Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2025 Apr;27(4):1815-1825.
doi: 10.1111/dom.16175. Epub 2025 Jan 20.

Safety and effectiveness in an uncontrolled setting of glucagon-like-peptide-1 receptor agonists in patients with familial partial lipodystrophy: Real-life experience from a national reference network

Sophie Lamothe  1 , Ines Belalem  1 , Marie-Christine Vantyghem  2 , Estelle Nobecourt  3 , Héléna Mosbah  4 , Sophie Béliard  5 , Brigitte Delemer  6 , Hippolyte Dupuis  2 , Paul Vandenbroere  2 , Nicolas Scheyer  7 , Chloé Amouyal  8 , Samy Hadjadj  9 , Sonja Janmaat  1   10 , Corinne Vigouroux  1   10 , Camille Vatier  1   10
Affiliations

Safety and effectiveness in an uncontrolled setting of glucagon-like-peptide-1 receptor agonists in patients with familial partial lipodystrophy: Real-life experience from a national reference network

Sophie Lamothe et al. Diabetes Obes Metab. 2025 Apr.

Abstract

Aim: To describe the effects of Glucagon-like peptide-1 receptor agonists (GLP-1RA) in patients with familial partial lipodystrophy (FPLD) assessed in a real-life setting in a national reference network.

Patients and methods: We retrospectively collected clinical and metabolic parameters in patients with FPLD in the French lipodystrophy reference network, who initiated GLP-1RA. Data were recorded before, at one-year (12 ± 6 months) and at the latest follow-up on GLP-1RA therapy (≥18 months).

Results: Seventy-six patients (89.4% of women), diagnosed with LMNA-related FPLD2 (n = 57), PPARG-related FPLD3 (n = 4), PLIN1-related FPLD4 (n = 5) or FPLD1 (n = 10) initiated GLP-1RA therapy between 2008 and 2024. Patients were aged a median (IQR) 48 years (34.5-57), body mass index (BMI) was 26.0 kg/m2 (23.9-29.5), HbA1c 8.3% (7.5-9.3), triglycerides 2.31 mmol/L (1.62-3.88). GLP-1RA were used in addition to previously used antidiabetics, 50% of patients being insulin-treated. After one year with GLP-1RA therapy, BMI, HbA1c and triglycerides significantly decreased to 25.6 kg/m2 (22.7-29.1), 7.3% (6.6-8.3) and 1.97 mmol/L (1.5-3.2) respectively (p < 0.001, p < 0.001 and p < 0.01, respectively), without significant changes in other antidiabetic and lipid-lowering drugs. Gamma-glutamyl-transferase and alanine-aminotransferase levels also significantly decreased. Effects on HbA1c, BMI and triglycerides persisted in the long term. One case of acute pancreatitis occurred during follow-up, associated with severe hypertriglyceridemia in a non-observant patient. Gastrointestinal symptoms affected 34% of patients, leading to GLP-1RA withdrawal in six patients.

Conclusion: GLP-1RA significantly improved BMI, HbA1c and triglycerides in a large majority of patients with FPLD. Larger and prospective controlled studies are warranted for identification of predictive factors and safety.

Keywords: GLP‐1 analogue; dyslipidaemia; fatty liver disease; glycaemic control; insulin resistance; real‐world evidence.

PubMed Disclaimer

Conflict of interest statement

H.M. served as speaker to Amryt Pharmaceuticals (now Chiesi Farmaceutici); C.Vi. to Amryt Pharmaceuticals, Sanofi, Ipsen and Lilly; C.Va. to Abbott, Advanz Pharma, Amryt Pharmaceuticals, AstraZeneca, Lilly, Novartis, Novo Nordisk, Sanofi; MCV to Amryt and Sanofi; and C.A to Lilly, Sanofi, Novo Nordisk, Decom. SH received institutional research grants from Zeneca, Asten Santé, Air Liquide Health Science, Bayer, Boehringer Ingelheim, Eli Lilly, Homeperf, Isis Diabète LVL, Nestle Home Care, NovoNordisk, Pierre Fabre, Sanofi, Valbiotis and Vitalaire and served as consultant and/or received honoraria from Astra Zeneca, Bayer, Boehringer Ingelheim, Eli Lilly, Mundipharma, NovoNordisk, Novartis, Sanofi, Servier and Valbiotis. SB has received honoraria for board, conferences, clinical trial or congress from Amarin, Amryt, Amgen, Akcea, Chiesi, Novartis, Sanofi and Ultragenyx.

Figures

FIGURE 1
FIGURE 1
Study flow diagram. Short term = 12 ± 6 months of GLP‐1RA therapy. Long term = last visit, more than 18 months of GLP‐1RA therapy.
FIGURE 2
FIGURE 2
Body mass index (BMI) (A), glycated haemoglobin (HbA1c) (B) and serum triglycerides (C) during GLP‐1RA treatment in patients with familial partial lipodystrophy (FPLD) at baseline, and after short‐ and long‐term follow‐up. NS, non‐significant. **p < 0.01, ***p < 0.001. Boxplots show median values, interquartile ranges (25th–75th percentile) with dots indicating outliers.
See this image and copyright information in PMC

References

    1. Brown RJ, Araujo‐Vilar D, Cheung PT, et al. The diagnosis and management of lipodystrophy syndromes: a multi‐society practice guideline. J Clin Endocrinol Metab. 2016;101(12):4500‐4511. - PMC - PubMed
    1. Garg A. Acquired and inherited lipodystrophies. N Engl J Med. 2004;350(12):1220‐1234. - PubMed
    1. Zammouri J, Vatier C, Capel E, et al. Molecular and cellular bases of lipodystrophy syndromes. Front Endocrinol (Lausanne). 2021;12:803189. - PMC - PubMed
    1. Fernandez‐Pombo A, Diaz‐Lopez EJ, Castro AI, et al. Clinical spectrum of LMNA‐associated type 2 familial partial lipodystrophy: a systematic review. Cells. 2023;12(5):725. - PMC - PubMed
    1. Guillín‐Amarelle C, Sánchez‐Iglesias S, Castro‐Pais A, et al. Type 1 familial partial lipodystrophy: understanding the Köbberling syndrome. Endocrine. 2016;54(2):411‐421. - PubMed

Substances

LinkOut - more resources