THE IMPACT OF PROPHYLACTIC TREATMENT ON CHILDREN WITH SEVERE HEMOPHILIA

Twenty-seven children with severe haemophilia receiving regular prophy lactic factor concentrate were evaluated to examine the overall effect iveness of prophylaxis in modern haemophilia care. The median age at t he start of prophylaxis was 6.2 years (range 13-15.9 years) and the cu mulative length of follow-up was 808 months (mean 30, range 7-76 month s). Nine patients required a central venous catheter for venous access (age range 1.3-5.2 years), eight boys could cannulate themselves and in 10 the parents performed regular venepuncture. The mean dose of con centrate given at the time of study was 31.8 U/kg three times weekly ( range 12.5-52.6U/kg) or 4900 U/kg/year (range 1900-8200). None develop ed an inhibitor on prophylaxis, though four had previously had an anti body. The median average annual number of bleeds in the 27 patients pr ior to prophylaxis was 14.7 (range 3.7-35.4). On prophylaxis this fell to 1.5 (range 0-12.5) (P < 0.001) and in the group as a whole the fre quency of bleeds diminished in successive years on prophylaxis. All 20 children with evidence of arthropathy improved on prophylaxis and eig ht had reversal of chronic damage such that their joints appeared norm al at the time of study. There were reductions in the need for walking aids, in hospital admissions, and in numbers of school days lost for bleeding episodes. All families feel that prophylaxis has brought abou t an improvement in quality of life.