Statistically significant improvement in hemophilia A control: a retrospective analysis of the effectiveness and safety of emicizumab in children with severe and inhibitor forms of hemophilia A

• DOI: 10.24287/1726-1708-2024-23-1-99-107
• Publication Date: 2024-04-19T14:25:39Z
• Authors: V. Yu. Petrov; I. N. Lavrentyeva; V. V. Vdovin; P. V. Svirin
• Keywords: 0301 basic medicine; 03 medical and health sciences

Hemophilia A presents a serious problem, especially in its severe and inhibitor forms, leading to bleeding complications. The importance of studying the effectiveness safety new treatment approaches, particularly emicizumab, is undeniable for improving quality life patients. Aim: evaluate emicizumab prevention children with forms hemophilia A. Ethical approval was not required since study only involved use generalized retrospective data obtained during routine clinical practice. All were depersonalized. analysis medical records 45 treated at Morozov Children's Hospital from 2006 2022 carried out. included two cohorts: those forms. an assessment frequency hemarthrosis episodes, hospital admissions, adverse reactions. patients who underwent 2018 2022. Of these, 30 had form A, 15 form. median follow-up period 17 months, ranging 12 23 while form, it 32 range 11 51 months. In group statistically significant (p < 0.001) reduction all types observed: 96.7 % no episodes therapy, compared 46.7 previously FVIII concentrates. No spontaneous registered. Similar results observed A: 93.3 using 13.3% previous bypassing agents. Over entire period, there 3 bleedings cohort 1 Prior out 391 that occurred children, 218 spontaneous. Adverse events recorded 7 manifesting as erythema injection site after first or second injections resolving spontaneously. There other events; 90 73.3 did report any emicizumab. Emicizumab demonstrates high both without inhibitors. drug significantly reduces improves