ECE2023 Poster Presentations Diabetes, Obesity, Metabolism and Nutrition (159 abstracts)
Weight Outcomes With Setmelanotide Over 3 Years in Patients With POMC or LEPR Deficiency Obesity
Karine Clement 1,2 , Martin Wabitsch 3 , Erica van den Akker 4 , Jesús Argente 5,6 , Andrea Navarria 7 , Madhura Srinivasan 7 , Guojun Yuan 7 , Sonali Malhotra 7,8,9 & Peter Kühnen 10
1Pitié−Salpêtrière Hospital, Assistance Publique Hôpitaux de Paris, Nutrition Department, Paris, France; 2Sorbonne Université, Inserm, NutriOmics Research Unit, Paris, France; 3University of Ulm, Division of Pediatric Endocrinology and Diabetes, Center for Rare Endocrine Diseases, Department of Pediatrics and Adolescent Medicine, Ulm, Germany; 4Sophia Children’s Hospital and Obesity Center CGG, Erasmus University Medical Center, Division of Pediatric Endocrinology, Department of Pediatrics, Rotterdam, Netherlands; 5Universidad Autónoma de Madrid, University Hospital Niño Jesús, Department of Pediatrics and Pediatric Endocrinology, CIBER “Fisiopatología de la obesidad y nutrición” (CIBEROBN), Instituto de Salud Carlos III, Madrid, Spain; 6IMDEA Food Institute, Madrid, Spain; 7Rhythm Pharmaceuticals, Inc., Boston, MA, United States; 8Massachusetts General Hospital, Boston, MA,; 9Harvard Medical School, Boston, MA,; 10Charité − Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin und Humboldt−Universität zu Berlin, Institute for Experimental Pediatric Endocrinology, Berlin, Germany
Background: Rare variants in the melanocortin-4 receptor (MC4R) pathway are associated with early-onset, severe obesity and hyperphagia. Setmelanotide, an MC4R agonist, reduced body mass index (BMI) and decreased hunger in patients with obesity due to biallelic variants in genes encoding proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) in Phase 3 trials. The current analysis assesses the durability of setmelanotide efficacy over 3 years in the subgroup of patients who achieved clinically beneficial weight loss.
Methods: Patients aged ≥6 years with POMC, including PCSK1, or LEPR deficiency obesity who demonstrated clinical benefit and acceptable safety after treatment with setmelanotide in a prior (index) trial continued treatment in a long-term extension trial (NCT03651765). Age-appropriate weight measures and safety were assessed. Changes in weight-related measures from index trial baseline were evaluated in patients who achieved ≥10% weight reduction (age ≥18 years) or ≥0.3-point BMI Z score reduction (age <18 years) after 1 year of treatment.
Results: Across all patients (n=24), mean (SD) percent changes in BMI were −24.8% (8.2%; n=24), −21.0% (13.0%; n=23), and −24.0% (17.9%; n=15) at 12, 24, and 36 months, respectively. In patients ≥18 years old (n=11), mean (SD) percent changes in weight were −25.1% (7.7%; n=11), −22.9% (12.5%; n=11), and −24.4% (13.2%; n=8) at 12, 24, and 36 months, respectively. In patients <18 years old (n=13), mean (SD) reductions in BMI Z score were observed at Month 12 (−1.31 [0.66]; n=13), 24 (−1.10 [0.79]; n=11), and 36 (−1.01 [1.22]; n=4). No new safety issues were observed during long-term treatment.
Conclusions: Patients who achieved ≥10% body weight or ≥0.3-point BMI Z score reduction at 1 year demonstrated persistent and clinically significant benefit at 3 years, supporting long-term use of setmelanotide in adult and pediatric patients with obesity due to POMC and LEPR deficiency.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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