Background: 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. Methods: We performed a retrospective review of patients with post-CP HO treated with setmelanotide at our center, collecting clinical, metabolic, and treatment-related data. Results: Three young adults with HO after surgery for CP were included. Treatment responses were variable: one patient discontinued setmelanotide after continued weight gain, while two experienced reductions in weight, BMI, waist circumference, triglycerides and insulin levels over 6–9 months. Adverse effects were mild and non-limiting. Conclusion: Setmelanotide may provide meaningful benefit for selected patients with severe, treatment-refractory HO after CP. Despite small sample size, this series supports further investigation of MC4R agonism as a targeted therapy for acquired HO.

Safety and effectiveness of setmelanotide in young patients with severe obesity due to craniopharyngioma: a real world monocentric experience

Angileri F.;
2026-01-01

Abstract

Background: 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. Methods: We performed a retrospective review of patients with post-CP HO treated with setmelanotide at our center, collecting clinical, metabolic, and treatment-related data. Results: Three young adults with HO after surgery for CP were included. Treatment responses were variable: one patient discontinued setmelanotide after continued weight gain, while two experienced reductions in weight, BMI, waist circumference, triglycerides and insulin levels over 6–9 months. Adverse effects were mild and non-limiting. Conclusion: Setmelanotide may provide meaningful benefit for selected patients with severe, treatment-refractory HO after CP. Despite small sample size, this series supports further investigation of MC4R agonism as a targeted therapy for acquired HO.
2026
Craniopharyngioma
Hypothalamic obesity
Setmelanotide
Weight outcomes
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11699/110365
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