Modern haemophilia care

Haemophilia care has undergone substantial improvements during the past 40-50 years. Early clotting factor concentrates were not sufficiently refined to enable self-administered treatment at home until the 1970s. Unfortunately, these advances led to transmission of viral diseases including HIV and hepatitis, resulting in an increased burden of morbidity and mortality, especially during the 1980s. Throughout the past two decades, product development, including the advent of recombinant concentrates, has greatly improved the safety and availability of therapy and the focus of care is shifting towards prevention and management of disease sequelae. Long-term substitution therapy (prophylaxis) of the missing clotting factor is the recommended treatment in severe haemophilia, but several research issues remain to be elucidated such as when to start and how to optimise these regimens, and when or whether to stop this expensive treatment. The major side-effect of treatment, development of inhibitors to the infused concentrate, is the main threat to the health of patients and consequently the goal of intense research. Development of new products with improved pharmacokinetics is the next step to improved therapy.