The treatment of haemophilia, including prophylaxis, constant infusion and DDAVP

Recent years have witnessed advances in the treatment of haemophilia such as the introduction of prophylaxis, continuous infusion and phamacological treatment with desmopressin (DDAVP). Prophylactic treatment on a long-term basis appears to be effective in preventing the development of arthropathy in severe haemophilia. The largest body of experience is that from Sweden, where prophylaxis is started at the age of 1-2 years. The dosage used is 25-40 U factor VIII/IX per kilogram bodyweight given three times or twice weekly, respectively. In some cases an intravenous access device has to be used during the first years of treatment. The patients grow up like normal boys and can live virtually normal lives. The beneficial psychological impact of prophylaxis on the family cannot be overestimated. Side-effects are not more frequent with prophylaxis than with on-demand treatment. The feasibility of continuous infusion of factor VIII/IX concentrates during bleeding episodes, or as cover for surgery, has been documented. This mode of delivery increases convenience and the cost-benefit ratio of the treatment, with savings in postoperative replacement of factor concentrate of about 50-75%. Many modern concentrates are stable enough for the purpose, and several pump systems, including portable ones, are available. The haemostatic drug DDAVP can be effectively used in most cases of mild haemophilia A. Intravenous administration is to be preferred as cover for surgery or in the case of severe bleeds. There is an effective nasal spray which can also be used for home therapy in mild or moderate bleedings.