Wilson A, Kragh N, DiBenedetti D, Pan-Petesch B, Wynn T, Neme D, Willemze A. Patient experience with efanesoctocog alfa: results from the XTEND-1 phase 3 clinical trial exit interviews in patients with severe haemophilia A. Poster presented at the 75th Bleeding Disorders Conference (BDC); August 17, 2023. Baltimore, MD. Previously presented at the 16th Annual Congress of European Association for Haemophilia and Allied Disorders 2023.

INTRODUCTION: Qualitative exit interviews complement clinical data and validate patient reported outcomes by providing insight into patient treatment experience. The aim of this analysis was to understand the on-study experience with respect to pain and physical functioning in patients with severe haemophilia A during treatment with efanesoctocog alfa.

METHODS: XTEND-1 (NCT04161495) was a phase 3, open-label, multicentre trial evaluating efanesoctocog alfa in previously treated patients ≥12 years of age with severe haemophilia A. Participants received once-weekly prophylactic efanesoctocog alfa, 50 IU/kg for 52 weeks (Arm A) or on-demand efanesoctocog alfa, 50 IU/kg for 26 weeks followed by 26 weeks once-weekly prophylaxis (50 IU/kg; Arm B). Optional qualitative interviews were conducted using a semi-structured interview guide in a subset of participants from clinical sites in 6 countries following completion of their treatment (52 week). The sites were selected based on feasibility, time to obtain ethics committee reviews and consent.

RESULTS: Exit interviews were conducted for 29 out of the total 159 patients enrolled in XTEND-1 trial (27 adults, 2 adolescents; 28 males, 1 female) with a mean age of 40 years (16−73 years). Interview participants were from Argentina (n=12), the United States (n = 9), South Korea (n=4), France (n=2), the United Kingdom (n=1), and Italy (n=1). Among the 17 participants (58.6%; 17/29) enrolled in Arm A, 13 (76.5%) had reported “wearing off” feeling with prior prophylactic treatment, including more pain and limited physical activities. Nearly all (96.6%; 28/29) participants reported at least moderate haemophilia-related pain and limited physical functioning before the trial. Among these 28 participants, 25 reported meaningful improvement in joint pain, and 1 reported improvement in the ability to move without pain after switching to efanesoctocog alfa. All 29 participants preferred efanesoctocog alfa over prior FVIII treatment. In addition to the decreased bleeding events and improvement in pain, participants also reported increased confidence in protection, less fatigue, and improved health related quality of life compared with previous treatments.

CONCLUSION: Once-weekly prophylaxis with efanesoctocog alfa showed meaningful improvement in pain and physical functioning in patients with severe haemophilia A. These interviews also highlighted additional benefits such as less fatigue and improved health related quality of life.

Share on: