Haemophilia care - beyond the treatment guidelines

Abstract

Care for people with haemophilia (PWH) has improved much over the last two decades leading to near normal lives for those receiving early regular prophylaxis with clotting factor concentrates (CFC). Yet, there are significant limitations of those practices. In the absence of a well-defined optimal prophylaxis protocol, there are wide variations in practices with a two to threefold difference in doses. In those parts of the world where there are constraints on the availability of CFC, episodic replacement remains the norm for most patients even though it is evident that this does not change the natural history of the disease over a wide range of doses. Suitable prophylactic protocols therefore need to be developed wherever possible at these doses. Finally, there are only limited data on long-term outcomes in haemophilia from anywhere in the world. The practice of documenting specific outcomes as part of the regular evaluation of PWH needs to be established and the appropriate instruments used to assess them. Definitions of clinical events and endpoints of interventions in clinical studies are being developed to help such data collection. The correlations between different replacement therapy protocols and specific outcomes will help define what is best at different dose levels. Such data will allow better health planning and treatment choices throughout the world.

Keywords: assessment; doses; haemophilia; optimal; outcomes; prophylaxis.