Abstract | Context: Objective: Methods: This analysis included data from 2 pivotal single-arm, open-label, Phase 3 trials (NCT02896192, NCT03287960). These were multicenter trials. Patients had obesity due to POMC/PCSK1 or LEPR deficiency. During the trial, patients were treated with setmelanotide. Historical data on measured weight and height were obtained during screening. Results: A total of 17 patients ( POMC, n = 8; PCSK1, n = 1; LEPR, n = 8) with historical weight and height data were included in this analysis. Before setmelanotide treatment, patients with obesity due to POMC/PCSK1 or LEPR deficiency were above the 95th percentile for weight throughout childhood, demonstrated continuous weight gain, and did not show long-term weight loss upon interventions (eg, diet, surgery, exercise). Setmelanotide treatment attenuated weight and body mass index trajectories over the observation period of 1 year. Conclusion: In patients with POMC, PCSK1, or LEPR deficiency, traditional interventions for weight loss had limited impact on the trajectory of severe early-onset obesity. However, setmelanotide treatment attenuated weight and body mass index trajectories and led to weight loss associated with health benefits in most individuals.
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Authors | Martin Wabitsch, Sadaf Farooqi, Christa E Flück, Natasa Bratina, Usha G Mallya, Murray Stewart, Jill Garrison, Erica van den Akker, Peter Kühnen |
Journal | Journal of the Endocrine Society
(J Endocr Soc)
Vol. 6
Issue 6
Pg. bvac057
(Jun 01 2022)
ISSN: 2472-1972 [Electronic] United States |
PMID | 35528826
(Publication Type: Journal Article)
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Copyright | © The Author(s) 2022. Published by Oxford University Press on behalf of the Endocrine Society. |