Real-World Effectiveness of rFIXFc Prophylaxis in Patients with HaemophiliaB Switched from Standard Half-Life Therapy in Three European Countries.

Abstract

The current study describes real-world clinical outcomes and factor usage among patients with haemophiliaB switching from standard half-life factorIX (SHL FIX) treatment to recombinant factorIX Fc fusion protein (rFIXFc) prophylaxis in European treatment centres. This non-interventional, retrospective, multicentre chart review evaluated medical records from adult and paediatric patients with haemophiliaB in Denmark, Germany and the UK. Patients had documented SHL FIX treatment, on-demand or prophylaxis, for ≥ 6months before starting rFIXFc prophylaxis, and subsequent data for ≥ 6months afterwards (up to 24months). Primary endpoints included annualised bleeding rates (ABRs), prophylactic factor consumption and injection frequency. Data from 30 patients (24/30 [80.0%] with severe disease) showed overall mean (standard deviation, SD) ABRs of 4.7 (6.3) on SHL FIX treatment and 1.7 (2.3) after switching to rFIXFc prophylaxis. The reduction in mean (SD) ABRs was greater when switching from SHL FIX on-demand treatment (n = 6), with a decrease from 10.5 (9.9) to 2.6 (4.5), than when switching from SHL FIX prophylaxis (n = 24), with a decrease from 3.3 (4.3) to 1.5 (1.4). Among prior SHL FIX prophylaxis patients, switching to rFIXFc prophylaxis increased the proportion of those with zero bleeds from 21.7% to 45.8% during the 6months before and after switching, respectively. In the total population, five of six target joints (83.3%) present when patients started rFIXFc prophylaxis subsequently resolved. In patients switching from SHL FIX prophylaxis to rFIXFc prophylaxis, mean (SD) weekly injection frequency was reduced by 1.0 (0.7) and mean (SD) factor consumption was reduced by 27.7 (49.6) IU/kg/week. This study demonstrates the effectiveness of rFIXFc prophylaxis in real-world clinical practice. Improvements in both clinical effectiveness and factor usage associated with rFIXFc prophylaxis may potentially reduce patient burden and improve quality of life.