Efficacy and Safety of GLP-1 Receptor Agonists in Familial Partial Lipodystrophy
abstract
This abstract is available on the publisher's site.
Access this abstract nowOBJECTIVE
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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Additional Info
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Efficacy and Safety of Glucagon-Like Peptide 1 Agonists in a Retrospective Study of Patients With Familial Partial Lipodystrophy
Diabetes Care 2024 Apr 01;47(4)653-659, MC Foss-Freitas, S Imam, A Neidert, AD Gomes, DT Broome, EA OralFrom MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.
Familial partial lipodystrophy (FPLD) is a rare genetic disorder characterized by the lack of subcutaneous adipose tissue in certain regions of the body with or without accumulation of adipose tissue in other areas. Common features of FPLD include type 2 diabetes, hypertriglyceridemia, hepatic steatosis, coronary artery disease, and insulin resistance. The cardiometabolic benefits of GLP-1 receptor agonists (GLP-1 RA) in type 2 diabetes and obesity make them an attractive therapeutic strategy for patients with FPLD, particularly given the limited treatment options available for this disorder. However, dedicated data on the efficacy and safety of GLP-1 RA in patients with FPLD are extremely limited, which may curtail their use.
This retrospective study examines outcomes in 14 patients with FPLD and 14 age- and sex-matched patients with type 2 diabetes alone who were treated clinically with GLP-1 RA over 6 months. Patients with FPLD experienced significant declines in weight (−4.0 ± 4.3%) and HbA1c (−0.5% ± 0.7%) as well as a nonsignificant decline in triglycerides (−16.7 ± 53.3%) over the treatment period. Reductions in weight and HbA1c on GLP-1 RA were comparable between patients with FPLD and those with type 2 diabetes, whereas reduction in triglycerides was greater in those with FPLD. Although there were no cases of gallstones or pancreatitis in the first 6 months of treatment within either group, two patients with FPLD developed acute pancreatitis with continued use of GLP-1 RA during the subsequent 12 months. Both individuals had a history of pancreatitis.
These data indicate that GLP-1 RA may be effective to reduce weight and improve glycemic control in individuals with FPLD. Of note, nearly all patients with FPLD in this study had type 1 FPLD, which is characterized by central fat accumulation that may be more likely to benefit from weight loss than other FPLD subtypes. Further studies involving larger sample sizes are needed to ensure the safety of GLP-1 RA in individuals with FPLD, particularly with regard to pancreatitis. Nonetheless, the findings from this study begin to address an important gap in knowledge and suggest that GLP-1 RA may hold promise for carefully selected patients with FPLD.