentrates but also provides albumin and immunoglobulin. The revenue generated from the sale of the other products has been used to improve and subsidize haemophilia care. It is concluded that local production of intermediate purity factor concentrates in developing countries is absolutely necessary. A well organized transfusion service is required to collect adequate quantities of plasma for fractionation.