Title :

Adherence with metreleptin therapy and health self-perception in patients with lipodystrophic syndromes

Author(s) :

Vatier, Camille [Auteur]
Kalbasi, Dina [Auteur]
Vantyghem, Marie-Christine [Auteur]
Recherche translationnelle sur le diabète (RTD) - U1190
Lascols, Olivier [Auteur]
Jéru, Isabelle [Auteur]
Daguenel, Anne [Auteur]
Gautier, Jean-François [Auteur]
Buyse, Marion [Auteur]
Vigouroux, Corinne [Auteur]

Journal title :

Orphanet Journal of Rare Diseases

Abbreviated title :

Orphanet J Rare Dis

Volume number :

14

Pages :

177

Publisher :

BioMed Central

Publication date :

2019-07-12

ISSN :

1750-1172

English keyword(s) :

Lipodystrophic syndromes
Metreleptin therapy
Health self-perception
Adherence
Social interactions

HAL domain(s) :

Sciences du Vivant [q-bio]
Sciences du Vivant [q-bio]/Médecine humaine et pathologie/Endocrinologie et métabolisme
Sciences du Vivant [q-bio]/Sciences pharmaceutiques/Pharmacologie

English abstract : [en]

Background Although metreleptin replacement therapy was shown to improve metabolic alterations in lipodystrophic syndromes, patients’ adherence and satisfaction with treatment have never been evaluated. The 20 patients ...
Show more >Background Although metreleptin replacement therapy was shown to improve metabolic alterations in lipodystrophic syndromes, patients’ adherence and satisfaction with treatment have never been evaluated. The 20 patients with lipodystrophic syndromes participating in the French compassionate program of metreleptin therapy filled in a self-questionnaire including an Adherence Evaluation Test, the Treatment Satisfaction Questionnaire for Medication (TSQM®-vII), and items about physical appearance. Results 15 patients were women, median age was 32.5 years (IQT 25–75 (16.2;49.5), 18 had diabetes. Adherence with metreleptin (one daily subcutaneous injection) was poor in 25%, excellent in 25% and acceptable in 50% of patients. On a 0-to-100 scale, patients’ satisfaction scores reached 66.7 (52.1;81.2) for effectiveness, 55.6 (44.4;66.7) for ease/comfort of use, and 83.3 (52.1;83.3) for global satisfaction with metreleptin therapy. Self-reported side effects were frequent injection site reactions 100 (79.2;100). Satisfaction scores did not differ in patients with partial (n = 10) or generalized (n = 10) lipodystrophic syndromes, did not correlate with metabolic improvement, but were significantly higher in compliant patients with fewer side effects. Morphological appearance was reported improved under metreleptin therapy in 13 among 17 patients. Conclusions Metreleptin increases health self-perception and decreases morphotype-associated stigmatization in most patients with lipodystrophic syndromes, but poor comfort of use and local side effects weaken adherence.Show less >

Language :

Anglais

Audience :

Internationale

Popular science :

Non

Administrative institution(s) :

Université de Lille
Inserm
CHU Lille

Collections :

• Recherche translationnelle sur le diabète (RTD) - U1190

Submission date :

2024-01-19T23:49:41Z
2024-04-25T15:14:48Z