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

Introduction: 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. Aim: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. Methods:The study was observational, retrospective and monocentric. Every patient over 18 years old receiving emicizumab from June 2020 and who underwent the 3 PC 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.Results: 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. Conclusion: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.

Evaluation of the care pathway in the context of the dispensing of emicizumab (Hemlibra) in community and hospital pharmacies in France: A patient satisfaction survey

“…Also, the information to participate relied on information for the general public on social networks and healthcare professionals in reference centre for haemophilia. The current representativeness of the collected sample is difficult to assess but the sample size of this national survey is consistent with other European studies, 20,22,23 covering all ages of patients with no significant differences between community and hospital pharmacy, and all the French regions.…”

“…Eighteen months after availability in French community pharmacies, treatment accessibility was significantly improved for the benefit of the patient; the door‐to‐door travel times were reduced to the community pharmacy, patients were mostly satisfied with the new dispensing circuit as shown in another study, 20 and safety and quality of the treatment administration were preserved. Also, the new scheme of care organization has made a place for the community pharmacists, with effective communication channels between healthcare professionals.…”

“…In addition, community pharmacists were more inclined to use innovative tools to maintain contact with patient or carers such as the mobile text messages 27 . Patients and carers were equally satisfied with the community pharmacist as with the hospital pharmacist 20 . Another initiative has been developed to improve the challenging treatment of haemophilia, particularly for patients who have limited access to haemophilia treatment centre or who have disabilities.…”

“…27 Patients and carers were equally satisfied with the community pharmacist as with the hospital pharmacist. 20 Another initiative has been developed to improve the challenging treatment of haemophilia, particularly for patients who have limited access to haemophilia treatment centre or who have disabilities. This initiative involves the implementation of a mobile haemophilia outpatient care program.…”

Gene Therapy in Hemophilia A: Achievements, Challenges, and Perspectives

“…11 Compared with CFC prophylaxis in patients with HA without inhibitors, emicizumab is associated with lower annualized bleeding rate (ABR). 12 Emicizumab has overall showed improved outcomes with respect to adherence, 13 14 satisfaction, 15 and HRQOL measures. 15 16 Hemostatic rebalancing therapies such as inhibitors of antithrombin (fitusiran), tissue factor pathway inhibitor (concizumab, marstacimab), and activated protein C (serpin PC) as prophylaxis in persons with HA or HB with and without inhibitors are in clinical development 17 with first approvals for concizumab in Japan and Canada.…”