Melanocortin-4 receptor regulation of endocrine axes and clinical effects of setmelanotide
Authors' conclusion
Setmelanotide exerts pleiotropic effects beyond weight reduction, modulating multiple endocrine axes in BBS.
Study context
- Population
- Genetically confirmed Bardet-Biedl syndrome (BBS) patients initiating setmelanotide therapy
- Participants
- 58 participants
- Outcome
- Changes over time on setmelanotide
- Follow-up
- 6 months
Methodological detailssingle-centre, longitudinal, linear mixed-effects models adjusted for age, sex and BMI z-score
- Single-centre
- Longitudinal
- Linear mixed-effects models adjusted for age, sex and BMI z-score
Main finding
As reported in the abstract
- Significant reductions in BMI and HbA1c
- Increases in gonadotropins, and in testosterone and estradiol age-dependently
- IGF-1 increased independently of weight change
- TSH decreased without corresponding changes in peripheral thyroid hormones
- Effects established within the first 6 months and stable thereafter
Observational data: associations, not effects.
Original publication
Melanocortin-4 receptor regulation of endocrine axes and clinical effects of setmelanotide. J Clin Endocrinol Metab. Published 1 August 2026. doi:10. 1210/ clinem/ dgag293
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