Patient Preferences for the Treatment of Haemophilia A and B in France
Abstract
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.
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BibTeX
@article{dargaud2026patient,
title = {{Patient Preferences for the Treatment of Haemophilia A and B in France}},
author = {Yesim Dargaud and Yasmine Fahfouhi and Monia Ezzalfani and Astrid Foix Colonier and Alaeddine Sidhom and Aline Gauthier and Jérémie Rudant and Meriem Kessouri and Nicolas Giraud and Benoit Guillet and Anaïs Reynaud},
journal = {Haemophilia},
year = {2026},
month = oct,
doi = {10.1111/hae.70298},
url = {https://hal.science/hal-05778903v1},
note = {CC BY-NC 4.0},
}
RIS
TY - JOUR
TI - Patient Preferences for the Treatment of Haemophilia A and B in France
AU - Yesim Dargaud
AU - Yasmine Fahfouhi
AU - Monia Ezzalfani
AU - Astrid Foix Colonier
AU - Alaeddine Sidhom
AU - Aline Gauthier
AU - Jérémie Rudant
AU - Meriem Kessouri
AU - Nicolas Giraud
AU - Benoit Guillet
AU - Anaïs Reynaud
PY - 2026
DA - 2026/10/05
JO - Haemophilia
DO - 10.1111/hae.70298
UR - https://hal.science/hal-05778903v1
AB - 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.
SN - 1351-8216
SN - 1365-2516
ER -