Long-term outcome of individualized prophylactic treatment of children with severe haemophilia

The development of arthropathy is a serious complication of severe haemophi lia. With the use of prophylaxis, bleeds can be prevented and arthropathy d elayed. We investigated whether an individually tailored prophylactic regim en can prevent arthropathy and whether it had a similar effect on orthopaed ic outcome compared with that of a high-dose regimen, Efficacy was determin ed clinically and by radiographs of six major joints. Prophylaxis was start ed in 70 patients at a mean age of 4.1 years. Mean follow-up was 15.6 years (range 8-24.5 years). The mean factor VIII consumption was 2319 IU/kg/year . The mean number of joint bleeds was 3.5/year and the mean clinical score (maximum score 90) was 1.0, with a mean Pettersson joint score (maximum sco re 78) of 3.0 at a mean age of 13.5 years. In conclusion, long-term, early- onset, individualized prophylaxis in haemophilia is feasible and prevents a rthropathy.