For a study, researchers sought to understand that there is a dearth of clinical trial-based and real-world data on emicizumab therapy for pediatric patients (PUPs) with hemophilia A. Particularly for minimally treated patients (MTPs) and pediatric patients who have received little to no treatment, the data are insufficient. A total of 13 pediatric hemophilia A patients’ annual bleeding rates (ABR) before and after being given emicizumab were retrospectively analyzed. Laboratory test results, safety data, and management information for minor surgery were acquired. The clinical characteristics of two PUPs and one MTP from their sample were also described. When emicizumab treatment began, 3 patients were under a year old, with a median age of 5.3 years (with a range of 0.26 to 17.5). For emicizumab, the average follow-up period was 23.8 months (with a range of 0.7 to 40). Total ABR (P=0.009), spontaneous ABR (P=0.018), traumatic ABR (P=0.018), and joint ABR (P=0.027) all showed significantly reduced post-Emicizumab transition ABR. Because there was only 1 local site reaction and no treatment interruption was necessary, the safety profile was good. Three patients who took rFVlla both before and after the procedure underwent the procedure satisfactorily. Emicizumab trough levels averaged 43.2 μg/ml (range: 23.9-56.8) after 3 doses of 3 mg/kg, and at the first follow-up with 1.5 mg/kg, they were 51.9 μg/ml (range: 30.4-75). With or without inhibitors, emicizumab was safe and effective for pediatric patients. More information was still needed, particularly regarding bigger multicenter groups and PUPs/MTPs.

Source: bmcpediatr.biomedcentral.com/articles/10.1186/s12887-022-03546-1