罕见的先天性肾上腺增生症。

IF 3 3区 医学 Q2 ENDOCRINOLOGY & METABOLISM
Busra Gurpinar Tosun, Tulay Guran
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引用次数: 0

摘要

先天性肾上腺皮质增生症(CAH)是一组常染色体隐性遗传疾病,是由于参与肾上腺类固醇生成的酶和辅助因子的编码基因发生了致病变异。虽然 21-羟化酶、11β-羟化酶、3β-羟类固醇脱氢酶 2 型、17α-羟化酶/17,20-裂解酶、P450 氧化还原酶、类固醇生成急性调节蛋白、胆固醇侧链裂解酶缺乏症都属于 CAH 的定义范围,但 "CAH "一词通常指 "21-羟化酶缺乏症(21OHD)",因为在大多数人群中,21OHD 约占 CAH 的 95%。罕见形式的 CAH 的发病率因种族和地理位置而异。在大多数情况下,类固醇生成受阻的生化指纹可确定 CAH 的特定亚型,通常需要进行基因检测才能确诊。尽管在临床特征和管理方面存在显著差异,但有关罕见 CAH 形式的大多数数据都是从《21 世纪营养不良杂志》中推断出来的。本综述文章旨在整理有关罕见 CAH 诊断和治疗的现有数据。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Rare forms of congenital adrenal hyperplasia.

Congenital adrenal hyperplasia (CAH) is a group of autosomal recessive disorders due to pathogenic variants in genes encoding enzymes and cofactors involved in adrenal steroidogenesis. Although 21-hydroxylase, 11β-hydroxylase, 3β-hydroxysteroid dehydrogenase type 2, 17α-hydroxylase/17,20-lyase, P450 oxidoreductase, steroidogenic acute regulatory protein, cholesterol side-chain cleavage enzyme deficiencies are considered within the definition of CAH, the term 'CAH' is often used to refer to '21-hydroxylase deficiency (21OHD)' since 21OHD accounts for approximately 95% of CAH in most populations. The prevalence of the rare forms of CAH varies according to ethnicity and geographical location. In most cases, the biochemical fingerprint of impaired steroidogenesis points to the specific subtypes of CAH, and genetic testing is usually required to confirm the diagnosis. Despite there are significant variations in clinical characteristics and management, most data about the rare CAH forms are extrapolated from 21OHD. This review article aims to collate the currently available data about the diagnosis and the management of rare forms of CAH.

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来源期刊
Clinical Endocrinology
Clinical Endocrinology 医学-内分泌学与代谢
CiteScore
6.40
自引率
3.10%
发文量
192
审稿时长
1 months
期刊介绍: Clinical Endocrinology publishes papers and reviews which focus on the clinical aspects of endocrinology, including the clinical application of molecular endocrinology. It does not publish papers relating directly to diabetes care and clinical management. It features reviews, original papers, commentaries, correspondence and Clinical Questions. Clinical Endocrinology is essential reading not only for those engaged in endocrinological research but also for those involved primarily in clinical practice.
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