ECE2023 Poster Presentations Diabetes, Obesity, Metabolism and Nutrition (159 abstracts)
Long-term Body Composition in Patients With POMC or LEPR Deficiency Obesity Following Setmelanotide
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, United States; 9Harvard Medical School, Boston, MA, United States; 10Institute for Experimental Pediatric Endocrinology, Charité − Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin und Humboldt−Universität zu Berlin, Berlin, Germany
Objective: Ideally, treatment strategies designed to reduce weight or body mass index in patients with obesity should reduce fat mass while preserving lean mass because reduced lean mass can negatively impact overall health and energy expenditure, thus favoring weight regain. In Phase 3 trials, 1 year of treatment with the melanocortin-4 receptor agonist setmelanotide led to weight reduction in patients with obesity due to biallelic variants in the genes encoding proopiomelanocortin (POMC)/proprotein convertase subtilisin/kexin type 1 (PCSK1; mean −25.6% change from baseline) or leptin receptor (LEPR; mean −12.5% change). The current analysis assesses changes in total fat and lean mass over 2 years in adult and pediatric patients who achieved beneficial weight loss at 1 year of treatment.
Methods: Patients with POMC, including PCSK1, or LEPR deficiency aged ≥6 years 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). Body weight measures and safety were assessed. Body composition was measured by dual-energy x-ray absorptiometry and reported as percent change in total fat and lean mass from index trial baseline in patients who achieved ≥10% weight reduction (≥18 years old) or ≥0.3-point body mass index Z score reduction (<18 years old) after 1 year of treatment.
Results: Across all patients (n=24), mean (standard deviation [SD]) percent changes in total fat mass were −37.2% (13.0%; n=22) at Month 12 and −32.4% (16.7%; n=17) at Month 24. Smaller mean (SD) percent changes were seen in lean mass at Month 12 (−7.8% [10.8%]) and 24 (−6.5% [16.2%]). No new safety issues were observed during long-term treatment.
Conclusions: Setmelanotide demonstrated clinical benefit in total fat mass reduction while achieving a relative maintenance of lean mass over 2 years of treatment.
Article tools
My recent searches
My recently viewed abstracts
Authors
Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
© Bioscientifica 2024 |
Privacy policy |
Cookie settings
BiosciAbstracts
Bioscientifica Abstracts is the gateway to a series of products that provide a permanent, citable record of abstracts for biomedical and life science conferences.
Find out more