Assessment of the clinical perception, quality of life and satisfaction of patients with severe congenital haemophilia A without inhibitor after 1 year of emicizumab therapy.

Abstract

Since the approval by the EMA of emicizumab for the care of severe haemophilia A without inhibitor, most of the patients of our haemophilia treatment centre started this new treatment. Thanks to the setting of a therapeutic patient education program including three pharmaceutical consultations (PC), we could follow patients' lifestyle evolution. The study aimed to assess the perceived clinical evolution, quality of life and treatment satisfaction of patients after 1 year of emicizumab therapy in real-life settings. The study was observational, retrospective and monocentric. Every patient over 18 years old receiving emicizumab from June 2020 and who underwent the 3PC until March 2022 were included. The clinical evolution was self-estimated by patients with zero-to-six scales before versus 1 year after emicizumab, according to the following parameters: general health state, pain and bleedings (spontaneous or post-traumatic, and patients' identification ability). Patients' quality of life was also estimated with the EQ-5D-3L survey. Their satisfaction, graduated with a zero-to-ten scale, and treatment management were reported during the third PC. Thirty-eight patients were enrolled. Their general health state improved significantly (p=.0023) with an EQ-5D-3L score at 69.6 (±19.4) out of 100. Although chronic pains remained a persistent issue for 33 (86.8%) patients, their intensity was significantly decreasing after 1 year. Perceived frequency of bleedings was significantly reduced too. On average, the satisfaction of emicizumab therapy was 9.1 (± 1.02) out of 10. After 1 year of emicizumab therapy, the general health state estimated by patients improved, the pain and the perceived frequency of bleedings diminished. Overall, this treatment received a high patients' satisfaction rate.