Factor replacement therapy in haemophilia – are there models for developing countries?

Abstract

Successful models for factor replacement in severe haemophilia involves prophylactic or on-demand administration of large quantities (1500-9000 IU kg-1 year-1) of very high purity factor concentrates starting early in life. The prohibitive cost of these protocols make them completely impractical in developing countries where the quantity of factor used for replacement therapy is much lower and varies considerably (25-500 IU kg-1 year-1). At this level of treatment, as some joint damage is inevitable, the aim of therapy shifts to preventing disability and preserving reasonable joint function rather than perfect architecture. There are no carefully recorded data on long-term outcome of musculo-skeletal function on these doses. Appropriate studies are needed to document such data. With regard to products for factor replacement, economic compulsions lead to the use a variety of factor concentrates and blood-bank products for replacement therapy. Most patients in the developing world do not have access to adequate replacement therapy. Among the rest, some get limited quantities of plasma-derived concentrates while others use cryoprecipitate, fresh frozen plasma or even whole blood. Since the superiority of virus-inactivated purified factor concentrates in achieving the aims of replacement therapy is well established, the aim should be to provide this for all people with haemophilia. This can be achieved by production of such concentrates locally or by importing them. Different models are possible depending on the circumstances in each country.

Hemophilia care in China: review of care for 417 hemophilia patients from 11 treatment centers in Shanxi Province

“…According to WHO, the average annual cost of prophylaxis for PWH is ‡US$100,000 per individual, with prophylactic consumption of pdFVIII being 1900-8200 IU/kg/year in developed countries [15,16]. In China, even short-term low-dose tertiary prophylaxis per hemophilic child is US$46,848 [17], and represents a huge financial strain on households [10].…”

Association Between Physical Activity and Risk of Bleeding in Children With Hemophilia

“…4 The clinical ef-fects of lower doses have not been rigorously tested. 5 The reduction in risk of bleeds conferred by prophylaxis may be such that the benefits of physical activity outweigh the risk of bleeds. However, the degree to which risk of bleeds is elevated by physical activity has not been determined.…”

Comprehensive Care and Delivery of Care in Hemophilia: The Developing World

“…Therefore selecting suitable protocols for use in each country becomes critical [13]. In situations without significant constraints on resources, the guiding principle is to use high doses that guarantee efficacy, albeit at an extremely high cost.…”