Management of children with hemophilia A: How emicizumab has changed the landscape

Abstract

The key to having a good quality of life for an adult with hemophilia rests largely on how he or she was managed as children. With effective prophylaxis, young men can begin their adult life with excellent joint function and few, if any, other sequelae from their disease. Unfortunately, this outcome is not always (nor often) attained because of the limitations of the mainstay of treatment, which is factor replacement therapy. In resource-rich countries with an adequate supply of factor concentrates, the treatment burden and formation of inhibitors limit the potential for an ideal outcome, whereas in much of the world, factor concentrates are too expensive to even be an option. The novel agent, emicizumab, which has become available in numerous countries around the world, is reshaping how one approaches the treatment of children with hemophilia A. This Forum Article, based on a State-of-the-Art lecture given at the 2020 International Society on Thrombosis and Haemostasis Virtual Meeting, presents an approach including clinically applicable algorithms for treating children with hemophilia A in the new era with emicizumab.