Abstract | OBJECTIVE: METHODS: We present the patient's clinical, biochemical, and imaging findings. RESULTS: A 16-year-old girl presented with classic Cushing disease. After transsphenoidal surgery, Cyberknife radiosurgery, ketoconazole, and metyrapone did not control her disease, and she was prescribed mifepristone, which was titrated to a maximal dosage of 1200 mg daily with subsequent symptom improvement. Mifepristone ( RU486) is a high-affinity, nonselective antagonist of the glucocorticoid receptor. There is limited literature on its use as an off-label medication to treat refractory Cushing disease. Over her 8-year treatment with mifepristone, her therapy was complicated by hypertension and hypokalemia requiring spironolactone and potassium chloride. She received a 2-month drug holiday every 4 to 6 months to allow for withdrawal menstrual bleeding with medroxyprogesterone acetate. Urinary cortisol, serum cortisol, and corticotropin levels remained elevated during mifepristone drug holidays. While on mifepristone, her signs and symptoms of Cushing disease resolved. Repeated magnetic resonance imaging demonstrated stable appearance of the residual pituitary mass. Bilateral adrenalectomy was performed, and mifepristone was discontinued after 95 months of medical therapy. CONCLUSIONS:
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Authors | Marina Basina, Hau Liu, Andrew R Hoffman, David Feldman |
Journal | Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists
(Endocr Pract)
2012 Sep-Oct
Vol. 18
Issue 5
Pg. e114-20
ISSN: 1934-2403 [Electronic] United States |
PMID | 22441000
(Publication Type: Case Reports, Journal Article)
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Chemical References |
- Hormone Antagonists
- Mifepristone
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Topics |
- Adolescent
- Female
- Hormone Antagonists
(therapeutic use)
- Humans
- Mifepristone
(therapeutic use)
- Pituitary ACTH Hypersecretion
(drug therapy)
- Treatment Outcome
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