Emicizumab Prophylaxis Provides Flexible and Effective Bleed Control in Children with Hemophilia Α with Inhibitors

BestPractice | Joulu 2018 | |

In this MEDtalk Guy Young present data from HAVEN 2 – the largest prospective study in pediatric PwHA with inhibitors to date. HAVEN 2 demonstrates that emicizumab prophylaxis is well tolerated and can prevent or substantially reduce bleeds in this population. Meaningful efficacy and PK were maintained with less frequent dosing, with no new safety signals, suggesting the potential for reduced treatment burden in the pediatric population. Additionally, the large number of CVAD removals suggests that prophylactic emicizumab may offer a new and effective standard of care for hemophilia that is also more convenient and less invasive, and may offer the potential for flexible treatment regimens based on patient needs.