title: Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome.
abstract: A multicentre study was performed to compare clotting factor use and outcome between on-demand and prophylactic treatment strategies for patients with severe haemophilia. Data on treatment and outcome of 49 Dutch patients with severe haemophilia, born 1970-80, primarily treated with prophylaxis, were compared with those of 106 French patients, who were primarily treated on demand. Dutch patients received intermediate dose prophylaxis, for a median duration of 12.7 years. Patients primarily treated with prophylaxis had fewer joint bleeds per year (median 2.8 vs. 11.5), a higher proportion of patients without joint bleeds (29% vs. 9%), lower clinical scores (median 2.0 vs. 8.0), and less arthropathy as measured by the Pettersson score (median 7 points vs. 16 points). Mean annual clotting factor use was equal at 1,488 +/- 783 IU kg-1 year-1 (mean +/- standard deviation) for patients primarily treated with prophylaxis and 1,612 +/- 1,442 IU kg-1 year-1 for patients primarily treated on demand. These findings suggest that, compared with a primarily on-demand treatment strategy, a primarily prophylactic treatment strategy leads to better outcome at equal treatment costs in young adults with severe haemophilia.
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 G
authors.initials: JG
authors.lastname: van der Bom
authors.forename: P
authors.initials: P
authors.lastname: Molho
authors.forename: C
authors.initials: C
authors.lastname: Negrier
authors.forename: E P
authors.initials: EP
authors.lastname: Mauser-Bunschoten
authors.forename: G
authors.initials: G
authors.lastname: Roosendaal
authors.forename: P
authors.initials: P
authors.lastname: De Kleijn
authors.forename: D E
authors.initials: DE
authors.lastname: Grobbee
authors.forename: H M
authors.initials: HM
authors.lastname: van den Berg
mesh_terms: D000328:Adult; D004334:Drug Administration Schedule; D005164:Factor IX / Q000008:administration & dosage* / Q000627:therapeutic use; D005169:Factor VIII / Q000008:administration & dosage* / Q000627:therapeutic use; 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; D012189:Retrospective Studies; D016896:Treatment Outcome
chemical_list: D005169:Factor VIII; D005164:Factor IX