Prophylaxis for severe XXXX : clinical and economical issues.

@entity1 @entity2720 @entity548 @entity447
@entity1 with severe @entity447 are treated either in case of @entity548 only (on demand), or with regular infusions of clotting factor to prevent @entity548 (prophylaxis). The introduction of prophylaxis has been hampered by issues of cost and viral safety. In order to compare results and treatment cost of different treatment strategies in adults, three cohorts of @entity1 with severe @entity447 (born 1970-1980) were compared. 106 French @entity1 were treated on demand, 49 Dutch @entity1 were treated with intermediate dose prophylaxis, and 24 Swedish @entity1 were treated with high dose prophylaxis. The annual number of joint @entity548 , and the radiological Pettersson score were used to compare outcome, annual clotting factor consumption was used to compare costs. Prophylaxis reduced @entity548 and @entity2720 : @entity1 treated on demand had a median of 11.5 joint @entity548 /year and a median Pettersson score of 16 points, for intermediate dose prophylaxis median @entity548 were 2.8 and Pettersson score was 7 points, and for high dose prophylaxis median @entity548 were 0.5 joint @entity548 and Pettersson score was 4 points. All differences were statistically significant, except the Pettersson scores in both prophylactic regimens. Treatment cost was only increased for high dose prophylaxis: mean clotting factor consumption was 1612 IU kg-1 yr-1 for on demand treatment, 1488 IU kg-1 yr-1 for intermediate dose prophylaxis, and 4012 IU kg-1 yr-1 for high dose prophylaxis. In young adults, the cost of intermediate dose prophylaxis is similar, but outcome is better than for on demand treatment. The cost of high dose prophylaxis is twofold higher, further improving outcome only slightly.
