Yesim Dargaud, Yasmine Fahfouhi, Monia Ezzalfani, Astrid Foix Colonier, Alaeddine Sidhom, Aline Gauthier, Jérémie Rudant, Meriem Kessouri, Nicolas Giraud, Benoît Guillet, Anaïs Reynaud
Treatment preferences among French patients with haemophilia are highly individualized, reflecting nuanced trade-offs between bleeding risk, treatment convenience and long-term safety that vary according to current therapeutic strategies and individual patient priorities. These findings underscore the importance of shared, personalized decision-making in contemporary haemophilia care.
INTRODUCTION: Haemophilia impacts health and quality of life. Advances in treatments such as factor replacement therapy (FRT), bispecific monoclonal antibody (BS-mAb), gene therapy (GT), rebalancing treatment and high sustained factor necessitate understanding patient preferences to inform therapeutic strategies.
AIM: This study quantified trade-offs of French adult patients with moderate-to-severe haemophilia A/B when choosing between two treatments.
METHODS: A non-interventional, web-based survey using the threshold technique was conducted. Six treatment attributes were identified through systematic review, semi-structured patient interviews and clinical expert consultations: administration frequency, number of annual bleeds, duration of prophylaxis discontinuation, duration of long-term safety data, joint pain after 6 months and preparation/administration requirements. Thresholds were interpreted under fixed attributes assumptions.
RESULTS: Of the 95 recruited patients, 86 were eligible for analysis. Most participants were male (97%), aged 41 on average; 88% had haemophilia A, 87% severe cases. On average, patients accepted 1.1 (median: 0) annual bleeds to switch from BS-mAb-like to high sustained factor-like profiles, 1.5 (median: 0.5) to switch from BS-mAb-like to rebalancing treatment-like profiles and 3.3 to switch from FRT-like to rebalancing treatment-like profiles, holding all else constant. To switch from FRT-like to GT-like profile, patients were willing to accept 4.8 annual bleeds or 12.5 years of prophylaxis discontinuation (median: 1). Patients required lower annual bleeds and longer prophylaxis discontinuation when switching from BS-mAb-like to GT-like profile (3.5 annual bleeds or 14.3 years of prophylaxis discontinuation [median: 2]).
CONCLUSIONS: Treatment preferences among French patients with haemophilia are highly individualized, reflecting nuanced trade-offs between bleeding risk, treatment convenience and long-term safety that vary according to current therapeutic strategies and individual patient priorities. These findings underscore the importance of shared, personalized decision-making in contemporary haemophilia care.