title: Prophylactic treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen.
abstract: A multicentre study was performed in Sweden and the Netherlands, comparing effects of two prophylactic regimens in 128 patients with severe haemophilia, born 1970-90. 42 Swedish patients (high-dose prophylaxis), were compared with 86 Dutch patients (intermediate-dose prophylaxis). Patients were evaluated at the date of their last radiological score according to Pettersson. Annual clotting factor consumption and bleeding frequency were registered for a period of three years before evaluation. Patients in the high-dose group were younger at evaluation (median 15.2 vs. 17.9 years), started prophylaxis earlier (median 2 vs. 5 years), and used 2.19 times more clotting factor kg-1 year-1. Patients treated with high-dose prophylaxis had fewer joint bleeds (median 0.3 year-1 vs. 3.3 year-1) and the proportion of patients without arthropathy as measured by the Pettersson score was higher (69% vs. 32%), however, the age-adjusted difference in scores (median 0 points vs. 4 points) was small and at present not statistically significant. Clinical scores and quality of life were similar. These findings suggest that, compared with intermediate-dose prophylaxis, high-dose prophylaxis significantly increases treatment costs and reduces joint bleeds over a period of 3 years, but only slightly reduces arthropathy after 17 years of follow-up.
authors.affiliation: Department of Paediatrics, University Medical Center Utrecht, The Netherlands. k.fischer@azu.nl
authors.forename: K
authors.initials: K
authors.lastname: Fischer
authors.forename: J
authors.initials: J
authors.lastname: Astermark
authors.forename: J G
authors.initials: JG
authors.lastname: van der Bom
authors.forename: R
authors.initials: R
authors.lastname: Ljung
authors.forename: E
authors.initials: E
authors.lastname: Berntorp
authors.forename: D E
authors.initials: DE
authors.lastname: Grobbee
authors.forename: H M
authors.initials: HM
authors.lastname: van den Berg
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D004305:Dose-Response Relationship, Drug; D005164:Factor IX / Q000008:administration & dosage* / Q000627:therapeutic use; D005169:Factor VIII / Q000008:administration & dosage* / Q000627:therapeutic use; D005500:Follow-Up Studies; D006395:Hemarthrosis / Q000209:etiology / Q000517:prevention & control; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy*; D002836:Hemophilia B / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D008297:Male; D011788:Quality of Life; D016896:Treatment Outcome
chemical_list: D005169:Factor VIII; D005164:Factor IX