EP1001 - ECE_3596 - Setmelanotide therapy in hypothalamic obesity following craniopharyngioma: a case series of three patients

Abstract

Hypothalamic obesity (HO) is a severe, often treatment-refractory complication after craniopharyngioma (CP) surgery. Setmelanotide, a melanocortin-4 receptor agonist (MC4R-A), offers a targeted therapeutic approach, but real-world data are limited. We performed a retrospective review of patients with post-CP HO treated with setmelanotide at our center over a period of 3-15 months, collecting clinical, metabolic, and treatment-related data. Three young adults with HO after surgery for CP were included. Treatment responses were variable: one patient discontinued setmelanotide after continued weight gain over 3 months, while two experienced significant reductions in weight (−14.4% and −25.9%), BMI (−11.9% and −25%), waist circumference (−4% and −20%) and triglycerides levels (−35% and −39%) over 7-15 months. Adverse effects as nausea, vomiting, headache and skin hyperpigmentation, were mild and non-limiting. One patient experienced sclera hyperpigmentation after 2 months of treatment. Setmelanotide may provide meaningful benefit for selected patients with severe HO following CP surgery. Despite small sample size, this series supports further investigation of MC4R agonism as a targeted therapy for acquired HO.