Endocrine Abstracts

Background: Prader–Willi syndrome (PWS) is a neurodevelopmental disorder characterised by hyperphagia, obesity, and increased risk of type 2 diabetes. Glucagon-like peptide-1 receptor agonists (GLP-1) are prescribed for weight management and glycaemic control. This study evaluated their efficacy in a PWS cohort.

Methods: We retrospectively reviewed outcomes of 28 patients with PWS (aged 17–33 years) attending a specialist PWS clinic between June 2023 – July 2025. Demographic, anthropometric, glycaemic (HbA1c), and treatment data were obtained from electronic health records. Patients were categorised by GLP-1 therapy (Saxenda, Ozempic, Mounjaro, Rybelsus) versus no GLP-1 use. Primary outcomes were changes in weight and BMI; secondary outcome was HbA1c change.

Results: Of 28 patients, 8 were excluded due to unavailable follow-up data, leaving 20 for analysis. At baseline, 5 patients were receiving GLP-1 therapy (80% diabetic [n = 4]) and 15 were not (46.67% diabetic [n = 7]). During ≤1 year of follow-up, the GLP-1 cohort experienced a mean weight gain of 2.38 ± 3.83 kg and BMI increase of 0.95 ± 1.22 kg/m² but achieved a substantial reduction in HbA1c 21.60 ± 30.54 mmol/mol. In contrast, the non-GLP-1 group gained 2.75 ± 9.09 kg with a BMI increase of 0.84 ± 3.70 kg/m², alongside a rise in HbA1c 1.08 ± 2.37 mmol/mol. Over the study period, 7 additional patients commenced GLP-1 therapy (Mounjaro), raising the treated group to 12 (60%). Extended follow-up data for 4 patients (all GLP-1) demonstrated mean weight reduction of 1.45 ± 2.63 kg and BMI reduction of 0.285 ± 0.76 kg/m².

Conclusion: In patients with PWS, GLP-1 receptor agonists were associated with significant HbA1c improvement but no short-term benefit in weight or BMI compared with non-GLP-1 patients. Uptake of GLP-1 therapy increased during follow-up, reflecting its growing role in clinical management. Longer-term studies are required for assessing metabolic and anthropometric effects in this population.