title: The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia.
abstract: To prevent hemophilic arthropathy, prophylactic treatment of children with severe hemophilia should be started before joint damage has occurred. However, treatment is expensive, and the burden of regular venipunctures in young children is high. With the aim of providing information on starting prophylaxis on the basis of individual patient characteristics, the effect of postponing prophylaxis on long-term arthropathy was studied in a cohort of 76 patients with severe hemophilia born between 1965 and 1985. The median age at first joint bleed was 2.2 years (range, 0.2-5.8). Prophylaxis was started at a median age of 6 years (interquartile range [IQR], 4-9), and the median annual clotting factor use on prophylaxis was 1750 IU/kg/y (31 IU/kg/wk). Hemophilic arthropathy was measured by the Pettersson score (maximum, 78 points). At a median age of 19 years, the median Pettersson score was 7 points (IQR, 0-17). After 2 decades of follow-up, the Pettersson score was 8% higher (95% confidence interval, 1%-16%) for every year prophylaxis was postponed after the first joint bleed. This effect was independent of age at Pettersson score, age at first joint bleed, and prophylactic dose used. In conclusion, most patients have their first joint bleed after the age of 2 years. Patients who start prophylaxis soon after the first joint bleed show little arthropathy in adulthood. The longer the start of prophylaxis is postponed after the first joint bleed, the higher the risk of developing arthropathy.
authors.affiliation: Van Creveldkliniek University Medical Center Utrecht, PO Box 85500, 3808 GA Utrecht, The Netherlands. kfischer@azu.nl
authors.forename: Kathelijn
authors.initials: K
authors.lastname: Fischer
authors.forename: Johanna G
authors.initials: JG
authors.lastname: van der Bom
authors.forename: Eveline P
authors.initials: EP
authors.lastname: Mauser-Bunschoten
authors.forename: Goris
authors.initials: G
authors.lastname: Roosendaal
authors.forename: Robert
authors.initials: R
authors.lastname: Prejs
authors.forename: Piet
authors.initials: P
authors.lastname: de Kleijn
authors.forename: Diederick E
authors.initials: DE
authors.lastname: Grobbee
authors.forename: Marijke
authors.initials: M
authors.lastname: van den Berg
mesh_terms: D017677:Age Distribution; D017668:Age of Onset; D002648:Child; D002675:Child, Preschool; D010450:Endopeptidases / Q000627:therapeutic use*; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy*; D006470:Hemorrhage / Q000517:prevention & control; D006801:Humans; D007223:Infant; D007596:Joints / Q000098:blood supply; D012189:Retrospective Studies; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D010450:Endopeptidases; C019914:clotting enzyme