Prospective, non-interventional study with a cohort to assess real-world effectiveness (RWE) and safety of emicizumab in persons with hemophilia A (PWHA) without inhibitors in Spain, hemolution cohort

ABSTRACT

Background: Haemophilia A (HA) is a disorder characterized by deficiency in clotting FVIII that affects daily lives of patients and caregivers. Emicizumab is a humanized, bispecific, monoclonal antibody, which substitutes missing activated FVIII. Aims: Effectiveness and safety on emicizumab in clinical practice. Methods: Hemolife study initiated in March 2020, describes the socio-occupational impact of HA without inhibitors in patients and caregivers, in terms of quality of life, activity, and productivity/absenteeism, among others. At that time, both COVID-19 pandemic and emicizumab approval for PwHA without inhibitors happened. A multidisciplinary Scientific Committee of experts in HA, rethought Hemolife study to include a cohort of patients treated with emicizumab (Hemolution), resulting in a more complete and stronger RWE study, and avoiding logistical duplications that a separate study would mean to centers and patients. 2020 has been a year for adaptation, efficiency and re-design;also, in RWE context. 100 PwHA will be followed for 12 months within Hemolife. At any time, a patient prescribed with emicizumab may be enrolled in Hemolution cohort, followed for at least 8 months after initiation of emicizumab to collect efficiency and safety data (Figure 1). Finally, Hemolife involves telemedicine;in a pandemic setting, Hemolution could also benefit from this live data collection and may shape a new and more dynamic form of care in HA that avoids being in the hospital. Results: Ongoing study. Protocol was submitted and approved by authorities/ethics committee's (Table 1). Conclusions: By this novel approach we will gather information on emicizumab in clinical practice and provide relevant information to the Haemophilia community, contributing to improve patient care in HA.