title: Endogenous clotting factor activity and long-term outcome in patients with moderate haemophilia.
abstract: In order to address the question of the optimum target level for prophylactic treatment of severe haemophilia patients, the association between endogenous clotting factor activity and outcome was studied in a cohort of 46 patients with moderate haemophilia. Data on treatment and outcome were collected annually. Median follow-up was 8.0 years (range 1-26). Median joint bleed frequency was 1 per year and median radiological score according to Pettersson was 1 point (max. 78) at the age of 25 years. One percent increase in clotting factor level was associated with a 4 months later onset of joint bleeds (95% Confidence Interval (CI): 2-6 months) and a 16% lower Pettersson score (95% CI: 3-27%). No statistically significant effect of clotting factor activity on joint bleed frequency could be demonstrated. These findings confirm that patients with moderate haemophilia experience only mild arthropathy, and provide evidence for a protective effect of higher clotting factor levels on joint damage.
authors.affiliation: Van Creveldkliniek, Julius Center for General Practice and Patient Oriented Research, University Medical Center Utrecht, The Netherlands. K.Fischer@digd.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: E P
authors.initials: EP
authors.lastname: Mauser-Bunschoten
authors.forename: G
authors.initials: G
authors.lastname: Roosendaal
authors.forename: F J
authors.initials: FJ
authors.lastname: Beek
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: D000293:Adolescent; D000328:Adult; D001779:Blood Coagulation Factors / Q000378:metabolism*; D002648:Child; D015331:Cohort Studies; D005164:Factor IX / Q000378:metabolism; D005169:Factor VIII / Q000378:metabolism; D005500:Follow-Up Studies; D006395:Hemarthrosis / Q000097:blood / Q000209:etiology; D006467:Hemophilia A / Q000097:blood* / Q000150:complications / Q000175:diagnosis; D002836:Hemophilia B / Q000097:blood / Q000150:complications / Q000175:diagnosis; D006801:Humans; D011379:Prognosis; D012044:Regression Analysis; D016896:Treatment Outcome
chemical_list: D001779:Blood Coagulation Factors; D005169:Factor VIII; D005164:Factor IX