Effects of emicizumab on the quality of life of children with hemophilia A and their families

Abstract

With the remarkable progress in the treatment of hemophilia in recent years, preventing bleeding and improving the quality of life (QOL) of patients and their families have become important indicators of treatment efficacy. Emicizumab, an antibody preparation, has distinct characteristics from coagulation factor VIII concentrates, such as a subcutaneous route of injection and a long half-life, which are expected to improve QOL. International clinical trials including the HAVEN trials; clinical trials in Japanese patients, such as the phase 1/2 trial of emicizumab and HOHOEMI study; and other surveys have reported that emicizumab improved QOL in children and adult patients regardless of the presence or absence of inhibitors. Furthermore, the Emicizumab Preference Survey showed that the treatment was highly preferred by both pediatric and adult patients, which was related to the aforementioned characteristics of emicizumab. Experiences at Miyagi Children’s Hospital have shown that emicizumab may contribute to the improvement of the QOL of children with ­hemophilia A and their families. In the future, widespread use of emicizumab is anticipated, and advances in new drugs will further improve the QOL of children with hemophilia and their families.