Perspectives on Selected Hemophilia Research Presented at ISTH 2025: Nonfactor Replacement Therapies

Conclusions

Dr. von Drygalski shares her thoughts about the importance of this research and its relevance to clinical practice.

Factor VIII Mimetics

Abstract 4: Effectiveness of emicizumab in hemophilia A: A 4th interim analysis of the real-world study EMIIL Abstract 5: Prospective evaluation of a defined replacement protocol for peri-operative hemostasis in patients with severe hemophilia A with and without inhibitors on emicizumab prophylaxis undergoing elective major surgeries-final results. (EmiSurg Study) Abstract 6: FRONTIER5 direct switch study: Safety of initiating Mim8 prophylaxis without washout of emicizumab

A Focus on Rebalancing Agents

Abstract 1: Association of antithrombin levels with efficacy of fitusiran prophylaxis in people with hemophilia A or B with and without inhibitors: a predictive modeling approach Abstract 2: Annualized bleeding rates in hemophilia A/B and target joints: Concizumab explorer8 study Abstract 3: Long-term efficacy of marstacimab in participants with severe hemophilia A or B without inhibitors