Emicizumab for Hemophilia: Takeaways from Phase 3 HAVEN Trial Data


Doris Quon, MD, Medical Director at the Orthopaedic Hemophilia Treatment Center, discusses the main takeaways from phase 3 HAVEN trial data for emicizumab (Hemlibra) prophylaxis in hemophilia. 


The phase 3 HAVEN 1 data was for adults and adolescents, and what it showed was that Hemlibra prophylaxis can reduce the annualized bleed rate and compared to no prophylaxis, the reduction in the bleed rate was 87%. And there was the ability to do the intra-patient comparison, so patients were monitored for 6 months prior to going into the HAVEN trial. They logged their bleeds as well as their use of clotting factor bypass agents, and even there there was a reduction in the bleeding episodes, 79% I believe.
So the takeaway shows that there is a reduction in the bleed rate. There were safety signals, but I think that good communication is the key. Talk to the patient prior to starting the drugs and make a plan when they have a bleeding event to either call the health care team or to have a plan for the bleeding event, what to use, what not to use, and how to treat it.