Recombinant factor IX-Fc fusion protein in severe hemophilia B: patient-reported outcomes and health-related quality of life
Introduction: In 2017, all people with severe hemophilia B in Ireland switched to recombinant factor IX Fc fusion protein concentrate (rFIXFc) prophylaxis. Patient-reported outcomes (PROs) and health-related quality of life (HRQoL) are important to evaluate with new treatments.
Aims: To assess HRQoL in people with severe hemophilia B and their experience after switching to rFIXFc prophylaxis.
Methods: Participants completed a Patient Reported Outcomes Burden and Experience (PROBE) questionnaire on initiation and following two years of rFIXFc prophylaxis. The PROBE questionnaire has four domains: demographics, general health, haemophilia-specific, and European Quality of Life 5-Dimensions (EQ-5D-5L) questionnaire.
Results: Twenty-three participants completed the questionnaire at both time points. The number of activities where chronic pain occurred and interfered with the activity was reduced by 25% and 33%, respectively (P < .001), following two years of rFIXFc prophylaxis. There was a 9% decrease in chronic pain during the second year of rFIXFc prophylaxis compared to baseline, but the rate remained high, at 74%. A 25% reduction in the number of affected activities of daily living (ADLs) was reported following 2 years of rFIXFc prophylaxis (P = .007). The most common health problems were arthritis, hypertension, anxiety/depression, and gingivitis. The median EQ-5D-5L score was similar following two years of rFIXFc prophylaxis, 0.76 (range, -0.01 to 0.95), compared to 0.77 (range, 0.36-1) at baseline.
Conclusion: This study of real-world patient experience using PROs demonstrates a reduction in chronic pain and improvement in ADLs in participants after switching to rFIXFc prophylaxis. It provides important insights into patient-identified health care needs and living with severe hemophilia B.
iPATH | Funder: Science Foundation Ireland (SFI) | Grant ID: 16/SPP/3303
CommentsThe original article is available at https://onlinelibrary.wiley.com/
Published CitationO'Donovan M. et al. Recombinant factor IX-Fc fusion protein in severe hemophilia B: patient-reported outcomes and health-related quality of life. Res Pract Thromb Haemost. 2021;5(7):e12602.
Publication Date11 October 2021
- School of Pharmacy and Biomolecular Sciences
- Vascular Biology
- Published Version (Version of Record)