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Early signs of glucocorticoid excess include increased appetite, weight gain, and growth arrest without a concomitant delay in bone age. Chronic glucocorticoid excess in children results in typical Cushingoid facies, but the “buffalo hump” and centripetal distribution of body fat that are characteristic of adult Cushing disease are seen only in long standing disease. Mineralocorticoid excess is characterized by hypertension, but patients receiving very low sodium diets (eg, the newborn) will not be hypertensive, as mineralocorticoids increase blood pressure primarily by retaining sodium and thus increasing intravascular volume. Moderate hypersecretion of adrenal androgens is characterized by mild signs of virilization; substantial hypersecretion of adrenal androgens is characterized by accelerated growth, increased bone age, increased muscle mass, acne, hirsutism, and deepening of the voice.

Cushing syndrome describes any form of glucocorticoid excess; Cushing disease designates hypercortisolism due to pituitary overproduction of corticotropin (ACTH). The related disorder caused by ACTH of nonpituitary origin is called the ectopic ACTH syndrome. Other causes of Cushing syndrome include adrenal adenoma, adrenal carcinoma, and multinodular adrenal hyperplasia. These disorders are distinct from iatrogenic Cushing syndrome, which is the clinical constellation resulting from administration of supraphysiologic quantities of ACTH or glucocorticoids.

Epidemiology and Associated Disorders

In adults and children over 7 years of age, the most common cause of Cushing syndrome is true Cushing disease (adrenal hyperplasia due to hypersecretion of pituitary ACTH).1 About 25% of patients referred for Cushing disease are children; thus, pediatric Cushing disease is more common than generally recognized. Many patients first seen as adults actually experience the onset of symptoms in childhood or adolescence. Boys are more frequently affected than girls in the prepubertal period, although the sex ratios are equal during adolescence, and women have a higher incidence of Cushing disease in adulthood.2Adrenal tumors, especially adrenal carcinomas, cause most cases of Cushing syndrome in children less than 7 years old3-21 (eTable 535.1). These tumors are more common in girls22 and may be associated with bodily hemihypertrophy as part of the Beckwith-Wiedemann syndrome, or with germline mutations or loss-of-heterozygocity of the tumor suppressor gene p53 as in the Li-Fraumeni syndrome.23-25 The ectopic ACTH syndrome is commonly seen in adults with oat cell carcinoma of the lung, carcinoid tumors, pancreatic islet cell carcinoma, and thymoma. Ectopic ACTH syndrome is rare in children; associated tumors include neuroblastoma, pheochromocytoma, and islet cell carcinoma of the pancreas.20ACTH independent multinodular adrenal hyperplasia, also called primary pigmented adrenocorticoid disease (PPNAD), is a rare entity characterized by hypersecretion of cortisol and adrenal androgens.26-29 It is seen in all age groups, more frequently in females. It is usually seen as part of the “Carney complex” (a form of multiple endocrine neoplasia), consisting of pigmented lentigines and blue nevi on the face, lips, and conjunctiva, and a variety of tumors including schwannomas and atrial myxomas, and, occasionally, GH-secreting pituitary adenomas, Leydig cell tumors, calcifying Sertoli ...

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