Witchel Selma Feldman
Division of Pediatric Endocrinology, Department of Pediatrics, Children's Hospital of Pittsburgh of UPMC, University of Pittsburgh, Pittsburgh, Pennsylvania.
J Pediatr Adolesc Gynecol. 2017 Oct;30(5):520-534. doi: 10.1016/j.jpag.2017.04.001. Epub 2017 Apr 24.
The congenital adrenal hyperplasias comprise a family of autosomal recessive disorders that disrupt adrenal steroidogenesis. The most common form is due to 21-hydroxylase deficiency associated with mutations in the 21-hydroxylase gene, which is located at chromosome 6p21. The clinical features associated with each disorder of adrenal steroidogenesis represent a clinical spectrum that reflect the consequences of the specific mutations. Treatment goals include normal linear growth velocity and "on-time" puberty in affected children. For adolescent and adult women, treatment goals include regularization of menses, prevention of progression of hirsutism, and preservation of fertility. For adolescent and adult men, prevention and early treatment of testicular adrenal rest tumors is beneficial. In this article key aspects regarding pathophysiology, diagnosis, and treatment of congenital adrenal hyperplasia are reviewed.
先天性肾上腺增生症是一组常染色体隐性疾病,会干扰肾上腺类固醇生成。最常见的形式是由于21-羟化酶缺乏,这与位于6号染色体p21区域的21-羟化酶基因突变有关。每种肾上腺类固醇生成障碍相关的临床特征代表了一个临床谱,反映了特定突变的后果。治疗目标包括使患病儿童的线性生长速度正常以及“按时”进入青春期。对于青少年和成年女性,治疗目标包括月经规律、预防多毛症进展以及保留生育能力。对于青少年和成年男性,预防和早期治疗睾丸肾上腺残余肿瘤是有益的。本文对先天性肾上腺增生症的病理生理学、诊断和治疗的关键方面进行了综述。