Recombinant factor IX-Fc fusion protein in severe hemophilia B: Patient-reported outcomes and health-related quality of life

Res Pract Thromb Haemost. 2021 Oct 11;5(7):e12602. doi: 10.1002/rth2.12602. eCollection 2021 Oct.ABSTRACTINTRODUCTION: 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
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Source Type: research

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