Haemophilia : the official journal of the World Federation of Hemophilia · 2026
Patient Preferences for the Treatment of Haemophilia A and B in France.
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Dargaud Yesim, Fahfouhi Yasmine, Ezzalfani Monia, Colonier Astrid Foix, Sidhom Alaeddine, Gauthier Aline, Rudant Jérémie, Kessouri Meriem, Giraud Nicolas, Guillet Benoît, Reynaud Anaïs
Аннотация
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.
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.