Long-term outcome of individualized prophylactic treatment of children with severe haemophilia.
The development of arthropathy is a serious complication of severe haemophilia. With the use of prophylaxis, bleeds can be prevented and arthropathy delayed. We investigated whether an individually tailored prophylactic regimen can prevent arthropathy and whether it had a similar effect on orthopaedic outcome compared with that of a high-dose regimen. Efficacy was determined clinically and by radiographs of six major joints. Prophylaxis was started 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 score 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 arthropathy.
Van Creveld Clinic, University Medical Centre Utrecht, Heidelberglaan 100, 3584 CX Utrecht, The Netherlands. h.m.vandeberg@digd.azu.nl
H M
HM
van den Berg
K
K
Fischer
E P
EP
Mauser-Bunschoten
F J
FJ
Beek
G
G
Roosendaal
J G
JG
van der Bom
H K
HK
Nieuwenhuis
D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D004334:Drug Administration Schedule; D005169:Factor VIII / Q000008:administration & dosage*; D005500:Follow-Up Studies; D006467:Hemophilia A / Q000150:complications* / Q000188:drug therapy; D002836:Hemophilia B / Q000150:complications / Q000188:drug therapy; D006470:Hemorrhage / Q000209:etiology / Q000517:prevention & control*; D006801:Humans; D007592:Joint Diseases / Q000209:etiology* / Q000517:prevention & control*; D013997:Time Factors; D016896:Treatment Outcome
D005169:Factor VIII