Efficacy, safety and surgical management with concizumab prophylaxis in three young patients affected by severe hemophilia with inhibitors

Concizumab is an anti-tissue factor inhibitor monoclonal antibody designed as a once-daily subcutaneous prophylactic treatment for all hemophilia patients. The efficacy and safety of concizumab have been documented in the Explorer research program. The management and clinical outcomes of 3 subjects treated with concizumab are described. During the 5 years following the switch to concizumab all the patients had a drastic reduction in bleeding events and therefore of treatment with rFVIIa. A significant improvement in quality of life, both in relation to pain reduction, movement ability and autonomy from caregivers, was achieved. No side effects were observed, and the management of 1 major and 14 minor surgeries did not present any bleeding or thrombotic complications. Concizumab represents a unique opportunity for patients with hemophilia B with inhibitors, but it may be of extreme value also for patients with hemophilia A with inhibitors. In our cases concizumab demonstrates and confirms clinical efficacy, positive impact on quality of life and safety even in surgical settings.