Health-related quality of life in children with severe hemophilia A on emicizumab prophylaxis

Abstract

Background Emicizumab is a novel nonreplacement therapy for patients with hemophilia A. It is intended to reduce the frequency and severity of serious bleeding episodes in patients with hemophilia A. Aim To assess how emicizumab affected quality of life and physical activity in children with severe hemophilia A. Patients and methods Thirty-seven children with severe hemophilia A received emicizumab prophylaxis. We used EQ-5D-Y and pedHAL activity list questionnaires to assess quality of life and physical activity, respectively, at baseline (before receiving emicizumab prophylaxis) and after 1 year of regular emicizumab prophylaxis. Results The mean age of the children included in the study was 9.32 ± 3.75 years. Of them, 19 patients were with FVIII inhibitors (51.35%), and 18 patients were negative for FVIII inhibitors (48.65%). The median (interquartile range) PedHAL sum score was 57.6 (48.8–62) at baseline, then reached 76 (70–81.5) after a year of emicizumab prophylaxis; this change was found to be statistically significant ( P <0.001). Also, there was a significant increase in the proportion of patients with no problem in all EQ-5d-Y dimensions after a year of emicizumab prophylaxis ( P <0.05). The median (interquartile range) value of the visual analog scale score at baseline was 67 (54–75) and became 76 (70–81.5) after a year of receiving emicizumab prophylaxis. This improvement was statistically significant ( P <0.001). Conclusion Emicizumab prophylaxis improves quality of life and physical activity in children with severe hemophilia A.