ECE2023 Poster Presentations Diabetes, Obesity, Metabolism and Nutrition (159 abstracts)
Exploration of Clinical Improvements Following Setmelanotide in Patients With Bardet-Biedl Syndrome
Andrea M. Haqq 1 , Wendy Chung 2 , Hélène Dollfus 3 , Anoop Iqbal 4 , Gabriel Á. Martos-Moreno 5 , Christine Poitou 6,7 , Jack A. Yanovski 8 , Sonali Malhotra 9,10,11 , Paul Miller 9 , Guojun Yuan 9 , Elizabeth Forsythe 12 , Karine Clement 6,7 & Jesús Argente 5,13
1University Of Alberta, Division of Pediatric Endocrinology, Edmonton, Canada; 2Columbia University, Division of Molecular Genetics, Department of Pediatrics, New York, NY, United States; 3Université de Strasbourg, Hôpitaux Universitaires de Strasbourg, CARGO and Department of Medical Genetics, Institut de Génétique Médicale d’Alsace, France; 4Marshfield Clinic Research Institute, Marshfield, WI, United States; 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; 6Pitié-Salpêtrière Hospital, Nutrition Department, Assistance Publique Hôpitaux de Paris, Paris, France; 7Sorbonne University, Inserm, Nutrition and Obesity, Systemic Approaches (NutriOmique) Research Group, Paris, France; 8National Institutes of Health, Section on Growth and Obesity, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, United States; 9Rhythm Pharmaceuticals, Inc., Boston, MA, United States; 10Massachusetts General Hospital, Boston, MA, United States; 11Harvard Medical School, Boston, MA, United States; 12University College London Great Ormond Street Institute of Child Health, Genetics and Genomic Medicine Programme, London, United Kingdom; 13IMDEA Food Institute, Madrid, Spain
Background: In patients with Bardet-Biedl syndrome (BBS), signaling impairments in the melanocortin-4 receptor (MC4R) pathway lead to hyperphagia and severe obesity, which negatively impact quality of life (QOL). We evaluated the impact of setmelanotide, an MC4R agonist, on age-appropriate weight-related parameters, hunger, and QOL in a Phase 3 trial of patients with BBS to further characterize clinical benefit in this patient population.
Methods: Patients ≥6 years old with BBS and obesity were enrolled into a Phase 3 trial (NCT03746522) and received 52 weeks of setmelanotide treatment. Hunger was assessed with an 11-point Likert scale in patients ≥12 years old without cognitive impairment. QOL was assessed in adults using the Impact of Weight on Quality of Life-Lite (IWQOL-Lite) and in pediatric patients using the Pediatric Quality of Life Inventory (PedsQL). Clinically meaningful improvements in weight-related parameters were defined as ≥5% reduction in body weight for adults and ≥0.2-point decrease in BMI Z score and/or ≥5–percentage point decrease in percent of the 95th BMI percentile for pediatric patients. Clinically meaningful improvement in hunger was defined as a ≥1-point within-patient reduction in hunger score. In adult and pediatric patients, patient-relevant improvements in QOL were defined as an increase in IWQOL-Lite score of 7.7-12 and increase in PedsQL score of >4.4, respectively. Stabilization of weight-related parameters was also assessed.
Results: In total, 28 of 32 patients (88%) had clinically meaningful or patient-relevant improvements that met thresholds in ≥1 measure at the last study visit. Three of 4 patients without improvement showed weight stabilization; thus, 31 of 32 patients (97%) experienced clinically meaningful and/or patient-relevant improvement or weight stabilization. Setmelanotide was generally well tolerated; 1 patient discontinued study drug during placebo treatment because of an adverse event.
Conclusions: Most patients with BBS showed clinically meaningful or patient-relevant improvement following setmelanotide, as measured by decreases in age-appropriate weight-related parameters, decreases in hunger, and overall improvements in QOL. Clinical benefit from antiobesity medications should be assessed beyond weight loss, especially in patients with BBS.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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