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因17-α-羟化酶/17,20-裂解酶缺乏导致的先天性肾上腺增生症伴高血压和假两性畸形:阿曼首例病例报告

Congenital Adrenal Hyperplasia due to 17-alpha-hydoxylase/17,20-lyase Deficiency Presenting with Hypertension and Pseudohermaphroditism: First Case Report from Oman.

作者信息

Mula-Abed Waad-Allah S, Pambinezhuth Fathima B, Al-Kindi Manal K, Al-Busaidi Noor B, Al-Muslahi Hilal N, Al-Lamki Mohammad A

机构信息

Department of Chemical Pathology, Royal Hospital, P.O. Box 1331, Seeb 111, Muscat, Sultanate of Oman.

Centre, Royal Hospital, P.O. Box 1331, Seeb 111, Muscat, Sultanate of Oman.

出版信息

Oman Med J. 2014 Jan;29(1):55-9. doi: 10.5001/omj.2014.12.

Abstract

This is the first report of congenital adrenal hyperplasia (CAH) due to combined 17α-hydroxylase/17,20 lyase deficiency in an Omani patient who was initially treated for many years as a case of hypertension. CAH is an uncommon disorder that results from a defect in steroid hormones biosynthesis in the adrenal cortex. The clinical presentation depends on the site of enzymatic mutations and the types of accumulated steroid precursors. A 22-year-old woman who was diagnosed to have hypertension since the age of 10 years who was treated with anti-hypertensive therapy was referred to the National Diabetes and Endocrine Centre, Royal Hospital, Oman. The patient also had primary amenorrhea and features of sexual infantilism. Full laboratory and radio-imaging investigations were done. Adrenal steroids, pituitary function and karyotyping study were performed and the diagnosis was confirmed by molecular mutation study. Laboratory investigations revealed adrenal steroids and pituitary hormones profile in addition to 46XY karyotype that are consistent with the diagnosis of CAH due to 17α-hydroxylase deficiency. Extensive laboratory workup revealed low levels of serum cortisol (and its precursors 17α-hydroxyprogesterone and 11-deoxycortisol), adrenal androgens (dehydroepiandrosterone sulfate and androstenedione), and estrogen (estradiol); and high levels of mineralocorticoids precursors (11-deoxycorticosterone and corticosterone) with high levels of ACTH, FSH and LH. Mutation analysis revealed CYP17A1-homozygous mutation (c.287G>A p.Arg96Gln) resulting in the complete absence of 17α-hydroxylase/17,20-lyase activity. The patient was treated with dexamethasone and ethinyl estradiol with cessation of anti-hypertensive therapy. A review of the literature was conducted to identify previous studies related to this subtype of CAH. This is the first biochemically and genetically proven case of CAH due to 17α-hydroxylase/17,20-lyase deficiency in Oman and in the Arab World described in the literature.

摘要

这是关于阿曼一名先天性肾上腺皮质增生症(CAH)患者的首例报告,该患者因17α-羟化酶/17,20裂解酶联合缺乏症,最初多年被当作高血压病例治疗。CAH是一种罕见疾病,由肾上腺皮质类固醇激素生物合成缺陷引起。临床表现取决于酶突变位点和累积的类固醇前体类型。一名自10岁起被诊断为高血压并接受抗高血压治疗的22岁女性,被转诊至阿曼皇家医院国家糖尿病与内分泌中心。该患者还存在原发性闭经和性幼稚特征。进行了全面的实验室和影像学检查。检测了肾上腺类固醇、垂体功能并进行了核型分析研究,分子突变研究确诊了病情。实验室检查显示,除了46XY核型外,肾上腺类固醇和垂体激素谱与17α-羟化酶缺乏所致CAH的诊断相符。广泛的实验室检查发现血清皮质醇(及其前体17α-羟孕酮和11-脱氧皮质醇)、肾上腺雄激素(硫酸脱氢表雄酮和雄烯二酮)和雌激素(雌二醇)水平较低;盐皮质激素前体(11-脱氧皮质酮和皮质酮)水平较高,同时促肾上腺皮质激素、促卵泡激素和促黄体生成素水平也较高。突变分析显示CYP17A1纯合突变(c.287G>A p.Arg96Gln),导致17α-羟化酶/17,20-裂解酶活性完全缺失。该患者接受了地塞米松和乙炔雌二醇治疗,停止了抗高血压治疗。对文献进行了回顾,以确定此前与这种CAH亚型相关的研究。这是阿曼及文献中描述的阿拉伯世界首例经生化和基因证实的17α-羟化酶/17,20-裂解酶缺乏所致的CAH病例。

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