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.

• Authors: Gaëlle Oka, Valérie Roussel-Robert, Clara Levivien, Isabelle Lopez, Rémi Pieragostini
• Journal: Haemophilia : the official journal of the World Federation of Hemophilia (Haemophilia) Vol. 29 Issue 3 Pg. 709-715 (May 2023) ISSN: 1365-2516 [Electronic] England
• PMID: 36748858 (Publication Type: Journal Article, Review)
• Copyright: © 2023 The Authors. Haemophilia published by John Wiley & Sons Ltd.

Chemical References

• emicizumab
• Antibodies, Bispecific

Topics

• Humans
• Adolescent
• Hemophilia A (drug therapy)
• Quality of Life
• Retrospective Studies
• Patient Satisfaction
• Antibodies, Bispecific (adverse effects)
• Hemorrhage (drug therapy)
• Personal Satisfaction
• Perception