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. 2024 Apr 1;47(4):653-659.
doi: 10.2337/dc23-1614.

Efficacy and Safety of Glucagon-Like Peptide 1 Agonists in a Retrospective Study of Patients With Familial Partial Lipodystrophy

Maria C Foss-Freitas  1 , Salman Imam  1   2 , Adam Neidert  1 , Anabela Dill Gomes  1 , David T Broome  1 , Elif A Oral  1
Affiliations

Efficacy and Safety of Glucagon-Like Peptide 1 Agonists in a Retrospective Study of Patients With Familial Partial Lipodystrophy

Maria C Foss-Freitas et al. Diabetes Care. .

Abstract

Objective: Glucagon-like peptide 1 receptor agonists (GLP-1RA) are widely used for the management of diabetes mellitus (DM), but their efficacy in familial partial lipodystrophy (FPLD) is unknown. In this retrospective study, we evaluated the effect of GLP-1RA in patients with FPLD.

Research design and methods: We analyzed data, reported with SDs, from 14 patients with FPLD (aged 58 ± 12 years; 76.47% female) and 14 patients with type 2 DM (aged 58 ± 13 years; 71% female) before and 6 months after starting GLP-1RA.

Results: We observed reduction in weight (95 ± 23 to 91 ± 22 kg; P = 0.002), BMI (33 ± 6 to 31 ± 6 kg/m2; P = 0.001), HbA1c (8.2% ± 1.4% to 7.7% ± 1.4%; P = 0.02), and fasting glucose (186 ± 64 to 166 ± 53 mg/dL; P = 0.04) in patients with FPLD. The change in triglycerides after treatment was greater in the FPLD group compared with the DM group (P = 0.02). We noted acute pancreatitis in two case subjects with FPLD with longer therapy.

Conclusions: Our study demonstrates the relative safety and effectiveness of GLP-1RA in patients with FPLD.

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Conflict of interest statement

Duality of Interest. E.A.O. has received grant support from Aegerion Pharmaceuticals (manufacturer of metreleptin), Akcea Therapeutics, Ionis Pharmaceuticals, Regeneron Pharmaceuticals, Gemphire Therapeutics, Novo Nordisk, Rhythm Pharmaceuticals, Fractyl Health Laboratories, and GI Dynamics and has served as an advisor and/or a consultant to Aegerion Pharmaceuticals, Akcea Therapeutics, Ionis Pharmaceuticals, and Regeneron. E.A.O. has royalty rights from the use of metreleptin in lipodystrophy. D.T.B. receives grant funding from Novo Nordisk, Rhythm Pharmaceuticals, Inc., and Fractyl Health Laboratories, Inc., and serves as a consultant for Tayco, Inc. No other potential conflicts of interest relevant to this article were reported.

Figures

None
Graphical abstract
Figure 1
Figure 1
Comparison between changes in triglycerides (A) after 6 months of treatment with GLP-1RA agonist in patients with LD (n = 14) and common diabetes mellitus (DM) (n = 14). *P = 0.02. Differences in changes for fasting glucose (B), HbA1c (C), weight (D), and BMI (E) were not significant. PO, by mouth.
See this image and copyright information in PMC

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