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: O Stasyshyn, S Antunes, V Mamonov, X Ye, J Epstein, Y Xiong, S Tangada
• Journal: Haemophilia : the official journal of the World Federation of Hemophilia (Haemophilia) Vol. 20 Issue 5 Pg. 644-50 (Sep 2014) ISSN: 1365-2516 [Electronic] England
• PMID: 24589084 (Publication Type: Clinical Trial, Phase III, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
• Copyright: © 2014 John Wiley & Sons Ltd.

Chemical References

• Blood Coagulation Factors
• Coagulants
• Factor VIII
• anti-inhibitor coagulant complex

Topics

• Adolescent
• Adult
• Blood Coagulation Factors (therapeutic use)
• Child
• Coagulants (administration & dosage)
• Factor VIII (administration & dosage)
• Health Status
• Hemophilia A (drug therapy)
• Humans
• Male
• Middle Aged
• Pain Measurement
• Prospective Studies
• Quality of Life
• Regression Analysis
• Surveys and Questionnaires
• Young Adult