Monthly Roundup

This study explored strategies for transitioning hemophilia A patients from emicizumab prophylaxis to gene therapy with valoctocogene roxaparvovec. Using pharmacokinetic simulations, the study assessed bleeding risks for two transition scenarios: administering the last emicizumab dose either on the same day or four weeks after gene therapy. Results showed comparable bleeding risks in both cases, as emicizumab levels decline gradually while Factor VIII levels rise rapidly post-therapy. Simulated clinical examples demonstrated that patients can safely transition without requiring a washout or reverting to clotting factor prophylaxis, provided Factor VIII is monitored using a bovine chromogenic assay.