Non-factor replacement therapy: A new chapter in hemophilia a prophylaxis

Abstract

Introduction/Aim. Hemophilia A is an antihemophilic factor deficiency which requires life-long treatment. The aim of this analysis was to present the effects of prophylactic non-factor replacement therapy in ten patients with hemophilia A. Patients and methods. This retrospective analysis was conducted on ten male patients (4 children, 1 adolescent, and 5 adults) with severe hemophilia A and a history of antihemophilic factor replacement prophylaxis, prior to the initiation of emicizumab prophylaxis. A single adult patient developed inhibitors during the course of factor replacement prophylaxis. Four adult patients had already developed hemophilic arthropathy before the initiation of non-factor replacement prophylaxis. Two adult patients received emicizumab prophylaxis every four weeks, while others received emicizumab every two weeks. After a 14-month period (average) of non-factor replacement prophylaxis, we analyzed the number of breakthrough bleeding episodes, annualized bleeding rate, involvement of target joints, adverse drug reactions, and interviewed the patients regarding their satisfaction with the non-factor replacement treatment. Results. None of the patients on emicizumab prophylaxis experienced breakthrough bleeding or clinical worsening of the affected target joints during the period of emicizumab prophylaxis. Annualized bleeding rate was zero in all patients on emicizumab prophylaxis. No adverse drug reactions occurred in our patients during emicizumab prophylaxis. All patients reported greater treatment satisfaction compared to the replacement prophylaxis. Conclusion. By providing safety from bleeding events and potentially the stability of the affected joints, emicizumab prophylaxis enables greater activity and increases the quality of life of treated patients.