{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T12:54:29Z","timestamp":1792068869741,"version":"4.4.0"},"reference-count":22,"publisher":"American Academy of Pediatrics (AAP)","issue":"6","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,6,1]]},"abstract":"<jats:p>ROHHAD (rapid-onset obesity with hypothalamic dysfunction, hypoventilation, and autonomic dysregulation) syndrome is a rare and complex pediatric condition marked by severe, early-onset hyperphagia and life-threatening obesity. For the past 2\u00a0years, we have been treating a boy, now aged 12\u00a0years, with ROHHAD syndrome. In light of the patient\u2019s progressive weight gain, intractable appetite, ventilator dependence, metabolic dysfunction-associated steatotic liver disease and behavioral dysregulation, including aggressive outbursts posing significant risk to self and others, we initiated off-label treatment with setmelanotide, a melanocortin-4 receptor (MC4R) agonist. Setmelanotide induced meaningful improvements, including substantial weight loss (28%, from 97 to 70\u00a0kg), measurable regression of hepatic steatosis as quantified by ultrasonography-based attenuation imaging, reduced ventilatory support, and a marked improvement in behavioral disorders that permitted the tapering of antipsychotic medication. After 18\u00a0months of therapy, insurance coverage for the off-label use was refused, and treatment was discontinued, which resulted in significant weight gain within 3\u00a0months (10%, from 70 to 77\u00a0kg). This treatment of hyperphagia-associated obesity in ROHHAD syndrome with setmelanotide suggests a potential pathophysiological origin in the MC4R pathway.<\/jats:p>","DOI":"10.1542\/peds.2025-074432","type":"journal-article","created":{"date-parts":[[2026,5,12]],"date-time":"2026-05-12T00:02:41Z","timestamp":1778544161000},"source":"Crossref","is-referenced-by-count":0,"title":["Melanocortin-4 Receptor Agonist Treatment of Hypothalamic Obesity in ROHHAD Syndrome"],"prefix":"10.1542","volume":"157","author":[{"given":"Anna","family":"Gr\u00fcnewald","sequence":"first","affiliation":[{"name":"1Department of Pediatrics, University Hospital of Essen, Essen, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emma","family":"Steidel","sequence":"additional","affiliation":[{"name":"1Department of Pediatrics, University Hospital of Essen, Essen, 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