title: Prophylaxis with anti-inhibitor coagulant complex improves health-related quality of life in haemophilia patients with inhibitors: results from FEIBA NF Prophylaxis Study.
abstract: The Pro-FEIBA study reported health-related quality of life (HRQoL) improved following 6-month of Factor Eight Inhibitor Bypassing Activity (FEIBA) prophylaxis. This study investigates whether 12-month of FEIBA prophylaxis improved HRQoL in haemophilia patients with inhibitors. Thirty-six subjects in a 1-year prospective, randomized, open-label, parallel-design study were randomized to prophylaxis (85 ± 15 U kg(-1) every other day) or on-demand treatment. HRQoL was assessed at screening, 6 and 12-month termination using the EQ-5D, Haem-A-QoL, Haemo-QoL and a general pain visual analog scale (VAS). To evaluate changes, paired t-tests and criteria for minimally important differences were applied. Repeated measures regression tested the association between annualized bleeding rate (ABR) and physical HRQoL. At 6 and 12 months, prophylaxis subjects reported clinically meaningful improvement in EQ-5D index (mean improvement, 0.10 and 0.08, respectively) and both clinically meaningful and statistically significant improvements in EQ-VAS scores (16.9 and 15.7, respectively; P < 0.05) vs. baseline. General pain was significantly reduced during prophylaxis at each follow-up (mean improvement, 20.3 and 23.2, respectively; both P <0.05). At 12 months, prophylaxis subjects achieved significant improvements in Haem-A-QoL Total Score and in four domains: Physical Health, Feeling, View, and Work and School (all P < 0.05). No statistically significant changes, except for Haem-A-QoL Physical Health at 6 months, were observed with on-demand treatment. ABR was decreased by 72.5% with prophylaxis vs. on-demand treatment (P = 0.0003) and reduced ABR was associated with better physical HRQoL (P < 0.05). FEIBA prophylaxis significantly reduced ABR and improved HRQoL in inhibitor patients. Subjects with lower ABR reported better physical HRQoL.
authors.affiliation: Institute of Blood Pathology and Transfusion Medicine, Ukrainian National Academy of Medical Sciences, Lviv, Ukraine.
authors.forename: O
authors.initials: O
authors.lastname: Stasyshyn
authors.forename: S
authors.initials: S
authors.lastname: Antunes
authors.forename: V
authors.initials: V
authors.lastname: Mamonov
authors.forename: X
authors.initials: X
authors.lastname: Ye
authors.forename: J
authors.initials: J
authors.lastname: Epstein
authors.forename: Y
authors.initials: Y
authors.lastname: Xiong
authors.forename: S
authors.initials: S
authors.lastname: Tangada
mesh_terms: D000293:Adolescent; D000328:Adult; D001779:Blood Coagulation Factors / Q000627:therapeutic use*; D002648:Child; D003029:Coagulants / Q000008:administration & dosage*; D005169:Factor VIII / Q000008:administration & dosage*; D006304:Health Status; D006467:Hemophilia A / Q000188:drug therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D010147:Pain Measurement; D011446:Prospective Studies; D011788:Quality of Life; D012044:Regression Analysis; D011795:Surveys and Questionnaires; D055815:Young Adult
chemical_list: D001779:Blood Coagulation Factors; D003029:Coagulants; D005169:Factor VIII; C065655:anti-inhibitor coagulant complex
keywords: anti-inhibitor coagulant complex; factor eight inhibitor bypassing activity; haemophilia; health-related quality of life; prophylaxis