ECEESPE2025 Oral Communications Oral Communications 15: Metabolism, Nutrition and Obesity (6 abstracts)
Real-world setmelanotide changes in BMI in French patients with acquired hypothalamic obesity
Pauline Faucher 1 , Sarah Chalopin 1 , Frédérique Albarel 2 , Ahlam Azar-Kolakez 3 , Natacha Bouhours 4 , Jean-Claude Carel 3 , Régis Coutant 4 , Justine Cristante 5 , Nicolas Farigon 6 , Blandine Gatta-Cherifi 7 , Iva Gueorguieva 8 , Marie Hoflack 9 , Dunlanjalee Kariyawasam 10 , Marie Michelet 7 , Patricia Pigeon Kherchiche 11 , Véronique Savey 12 , Bérénice Ségrestin 13 , Caroline Storey 3 , Géraldine Vitellius 14 , Karine Clément 1 & Christine Poitou 1
1Assistance Publique Hôpitaux de Paris, Sorbonne Université, Service de Nutrition, Hôpital de la Pitié-Salpêtrière, Paris, France; 2Conception Hospital AP-HM, Endocrinology Department, Marseille, France; 3Robert-Debré Hospital APHP, Pediatric Endocrinology-Diabetology Department, Paris, France; 4University Hospital of Angers, Pediatric Endocrinology and Diabetology Unit, Angers, France; 5University Hospital of Grenoble, Nutrition and Endocrinology Department, Grenoble, France; 6University Hospital of Clermont-Ferrand, Clinical Nutrition Department, Clermont-Ferrand, France; 7University Hospital of Bordeaux, Department of Endocrinology, Diabetology and Nutrition, Bordeaux, France; 8Jeanne de Flandre Hospital, Pediatric Endocrinology Department, Lille, France; 9Lenval Foundation, Pediatric Endocrinology Department, Nice, France; 10Necker-Enfants Malades Hospital APHP, Pediatric Endocrinology, Diabetology and Gynecology Department, Paris, France; 11Felix Guyon Hospital, Pediatric Departmen, Saint-Denis, La Réunion, France; 12Caen-Normandy Hospital, Hepato-Gastroenterology and Nutrition Department, Caen, France; 13Lyon Sud Hospital, Endocrinology Department, Lyon, France; 14Robert-Debré University Hospital of Reims, Endocrinology, Diabetology and Nutrition Department, Reims, France
JOINT2287
Introduction: Acquired hypothalamic obesity (aHO) results from physical hypothalamic damage due to trauma, tumours, treatment-related injuries, or inflammation, which can disrupt the melanocortin-4 receptor (MC4R) signalling pathway. aHO is characterized by rapid and excessive weight gain following the damage. A 16-week Phase 2 open-label trial, treating aHO with the MC4R agonist setmelanotide produced consistent and clinically significant responses, maintained or improved over a 12-month follow-up period. This report presents real-world data for 25 patients in France with aHO treated with setmelanotide under pre-marketing early access authorization.
Methods: For patients with aHO in France under early access treatment with setmelanotide for a minimum of 3 months changes in BMI are reported.
Results: Twenty-five patients (14 females), including 9 children, aged 7-42 years old, were included. Seventeen patients had craniopharyngioma, 3 astrocytoma, and 1 each AQP4 antibody encephalitis, ganglioma, Langerhans cell histiocytosis, neuroglial tumour or viral inflammation affecting the pituitary region. Patients started treatment at 0.25 to 2 mg/day. Depending on the duration of treatment, treatment response, and adverse events, patients received 1 to 3 mg/day at last included visit. For all 25 patients, mean BMI (SD) decreased from 40.6 (8.7) kg/m2 to 36.2 (7.9) kg/m2 after 3-month treatment, indicating a 10.8% decrease from baseline. For patients with 6-month data (n=12), mean BMI decreased from 42.5 (7.9) kg/m2 to 37.6 (6.6) kg/m2 to 34.9 (5.7) kg/m2 after 3- and 6-month treatment, respectively, representing an 11.6% and 18.0% decrease from baseline. For patients with 9-month data (n=7), BMI decreased from 43.7 (7.9) kg/m2 to 38.8 (7.0) kg/m2 to 35.7 (6.0) kg/m2 to 34.5 (6.0) kg/m2 after 3-, 6- and 9-month treatment, respectively, indicating an 11.4%, 18.3% and 21.0% decrease from baseline. All patients except 1 had a lower BMI at each included time point. The patient with an increase in BMI, at Month 6, had surgery after 3 months due to disease progression and stopped treatment for 10 days. No new safety signals were observed.
Conclusions: This real-world evidence of 25 patients with aHO, including 3 with non-tumour-related causes, who received at least 3 months of setmelanotide under pre-marketing early access authorization in France showed consistent improvements in BMI in the majority of patients. Efficacy and safety outcomes are consistent with Phase 2 trial data that demonstrate beneficial outcomes of setmelanotide treatment in patients between 6 and 40 years of age with aHO.
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Endocrine Abstracts
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