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Background <p>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.</p> Methods <p>We performed a retrospective review of patients with post-CP HO treated with setmelanotide at our center, collecting clinical, metabolic, and treatment-related data.</p> Results <p>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.</p> Conclusion <p>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.</p>

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

  • 期刊论文

  • 发表日期：

    2026年6月22日

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  • Oana Ruxandra Cotta,
  • Cesare Morgante,
  • Annalisa Giandalia,
  • Flavio Angileri,
  • Francesco Ferrau’,
  • Salvatore Cannavo’

摘要

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.