Key clinical point: Administration of twice monthly or monthly emicizumab appears safe and effective for children with severe hemophilia A without inhibitors.
Major finding: Among patients in the 2- and 4-week dosing cohorts, the annualized bleeding rates for treated bleeding episodes were 1.3 and 0.7, respectively.
Study details: A nonrandomized study of 13 pediatric patients with severe hemophilia A without inhibitors.
Disclosures: The authors reported having financial affiliations with Chugai Pharmaceutical Co., which funded the study, and other companies.
Shima M et al. Haemophilia. 2019 Sep 12. doi: 10.1111/hae.13848.