Development and testing of the Satisfaction Questionnaire with Intravenous or Subcutaneous Hemophilia Injection and results from the Phase 3 HAVEN 3 study of emicizumab prophylaxis in persons with haemophilia A without FVIII inhibitors

• Type of Material: Article
• Authors: Christine Kempton, Emory University; Peter Trask, Genentech Inc; Aric Parnes, Brigham & Womens Hospital; Markus Niggli, F Hoffmann La Roche Ltd; Avrita Campinha-Bacote, Genentech Inc; Michael U. Callaghan, Childrens Hospital Michigan; Niamh O'Connell, St James Hospital; Ido Paz-Priel, Genentech Inc; Johnny N. Mahlangu, University of Witwatersrand
• Language: English
• Date: 2021-01-28
• Publisher: Wiley
• Publication Version: Final Published Version
• Copyright Statement: © 2020 The Authors. Haemophilia published by John Wiley & Sons Ltd.
• License: Creative Commons Attribution-NonCommercial 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1111/hae.14222
• Title of Journal or Parent Work: Haemophilia
• Volume: 27
• Issue: 2
• Start Page: 221
• End Page: 228
• Grant/Funding Information: None declared
• Keywords: SQ-ISHI; Hematology; patient-reported outcomes; haemophilia A; Science & Technology; questionnaire; treatment satisfaction; Life Sciences & Biomedicine; emicizumab
• Research Categories: Health Sciences, Medicine and Surgery; Health Sciences, Health Care Management; Health Sciences, Oncology

Abstract

Introduction Emicizumab is a subcutaneously (SC) administered prophylactic agent for persons with haemophilia A (PwHA). As part of its clinical development, a new instrument was required to measure treatment satisfaction. Aim Describe development of the Satisfaction Questionnaire with Intravenous or Subcutaneous Hemophilia Injection (SQ‐ISHI) and its subsequent testing with HAVEN 3 study participants to measure patient satisfaction with emicizumab. Methods To develop the SQ‐ISHI, we conducted four rounds of in‐person interviews at five qualitative research facilities. Participants aged ≥12 years with moderate or severe haemophilia A, receiving intravenous factor VIII (FVIII) prophylaxis, provided feedback to optimize content understanding, ease of completion and item relevance. The final SQ‐ISHI was completed by HAVEN 3 participants who previously received FVIII prophylaxis; baseline scores were compared with those at Week 21 or 25 of emicizumab prophylaxis. Results Sixty‐three HAVEN 3 participants were eligible to complete the questionnaire and rate their satisfaction on a scale of 0 (‘not at all satisfied’) to 10 (‘extremely satisfied’). Mean ‘overall satisfaction’ with previous FVIII prophylaxis at baseline was 6.9 (95% confidence interval [CI]: 6.2 to 7.7) increasing to 8.8 (95% CI: 8.4 to 9.3) at follow‐up (Week 21/25 of treatment with emicizumab). The greatest improvement was observed in satisfaction with treatment half‐life (mean score at baseline: 5.8 [95% CI: 4.9 to 6.6] vs 8.6 [95% CI: 8.0 to 9.2] at follow‐up). Conclusion These results demonstrate that emicizumab prophylaxis leads to greater treatment satisfaction compared with FVIII prophylaxis, reflecting in part the low treatment burden of emicizumab associated with its infrequent, SC administration.