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

ABSTRACT:

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 que

SUBMITTER: O'Donovan M 

PROVIDER: S-EPMC8505226 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature