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
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
› Institutsangaben
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.
Publikationsverlauf
Artikel online veröffentlicht:
20. September 2024
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