New Maria I
Pediatric Endocrinology, The New York Presbyterian Hospital/Weill Medical College of Cornell University, 525 East 68th Street, M-630 New York, NY 10021, USA.
Mol Cell Endocrinol. 2003 Dec 15;211(1-2):75-83. doi: 10.1016/j.mce.2003.09.013.
Congenital adrenal hyperplasia (CAH) refers to a family of inherited disorders of adrenal steroidogenesis in which each disorder is characterized by a specific enzyme deficiency that impairs cortisol production by the adrenal cortex. The enzymes most commonly affected are 21-hydroxylase (21-OH), 11beta-hydroxylase, 3beta-hydroxysteroid dehydrogenase, and less often, 17alpha-hydroxylase/17,20-lyase and cholesterol desmolase. Many of the corresponding genes for the described enzymes have been isolated and characterized, and specific mutations causing CAH have been identified. In classical CAH (simple virilizing and salt wasting forms), androgen excess causes external genital ambiguity in newborn females and progressive postnatal virilization in both sexes. In nonclassical CAH, 21-OHD is partial and occurs with milder symptoms. A deficiency of 11beta-Hydroxylase deficiency results in ambiguous genitalia in the newborn genetic female and androgen excess and hypertension in both males and females. In 3beta-hydroxysteroid deficiency adrenal and gonadal androgen production is deficient resulting in incomplete genital development in genetic males and limited androgen affect in females. Two less frequent causes of CAH 17alpha-Hydroxylase/17,20-lyase and cholesterol desmolase result in external female genitalia in both sexes. Hormonal diagnosis is described for each disorder.
先天性肾上腺皮质增生症(CAH)是指肾上腺类固醇生成的一系列遗传性疾病,每种疾病的特征是特定酶缺乏,损害肾上腺皮质产生皮质醇。最常受影响的酶是21-羟化酶(21-OH)、11β-羟化酶、3β-羟类固醇脱氢酶,较少见的是17α-羟化酶/17,20-裂解酶和胆固醇侧链裂解酶。已分离并鉴定了许多上述酶的相应基因,并确定了导致CAH的特定突变。在典型的CAH(单纯男性化和失盐型)中,雄激素过多导致新生女婴外生殖器模糊,以及两性出生后渐进性男性化。在非典型CAH中,21-羟化酶缺乏是部分性的,症状较轻。11β-羟化酶缺乏导致新生女婴生殖器模糊,以及男女两性雄激素过多和高血压。在3β-羟类固醇缺乏症中,肾上腺和性腺雄激素生成不足,导致遗传男性生殖器发育不全,女性雄激素影响有限。CAH的另外两种较罕见病因,即17α-羟化酶/17,20-裂解酶和胆固醇侧链裂解酶缺乏,导致两性均出现女性外生殖器。描述了每种疾病的激素诊断方法。