Therapy for haemophilia: recent advances and goals for the future

In the 20th century, haemophilia evolved from a life-threatening, crippling disease to one for which the prognosis is excellent and many patients lead normal, productive lives. Although dramatic achievements in the treatment of haemophilia have occurred, the current therapies have significant drawbacks. Among these is the relatively high incidence of inhibitor development, the requirement for frequent intravenous infusions to prevent bleeding complications, the lack of effective treatment for established joint disease, and the high cost of treatment. The future goal of haemophilia treatment first and foremost is curing this genetic condition via gene therapy. As this goal is likely many years away, improvements in the current factor products in order to reduce the development of inhibitors and to reduce the frequency of therapy are more immediately achievable goals. Finally, improving the treatment of bleeding complications, particularly in inhibitor patients, and developing novel adjunctive therapies for the management of joint disease are also important goals for the near future. This review will discuss in detail the cu-rrent and future goals of haemophilia therapy.