OBJECTIVES

: Emicizumab is a monoclonal antibody developed for treatment of patients with haemophilia A (HA). Alongside clinical development program; qualitative data have been collected from patients and healthcare professionals (HCPs).

METHODS

: Japanese HA patients with inhibitors from the emicizumab phase 1 study answered open-ended questions about their daily life while treated with emicizumab. Translation of their written answers was analyzed. Six French HCPs who had been in contact with French patients with inhibitors treated with emicizumab as part of the HAVEN 1 and 2 phase 3 studies were interviewed by phone. Specific interview guides were used, to collect information about the impact of emicizumab treatment in patients’ daily life. Interviews were exploratory, semi-structured, and lasted 1 hour. Thematic qualitative analysis on the interview transcripts and written answers of the phase 1 study was conducted. Then, a conceptual model was developed describing the experience of patients with HA treated with emicizumab.

RESULTS

: French HCPs (n=3 physicians, 2 nurses and 1 CRA) and Japanese patients (n=11) reported “spectacular” changes in patients’ daily lifestyle while treated with emicizumab. Patients reported changes they qualified significant: decrease in bleeding frequency, severity and associated joint/muscle pain. This led to improved emotional state, increased daily activities, better autonomy, and for some patients a major change in their daily lifestyle. Patients could travel further away from home thanks to treatment’s portability, ease of use of subcutaneous injection, and low frequency of injections. HCPs reported that patients were feeling more confident in the future, were able to make plans again, and treatment was so effective and convenient to use that some patients were not feeling ill anymore.

CONCLUSIONS

: Emicizumab significantly changed the way patients handled HA, and resulted in a transformation to their lifestyle. This may also have an impact on organization in HCPs’ health unit and medical practices.