Hemophilia A is an inherited bleeding disorder characterized by deficiency of the coagulation protein factor VIII. Development of clotting factor concentrates has resulted in an excellent prognosis for this historically fatal disease. However, neutralizing antidrug antibodies to factor concentrates can develop, complicating management and worsening the prognosis, and thus creating an unmet need for novel therapies. One such agent is emicizumab, a bispecific monoclonal antibody which mimics the function of factor VIII. Collectively across the HAVEN clinical trial program, the rate of antidrug antibodies with neutralizing potential was 0.75%. Since its licensure, there have been no further reports of such antibodies, despite its use in thousands of patients. In this report, we describe a patient with severe hemophilia A with inhibitors who developed a neutralizing antidrug antibody to emicizumab, for whom we performed extensive testing in the special coagulation laboratory.
Keywords: antidrug antibodies; emicizumab; hemophilia; inhibitors; pediatrics.
© 2020 International Society on Thrombosis and Haemostasis.