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.
Haemophilia
; 29(3): 709-715, 2023 May.
Article
de En
| MEDLINE
| ID: mdl-36748858
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.Mots clés
Texte intégral:
1
Collection:
01-internacional
Base de données:
MEDLINE
Sujet principal:
Anticorps bispécifiques
/
Hémophilie A
Type d'étude:
Observational_studies
/
Prognostic_studies
Aspects:
Patient_preference
Limites:
Adolescent
/
Humans
Langue:
En
Journal:
Haemophilia
Sujet du journal:
HEMATOLOGIA
Année:
2023
Type de document:
Article
Pays d'affiliation:
France