Bispecific antibodies for the treatment of hemophilia A

This is the authors' abstract. We don't add key points for this paper.

INTRODUCTION: Emicizumab is a bispecific antibody exerting cofactor function of FVIIIa irrespective of the presence of FVIII inhibitors. Long-term data of phase 1/2 and phase 3 studies have been accumulated. Various questions such as indicated patients, ITI, application to PUPs, hemostatic treatment including surgeries, and emicizumab-related morbidity remain to be solved. AREAS COVERED: The review describes the mode of action, data from pre-/post-marketing and ongoing clinical studies according to PubMed search and our own works. EXPERT OPINION: For patients with a persistent inhibitor, emicizumab is a definite therapeutic option, although the possibility of BPAs-associated thromboembolic/TMA events raises concerns. The use of ITI together with emicizumab prophylaxis is being examined in clinical trials. For non-inhibitor, especially pediatric patients, emicizumab prophylaxis can be an option. Outcome assessment 'beyond ABR' such as joint health, physical/mental activity, QOL is required. Furthermore, continuous data collection for emicizumab-related adverse events and morbidity would be recommended.