313-OR: The Efficacy and Safety of Glucagon-Like-Peptide-1 Agonists in Patients with Familial Partial Lipodystrophy

Abstract

Familial partial lipodystrophy (FPLD) syndromes are a rare group of conditions with abnormal fat distribution and deposition of fat in ectopic tissues such as liver and muscle, leading to challenging metabolic abnormalities. Glucagon-like peptide-1 receptor agonists (GLP1R-A) are used for management of other metabolic dysfunction, but their efficacy in FPLD syndromes is not known. In this retrospective EMR review, we evaluate the effect of GLP1R-A for DM management in patients with FPLD for whom there are no approved treatments in the US. We collected data from 17 patients with FPLD (age 58 ± 12; 76.47 % females) and 17 patients with type 2 DM (age 58 ± 13; 71% females) before and 6 months after starting GLP1R-A. Only cases where the sole change was the addition of GLP1R-A were included in the analyses. Reduction in weight (p=0.002), BMI (p=0.001), HbA1c (p=0.02), and fasting glucose (p=0.04) (Figure 1A-D) were observed in patients with FPLD. Only the change in triglycerides after six months of treatment was greater in patients with FPLD compared to patients with DM (p=0.02, 95%CI: 13.6 to 215.8) (Figure 1E). Overall, there were no differences in changes in other parameters, and no serious events were reported. However, we observed acute pancreatitis in two cases with FPLD with longer therapy. Our study demonstrates the relative safety and effectiveness of GLP1R-A in patients with FPLD for the first time. Disclosure S.K.Imam: None. M.C.Foss-freitas: None. R.Meral: None. E.A.Oral: Advisory Panel; Amryt Pharma Plc, Regeneron, Rejuvenate Bio, Third Rock Ventures, Consultant; Amryt Pharma Plc, Ionis Pharmaceuticals, Regeneron, Other Relationship; Amryt Pharma Plc, Research Support; Amryt Pharma Plc, Novo Nordisk, Fractyl Health, Inc., Ionis Pharmaceuticals, Regeneron, GI Dynamics, Rejuvenate Bio. A.Dill gomes: None. F.Grissi: None.