Adipositas - Ursachen, Folgeerkrankungen, Therapie 2024; 18(03): 141-142
DOI: 10.1055/s-0044-1788822
Abstracts
Freitag, 18.10.24 | 09:00–10:30 Uhr
Neue Erkenntnisse aus der translationalen Adpositasforschung

4-Year Setmelanotide Weight Outcomes of Patients with POMC and LEPR Deficiency Obesity

Authors

  • M. Wabitsch

    1   University of Ulm, Division of Pediatric Endocrinology and Diabetes, Department of Pediatrics and Adolescent Medicine, Ulm

  • W. Chung

    2   Columbia University, Division of Molecular Genetics, Department of Pediatrics, New York

  • J. M. Swain

    3   Honor Health Research Institute, Scottsdale

  • P. Kühnen

    4   Charité – Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin und Humboldt-Universität zu Berlin, Institute for Experimental Pediatric Endocrinology, Berlin

  • E.V. D. Akker

    5   Erasmus University Medical Center, Division of Pediatric Endocrinology, Department of Pediatrics, Sophia Children’s Hospital and Obesity Center CGG, Rotterdam

  • J. Garrison

    6   Rhythm Pharmaceuticals, Inc., Boston

  • G. Yuan

    6   Rhythm Pharmaceuticals, Inc., Boston

  • J. Argente

    7   Instituto de Salud Carlos III, Department of Pediatrics and Pediatric Endocrinology, Universidad Autónoma de Madrid, University Hospital Niño Jesús, CIBER “Fisiopatología de la obesidad y nutrición” (CIBEROBN), Madrid
    10   IMDEA Food Institute, Madrid

  • K. Clément

    8   Pitié-Salpêtrière Hospital, Assistance Publique Hôpitaux de Paris, Nutrition Department, Paris
    11   Sorbonne University, Nutrition and Obesity, Systemic Approaches (NutriOmique) Research Group, Paris

  • S. Farooqi

    9   University of Cambridge, Wellcome-MRC Institute of Metabolic Science and NIHR Cambridge Biomedical Research Centre, Cambridge
Further Information
Also available at
 

Einleitung Proopiomelanocortin (POMC; including variants in PCSK1) and leptin receptor (LEPR) deficiency are associated with hyperphagia and early-onset, severe obesity. Setmelanotide treatment in this population improved weight-related measures and hunger severity and was well tolerated. We report long-term extension (LTE) outcomes after 4 years of setmelanotide treatment.

Methoden Patients with POMC or LEPR deficiency who achieved clinical benefit and acceptable safety in a prior trial of setmelanotide could enroll in the LTE (NTC03651765) and continue setmelanotide for ≥5 years.

Ergebnisse Twelve patients had 4 years of on-treatment measurements and were included in this analysis. Compared with index trial baseline, the mean (SD) change in body weight was −32.6 kg (36.7) for patients aged ≥18 years (n=8) and −42.7 (22.44) percentage points in %BMI95 for patients aged<18 years (n=4). No new safety signals were observed between the index trial and the LTE.

Schlussfolgerung Continuous setmelanotide treatment in patients with POMC or LEPR deficiency who achieved a clinical benefit at 1 year is supported by sustained meaningful benefit in weight-related measures with no new safety signals at 4 years of treatment in this population.



Publication History

Article published online:
20 September 2024

© 2024. Thieme. All rights reserved.

Georg Thieme Verlag KG
Rüdigerstraße 14, 70469 Stuttgart, Germany