ASH: Hematology

by Mike Bassett, Staff Writer, MedPage Today

December 10, 2023

SAN DIEGO — Administering emicizumab (Hemlibra) to previously untreated or minimally treated infants with severe hemophilia A without factor VIII inhibitors is effective and safe, according to results from research presented here.

The phase III HAVEN-7 study showed that at a median follow-up of 101.9 weeks, the annualized treated bleeding rate was 0.4 (95% CI 0.30-0.63) among 55 study participants, with 30 children (54.5%) having no treated bleeds, reported Steven Pipe, MD, of the University of Michigan in Ann Arbor.

The annualized bleeding rate for all bleeds was 2.0 (95% CI 1.49–2.66), with no treated spontaneous bleeds.

“This was over a median follow up of over 100 weeks, and the median age was 29 months — so these are totally mobilized toddlers through this clinical cut-off date.” Pipe said during a press briefing at the annual meeting of the American Society of Hematology, “Emicizumab prophylaxis really demonstrated consistent efficacy across all the bleeding endpoints.”

For decades the approach to managing hemophilia has been the use of prophylactic factor VIII protein with factor VIII concentrates. Pipe pointed out this is a challenging process because it requires intravenous infusions frequently because of the short half-life of factor VIII.