Related Experiment Video
Updated: Nov 2, 2025
Tail Vein Transection Bleeding Model in Fully Anesthetized Hemophilia A Mice
Published on: September 30, 2021
Bispecific antibodies for the treatment of hemophilia A
1Thrombosis and Hemostasis Research Center, Nara Medical University, Kashihara-shi, Japan.
Introduction:
Emicizumab is a bispecific antibody exerting cofactor function of FVIIIa irrespective of the presence of FVIII inhibitors. Long-term data of phase 1/2 and phase 3 studies have been accumulated. Various questions such as indicated patients, ITI, application to PUPs, hemostatic treatment including surgeries, and emicizumab-related morbidity remain to be solved.
Areas Covered:
The review describes the mode of action, data from pre-/post-marketing and ongoing clinical studies according to PubMed search and our own works.
Expert Opinion:
For patients with a persistent inhibitor, emicizumab is a definite therapeutic option, although the possibility of BPAs-associated thromboembolic/TMA events raises concerns. The use of ITI together with emicizumab prophylaxis is being examined in clinical trials. For non-inhibitor, especially pediatric patients, emicizumab prophylaxis can be an option. Outcome assessment 'beyond ABR' such as joint health, physical/mental activity, QOL is required. Furthermore, continuous data collection for emicizumab-related adverse events and morbidity would be recommended.
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