title: Secondary prophylaxis with recombinant activated factor VII improves health-related quality of life of haemophilia patients with inhibitors.
abstract: Haemophilia patients with inhibitors characteristically have impaired joint function and reduced health-related quality of life (HRQoL). This analysis examined whether secondary prophylaxis with recombinant activated factor VII (rFVIIa) improves HRQoL vs. conventional on-demand therapy in patients with haemophilia with inhibitors and frequent bleeds. After a 3-month preprophylaxis period, 22 patients received daily rFVIIa prophylaxis (90 or 270 microg kg(-1)) for 3 months, followed by 3 months' postprophylaxis. Days of hospitalization, absence from school/work and mobility aids requirements were recorded. HRQoL was assessed by EuroQoL (EQ-5D) questionnaire, visual analogue scale (VAS), derived Time to Trade-Off (TTO) scores and Quality Adjusted Life Years (QALYs). rFVIIa prophylaxis significantly (P < 0.0001) reduced bleeding frequency vs. prior on-demand therapy. Hospitalization (5.9% vs. 13.5%; P = 0.0026) and absenteeism from school/work (16.7% vs. 38.7%; P = 0.0127) decreased during prophylaxis; these effects tended to be maintained during postprophylaxis. HRQoL (evaluated by EQ-5D) tended to improve during and after rFVIIa prophylaxis. Notably, pain decreased and mobility increased in 40.9% and 27.3% of patients, respectively, at the end of the postprophylaxis period vs. preprophylaxis. Median VAS score increased from 66 to 73 (P = 0.048), and TTO scores suggested better HRQoL (0.62 vs. 0.76; P = 0.054) during postprophylaxis than preprophylaxis. Small to moderate changes in effect sizes were reported for VAS and TTO scores. Median QALYs were 0.68 (VAS) and 0.73 (TTO). Reductions in bleeding frequency with secondary rFVIIa prophylaxis were associated with improved HRQoL vs. on-demand therapy.
authors.affiliation: Department of Pediatrics and Internal Medicine, University of Texas Medical School, Houston, TX 77030, USA. keith.hoots@uth.tmc.edu
authors.forename: W K
authors.initials: WK
authors.lastname: Hoots
authors.forename: L S
authors.initials: LS
authors.lastname: Ebbesen
authors.forename: B A
authors.initials: BA
authors.lastname: Konkle
authors.forename: G K-H
authors.initials: GK
authors.lastname: Auerswald
authors.forename: H R
authors.initials: HR
authors.lastname: Roberts
authors.forename: J
authors.initials: J
authors.lastname: Weatherall
authors.forename: J-M
authors.initials: JM
authors.lastname: Ferran
authors.forename: R C R
authors.initials: RC
authors.lastname: Ljung
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D003029:Coagulants / Q000627:therapeutic use*; D015942:Factor VIIa / Q000627:therapeutic use*; D006395:Hemarthrosis / Q000517:prevention & control*; D006467:Hemophilia A / Q000188:drug therapy* / Q000503:physiopathology; D002836:Hemophilia B / Q000188:drug therapy* / Q000503:physiopathology; D006801:Humans; D017063:Outcome Assessment, Health Care / Q000706:statistics & numerical data*; D011446:Prospective Studies; D011788:Quality of Life; D019057:Quality-Adjusted Life Years; D011994:Recombinant Proteins / Q000627:therapeutic use
chemical_list: D003029:Coagulants; D011994:Recombinant Proteins; C103587:recombinant FVIIa; D015942:Factor VIIa