Proopiomelanocortin Deficiency Treated with a Melanocortin-4 Receptor Agonist - PubMed (original) (raw)
Clinical Trial
. 2016 Jul 21;375(3):240-6.
doi: 10.1056/NEJMoa1512693.
Affiliations
- PMID: 27468060
- DOI: 10.1056/NEJMoa1512693
Free article
Clinical Trial
Proopiomelanocortin Deficiency Treated with a Melanocortin-4 Receptor Agonist
Peter Kühnen et al. N Engl J Med. 2016.
Free article
Abstract
Patients with rare defects in the gene encoding proopiomelanocortin (POMC) have extreme early-onset obesity, hyperphagia, hypopigmentation, and hypocortisolism, resulting from the lack of the proopiomelanocortin-derived peptides melanocyte-stimulating hormone and corticotropin. In such patients, adrenal insufficiency must be treated with hydrocortisone early in life. No effective pharmacologic treatments have been available for the hyperphagia and obesity that characterize the condition. In this investigator-initiated, open-label study, two patients with proopiomelanocortin deficiency were treated with setmelanotide, a new melanocortin-4 receptor agonist. The patients had a sustainable reduction in hunger and substantial weight loss (51.0 kg after 42 weeks in Patient 1 and 20.5 kg after 12 weeks in Patient 2).
Comment in
- Hormone-Replacement Therapy for Melanocyte-Stimulating Hormone Deficiency.
Reitman ML. Reitman ML. N Engl J Med. 2016 Jul 21;375(3):278-9. doi: 10.1056/NEJMe1606919. N Engl J Med. 2016. PMID: 27468065 No abstract available. - Neuroendocrinology: New hormone treatment for obesity caused by POMC-deficiency.
Low MJ. Low MJ. Nat Rev Endocrinol. 2016 Nov;12(11):627-628. doi: 10.1038/nrendo.2016.156. Epub 2016 Sep 23. Nat Rev Endocrinol. 2016. PMID: 27658726 No abstract available.
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