Real-life experience on efficacy and safety of setmelanotide treatment in prepubertal children


Kahveci A., Kurt I., Turan S., Guran T., Bereket A., Haliloglu B.

European Journal of Endocrinology, cilt.192, sa.2, 2025 (SCI-Expanded, Scopus)

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 192 Sayı: 2
  • Basım Tarihi: 2025
  • Doi Numarası: 10.1093/ejendo/lvaf008
  • Dergi Adı: European Journal of Endocrinology
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, BIOSIS, EMBASE, MEDLINE
  • Anahtar Kelimeler: setmelanotide, prepubertal children, gonadotropin hormones, height velocity
  • Marmara Üniversitesi Adresli: Evet

Özet

Monogenic obesity, characterized by severe, early-onset obesity due to single-gene defects, often resists traditional weight management strategies. This report presents real-life experiences on the efficacy and safety of setmelanotide, an MC4R agonist, in 4 prepubertal children (ages 3-9) with LEPR and POMC deficiencies. Findings indicate that setmelanotide is effective at lower doses in our patients with POMC deficiency (0.3-0.5 mg/day) than the patients with LEPR deficiency (2.5 mg/day). Treatment was generally well-tolerated, with injection site reactions and hyperpigmentation as common side effects. As novel findings, gonadotropin-related effects such as hypothalamo-pituitary-gonadal axis activation and testicular descent were observed in 2 patients. Growth deceleration was noted in 2 children, and recovery from central hypothyroidism in 1 patient with POMC deficiency. Overall, setmelanotide appears to be effective and well-tolerated in young children with monogenic obesity. However, further studies are necessary to evaluate the long-term effects of early intervention on growth and pubertal development.