{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T08:03:44Z","timestamp":1791273824812,"version":"4.1.0"},"reference-count":10,"publisher":"American Academy of Pediatrics (AAP)","issue":"5","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2015,11,1]]},"abstract":"<jats:p>Cushing\u2019s syndrome (CS) in the pediatric population is challenging to diagnose and treat. Although next-generation medical therapies are emerging for adults with CS, none are currently approved or used in children. Here we describe the first use of mifepristone, a glucocorticoid receptor antagonist, to treat CS in a pediatric subject. The patient, a 14-year-old girl with an 18-month history of metastatic neuroendocrine carcinoma, suffered from fatigue, profound myopathy, irritability, and depression. She was found to have hypertension, hypokalemia, and worsening control of her preexisting type 1 diabetes. In this report, we detail our clinical evaluation that confirmed CS caused by an ectopic adrenocorticotropic hormone secreting tumor. Surgical and radiation therapies were not pursued because of her poor functional status and limited life expectancy, and medical treatment of CS was indicated for symptom relief. Mifepristone treatment provided rapid improvement in glycemic control, insulin resistance, and hypertension as well as significant diminishment of her myopathy and fatigue. Hypokalemia was managed with an oral potassium replacement and dose escalation of spironolactone; no other significant adverse effects were observed. Despite successful palliation of Cushing\u2019s signs and symptoms, the patient died of progression of her cancer. This case demonstrates the safety and efficacy of mifepristone treatment in a pediatric patient with symptomatic, ectopic CS. We conclude that, in appropriate pediatric patients with CS, glucocorticoid receptor antagonism with mifepristone should be considered to control the effects of hypercortisolism and to improve quality of life.<\/jats:p>","DOI":"10.1542\/peds.2015-0684","type":"journal-article","created":{"date-parts":[[2015,10,13]],"date-time":"2015-10-13T18:52:53Z","timestamp":1444762373000},"page":"e1377-e1381","source":"Crossref","is-referenced-by-count":10,"title":["Mifepristone Treatment of Cushing\u2019s Syndrome in a Pediatric Patient"],"prefix":"10.1542","volume":"136","author":[{"given":"Ronadip R.","family":"Banerjee","sequence":"first","affiliation":[{"name":"Departments of Medicine,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Neyssa","family":"Marina","sequence":"additional","affiliation":[{"name":"Pediatrics, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laurence","family":"Katznelson","sequence":"additional","affiliation":[{"name":"Departments of Medicine,"},{"name":"Neurosurgery, Stanford School of Medicine, Stanford, California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian J.","family":"Feldman","sequence":"additional","affiliation":[{"name":"Pediatrics, and"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"417","published-online":{"date-parts":[[2015,11,1]]},"reference":[{"issue":"4","key":"2021120906093739000_B1","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1016\/j.tem.2007.03.005","article-title":"Paediatric Cushing\u2019s syndrome: epidemiology, investigation and therapeutic advances.","volume":"18","author":"Storr","year":"2007","journal-title":"Trends Endocrinol Metab"},{"key":"2021120906093739000_B2","doi-asserted-by":"crossref","first-page":"185","DOI":"10.1016\/j.mce.2014.09.021","article-title":"Current approaches to the pharmacological management of Cushing\u2019s disease.","volume":"408","author":"Molitch","year":"2015","journal-title":"Mol Cell 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syndrome.","volume":"97","author":"Fleseriu","year":"2012","journal-title":"J Clin Endocrinol Metab"},{"issue":"4","key":"2021120906093739000_B9","doi-asserted-by":"crossref","first-page":"562","DOI":"10.1111\/cen.12332","article-title":"Global clinical response in Cushing\u2019s syndrome patients treated with mifepristone.","volume":"80","author":"Katznelson","year":"2014","journal-title":"Clin Endocrinol (Oxf)"},{"issue":"2","key":"2021120906093739000_B10","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1007\/s11102-015-0641-x","article-title":"Medical combination therapies in Cushing\u2019s 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