PracticeUpdate Conference Series: ASH 2017

Weekly Subcutaneous Emicizumab a New Standard of Care in Hemophilia Management

In the largest study of pediatric hemophilia A with inhibitors to date, emicizumab prophylaxis prevented or reduced bleeds substantially and was well tolerated, updated analysis of the multicenter, open-label, phase III HAVEN 2 trial reports. G uy Young, MD, of the Children's Hospital of Los Angeles, and University of Southern California Keck School of Medicine, explained once-weekly subcutaneous emicizumab prophy- laxis in pediatric hemophilia A with inhibitors.

“I chose to participate in this study,” Dr. Young told Elsevier’s PracticeUpdate , “because I felt this medication could offer a great benefit for my inhibitor patients who were continuing to suffer with repeated bleeding events and the associated pain and morbidity. In addition, our center makes every effort to bring innovative therapies to our patients as soon as possible.” The study enrolled children with hemophilia A with inhibitors age 2–12 years (or 12–17 years of age in those weighing <40 kg), and is enrolling those <2 years of age treated previously with bypassing agents to emicizumab prophylaxis for ≥52 weeks. Efficacy analyses included annual bleed rate and bleed reduction vs annual bleed rate on prior bypassing agent treatment from a prospective non-interventional study. Health-related quality of life, aspects of caregiver burden, and safety parameters were also assessed.

that emicizumab is a bispecific humanized mon- oclonal antibody administered subcutaneously. Emicizumab bridges factors IXa and X to restore the function of missing factor VIIIa. Emicizumab is being developed to prevent bleeds in patients with hemophilia A with and without inhibitors. In 2017, an interim analysis of the HAVEN 2 study (n=20) in patients age 2–12 years (data cut-off in October of 2016) showed that subcutaneous, once-weekly emicizumab prophylaxis prevented or reduced bleeds successfully, provided clinically meaningful reductions in the annualized bleed rate vs prior bypassing agent treatment, and was well tolerated. At ASH, Dr. Young presented an updated, much larger (40 additional patients, 60 total) analy- sis of efficacy, safety, and pharmacokinetics of

4

PRACTICEUPDATE CONFERENCE SERIES • ASH 2017