Title :

Therapeutic indications and metabolic effects of metreleptin in patients with lipodystrophy syndromes: Real-life experience from a national reference network

Author(s) :

Mosbah, H [Auteur]
Assistance publique - Hôpitaux de Paris (AP-HP) [AP-HP]
Vantyghem, M [Auteur]
Recherche translationnelle sur le diabète - U 1190 [RTD]
Nobécourt, Estelle [Auteur]
Diabète athérothrombose et thérapies Réunion Océan Indien [DéTROI]
Andreelli, F [Auteur]
Nutrition et obésités: approches systémiques (UMR-S 1269) [Nutriomics]
Archambeaud, F [Auteur]
Hôpital Dupuytren [CHU Limoges]
Bismuth, E [Auteur]
AP-HP Hôpital universitaire Robert-Debré [Paris]
Briet, C [Auteur]
MitoVasc - Physiopathologie Cardiovasculaire et Mitochondriale [MITOVASC]
Cartigny, M [Auteur]
Centre Hospitalier Régional Universitaire [CHU Lille] [CHRU Lille]
Donadille, B [Auteur]
Centre d’Infection et d’Immunité de Lille - INSERM U 1019 - UMR 9017 - UMR 8204 [CIIL]
Daguenel, A [Auteur]
Assistance publique - Hôpitaux de Paris (AP-HP) [AP-HP]
Fichet, M [Auteur]
Centre Hospitalier Universitaire de Rennes [CHU Rennes] = Rennes University Hospital [Pontchaillou]
Gautier, J [Auteur]
Assistance publique - Hôpitaux de Paris (AP-HP) [AP-HP]
Janmaat, S [Auteur]
Assistance publique - Hôpitaux de Paris (AP-HP) [AP-HP]
Jéru, I [Auteur]
Assistance publique - Hôpitaux de Paris (AP-HP) [AP-HP]
Legagneur, C [Auteur]
Cardiovascular and Renal Clinical Trialists [Vandoeuvre-les-Nancy] [INI-CRCT]
Leguier, L [Auteur]
Centre Hospitalier Régional Universitaire [CHU Lille] [CHRU Lille]
Maitre, J [Auteur]
Centre Hospitalier Régional d'Orléans [CHRO]
Mongeois, E [Auteur]
Centre Hospitalier Régional d'Orléans [CHRO]
Poitou, C [Auteur]
Assistance publique - Hôpitaux de Paris (AP-HP) [AP-HP]
Renard, Eric [Auteur]
Institut National de la Santé et de la Recherche Médicale [INSERM]
Institut de Génomique Fonctionnelle [IGF]
Reznik, Y [Auteur]
Hôpital Côte de Nacre [CHU Caen]
Spiteri, A [Auteur]
Centre Hospitalier Universitaire [CHU Grenoble] [CHUGA]
Travert, F [Auteur]
Assistance publique - Hôpitaux de Paris (AP-HP) [AP-HP]
Vergès, B [Auteur]
Hôpital du Bocage
Zammouri, J [Auteur]
Centre de Recherche Saint-Antoine [CRSA]
Vigouroux, Corinne [Auteur]
Unité de Recherche sur les Maladies Cardiovasculaires, du Métabolisme et de la Nutrition = Research Unit on Cardiovascular and Metabolic Diseases [ICAN]
Centre de Recherche Saint-Antoine [CRSA]
Vatier, C [Auteur]
Centre de Recherche Saint-Antoine [CRSA]

Journal title :

Diabetes, Obesity and Metabolism

Pages :

1565-1577

Publisher :

Wiley

Publication date :

2022-08

ISSN :

1462-8902

HAL domain(s) :

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

English abstract : [en]

Aim: To describe baseline characteristics and follow-up data in patients with lipodystrophy syndromes treated with metreleptin in a national reference network, in a real-life setting.Patients and methods: Clinical and ...
Show more >Aim: To describe baseline characteristics and follow-up data in patients with lipodystrophy syndromes treated with metreleptin in a national reference network, in a real-life setting.Patients and methods: Clinical and metabolic data from patients receiving metreleptin in France were retrospectively collected, at baseline, at 1 year and at the latest follow-up during treatment.Results: Forty-seven patients with lipodystrophy including generalized lipodystrophy (GLD; n = 28) and partial lipodystrophy (PLD; n = 19) received metreleptin over the last decade. At baseline, the median (interquartile range [IQR]) patient age was 29.3 (16.6-47.6) years, body mass index was 23.8 (21.2-25.7) kg/m2 and serum leptin was 3.2 (1.0-4.9) ng/mL, 94% of patients had diabetes (66% insulin-treated), 53% had hypertension and 87% had dyslipidaemia. Metreleptin therapy, administered for a median (IQR) of 31.7 (14.2-76.0) months, was ongoing in 77% of patients at the latest follow-up. In patients with GLD, glycated haemoglobin (HbA1c) and fasting triglyceride levels significantly decreased from baseline to 1 year of metreleptin treatment, from 8.4 (6.5-9.9)% [68 (48-85) mmol/mol] to 6.8 (5.6-7.4)% [51(38-57) mmol/mol], and 3.6 (1.7-8.5) mmol/L to 2.2 (1.1-3.7) mmol/L, respectively (P < 0.001), with sustained efficacy thereafter. In patients with PLD, HbA1c was not significantly modified (7.7 [7.1-9.1]% [61 (54-76) mmol/mol] at baseline vs. 7.7 [7.4-9.5]% [61(57-80) mmol/mol] at 1 year), and the decrease in fasting triglycerides (from 3.3 [1.9-9.9] mmol/L to 2.5 [1.6-5.3] mmol/L; P < 0.01) was not confirmed at the latest assessment (5.2 [2.2-11.3] mmol/L). However, among PLD patients, at 1 year, 61% were responders regarding glucose homeostasis, with lower baseline leptin levels compared to nonresponders, and 61% were responders regarding triglyceridaemia. Liver enzymes significantly decreased only in the GLD group.Conclusions: In this real-life setting study, metabolic outcomes are improved by metreleptin therapy in patients with GLD. The therapeutic indication for metreleptin needs to be clarified in patients with PLD.Show less >

Language :

Anglais

Popular science :

Non

ANR Project :

Institut de Cardiologie-Métabolisme-Nutrition

Collections :

• Centre d'Infection et d'Immunité de Lille (CIIL) - U1019 - UMR 9017

Source :

Harvested from HAL