Shima M, Nogami K, Nagami S, et al. - Researchers focused on the effectiveness, safety as well as pharmacokinetics of emicizumab (a recombinant humanized bispecific monoclonal antibody imitating the cofactor function of activated factor VIII) in Japanese paediatric patients aged < 12 years with severe haemophilia A without factor VIII inhibitors. At least 24 weeks of therapy was received by all participants. Via subcutaneous route, emicizumab was delivered with 4 loading doses of 3 mg/kg every week followed by maintenance doses of 3 mg/kg every 2 weeks (Q2W) or 6 mg/kg every 4 weeks (Q4W) in 6 and 7 patients, respectively. No treated bleeding events were reported in 2/6 and 5/7 patients in the respective Q2W and Q4W cohorts, and 1.3 and 0.7 were the annualized bleeding rates for treated bleeding events. Compared with the patient's previous treatment, emicizumab was favored by all caregivers. There was only one associated adverse event (injection site reaction). No thromboembolic events or thrombotic microangiopathy was reported. Findings revealed the safety as well as the efficacy of emicizumab administered Q2W or Q4W for the treatment of paediatric patients with severe haemophilia A without inhibitors.