Recombinant factor IX-Fc fusion protein in severe hemophilia B. Patient-reported outcomes and health-related quality of life.pdf (1.06 MB)
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Recombinant factor IX-Fc fusion protein in severe hemophilia B: patient-reported outcomes and health-related quality of life

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posted on 03.06.2022, 16:12 by Mairead O’Donovan, Eimear Quinn, Kate Johnston, Evelyn Singleton, Julie Benson, Brian O'Mahony, Declan Noone, Cleona Duggan, Ruth Gilmore, Kevin Ryan, James O'DonnellJames O'Donnell, Niamh M O’Connell

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.

Funding

iPATH | Funder: Science Foundation Ireland (SFI) | Grant ID: 16/SPP/3303

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History

Comments

The original article is available at https://onlinelibrary.wiley.com/

Published Citation

O'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 Date

11 October 2021

PubMed ID

34667923

Department/Unit

  • School of Pharmacy and Biomolecular Sciences

Research Area

  • Vascular Biology

Publisher

Wiley

Version

  • Published Version (Version of Record)