Understanding unmet needs for people with haemophilia a receiving factor and non-factor treatments

Abstract

Abstract BACKGROUND: There is limited published real-world evidence on treatment patterns among people with severe haemophilia A (PwSHA), particularly those with unmet clinical needs. This qualitative study explored the experiences of PwSHA without inhibitors, treated with extended half-life (EHL) factor VIII or emicizumab, to shed light on the psychological, social, and physical impact of prophylactic treatments. METHODS: This was a qualitative study with a sample of n=69 adults with severe haemophilia A without inhibitors recruited from the United Kingdom, Spain, Italy, France, Germany (n=10 interviews per country), and the United States (n=19 interviews). 54% of the sample received emicizumab and 46% received EHL factor products, for a minimum of one year. Study materials were created in collaboration with patient advocacy group representatives. PwSHA who were not satisfied with their treatment, or were satisfied but reported unmet needs (such as persistent symptoms or use of additional factor medication) were recruited via a patient panel and screened for eligibility via an online survey. Respondents then participated in 30-minute online semi-structured interviews conducted by trained moderators. The interviews were recorded, translated and transcribed for analysis. Interviews took place from mid-October 2024 to end of March 2025. The transcripts were coded using framework analysis, then the most appropriate coded excerpts were extracted and condensed. The codes were then grouped into over-arching themes based on patterns, similarities, and recurring concepts, as well as concepts most relevant to the research question. RESULTS: All participants were male, with an average age of 43 years in the emicizumab group and 37 years in the EHL group. The qualitative interviews provided an in-depth picture of the treatment patterns and unmet needs of the PwSHA. Five themes identified across both EHL and emicizumab interview groups were: High symptom burden, Treatment/management patterns, Impact of symptoms on daily activities, Satisfaction with current treatment, and Ideal treatment characteristics. Both groups reported a substantial disease burden, including breakthrough bleeds (either spontaneous or injury-related), pain, and inflammation from long-term joint damage. Symptom management often required additional factor use (reported by approximately 90% of both emicizumab and EHL users). Other management included pain medication and alternative therapies to maintain mobility. While all participants aimed to stay active, both emicizumab and EHL users often pushed through haemophilia-related pain, with some avoiding certain physical activities due to bleed risk. In addition, over 90% of PwSHA in both emicizumab and EHL groups reported some level of daily disability adaptation to the disease burden. Both groups were generally satisfied with the benefits provided by their treatment, however in spite of the benefits observed, there were still several ways in which PwSHA felt their treatment could be improved. A proportion of PwSHA (65% in the emicizumab group and 78% in the EHL group) also described the need for a treatment to provide a higher level of protection. Moreover, EHL users indicated that longer factor coverage would lessen the mental burden of missing a dose or not carrying enough factor. Study findings may be subject to certain limitations, such as recall bias, limited transferability, and moderator effects. However, these are inherent to the retrospective and qualitative nature of this research. CONCLUSIONS: This study offers an in-depth picture of treatment patterns and remaining unmet needs among adults with severe haemophilia A, taking emicizumab or EHL FVIII prophylaxis. Interviews revealed a persistent symptom burden- bleeds, pain and joint damage – affecting daily life. Despite their efforts to remain active, PwSHA described ongoing limitations due to haemophilia pain and various adaptations required to manage their condition and maintain their current lifestyle. In addition, despite differences in route of administration and dosing frequency between treatments, both emicizumab and EHL groups reported a continued use of additional factor, alongside their regular prophylactic treatment. Other unmet needs were also prevalent across groups, with PwSHA reporting dissatisfaction with the duration of treatment coverage and with treatment administration frequency.