Patient Perspectives in Haemophilia A: Unmet Needs of Individuals Receiving Factor and Non-Factor Treatments.

INTRODUCTION

There is limited qualitative published real-world data on treatment patterns among people with severe haemophilia A (PwSHA), particularly those with unmet needs.

AIM: This qualitative study aimed to explore the impact of taking prophylactic extended half-life factor VIII (EHL-FVIII) or emicizumab on psychosocial and physical experiences of PwSHA.

METHODS

This study included 69 adults with severe haemophilia A without inhibitors from France, Germany, Italy, Spain, UK (n = 10 per country), and US (n = 19), all receiving emicizumab (54%) or EHL FVIII (46%) for ≥1 year. Participants reporting treatment dissatisfaction or unmet needs were recruited via a patient panel and completed 30-minute online semi-structured interviews. Interviews were recorded, transcribed, and analysed using framework analysis, with codes condensed and grouped into overarching themes reflecting recurring patterns and relevance to the research question.

RESULTS

Five themes were identified from the study interviews: high disease burden, treatment/management patterns, impact of symptoms on daily activities, satisfaction with current treatment, and ideal treatment characteristics. Both subgroups reported breakthrough bleeds, pain, and joint damage, often requiring additional factor (reported by ∼90%) and other strategies to maintain mobility. While generally satisfied, 65% of emicizumab users and 78% of EHL-FVIII users expressed a need for greater protection.

CONCLUSION

This study examined treatment patterns and unmet needs in adults with severe haemophilia A receiving emicizumab or EHL-FVIII. Persistent symptoms (bleeds, pain, and joint damage) imposed limitations on PwSHA's daily life, and both subgroups reported continued use of additional factor therapy, as well as reduced satisfaction with treatment protection and administration frequency.