LAUSR

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, emicizumab-related morbidity remains to be solved. AREAS COVERED : The review describes the mode of action, data from pre- /post-marketing and on-going 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 thromboemobolic/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 are required. Furthermore, continuous data collection for emicizumab-related adverse events and morbidity would be recommended.