Lessons from a qualitative study of treatment experiences and perceptions in people with haemophilia in France

Abstract Introduction Haemophilia A and B (HA/HB) are congenital, X-linked recessive bleeding disorders caused by deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively. People with haemophilia (PwH) have increased risk of spontaneous or traumatic bleeding in joints, muscles, or soft tissues, which can be severe in people with HA/HB with inhibitors (HAwI/HBwI). Despite advances in haemophilia treatment, there are remaining and emerging unmet needs in PwH. Aims This study explored emerging unmet needs in PwH in France, with a focus on treatment burden, by investigating patient perspectives using ethnographic interviews. Methods PwH were screened to encompass diverse characteristics including treatment and haemophilia types. Ethnographic, semi-structured interviews were conducted using open-ended questions on topics such as identity and social perception, treatment history and routines around managing haemophilia. Patient responses were coded to identify core themes. Results PwH (N=13 [HAwI: n=6; HB: n=7]) from France evaluated their treatment (emicizumab [n=4] or various factor replacement therapies [n=9]) primarily based on three emerging themes from the interviews: perceived efficiency, autonomy, and normalcy. Understanding of a treatment’s mechanism of action was linked to their perception of efficiency. In terms of autonomy, administering treatment independently from healthcare system structures, and treatment accessibility and storage were highlighted as key. The need to feel normal without the stigma associated with treatment administration, was ranked as the highest aspirational treatment need among PwH compared with the other themes. Conclusion These findings support the need for targeted and personalised treatment approaches in haemophilia care.