Hashemipour Mahin, Ghasemi Mahmoud, Hovsepian Silva
Department of Pediatrics, Child Growth and Development Research Center, Isfahan University of Medical Sciences, Isfahan, Iran.
Int J Prev Med. 2012 Jul;3(7):510-4.
Lipoid congenital adrenal hyperplasia (lipoid CAH), a rare disorder of steroid biosynthesis, is the most severe form of CAH. In this disorder the synthesis of glucocorticoids, mineralocorticoids and sex steroids is impaired which result in adrenal failure, severe salt wasting crisis and hyperpigmentation in phenotypical female infants irrespective of genetic sex. In this report, we presented a 28-day-old phenotypic female infant, which referred with lethargy, failure to thrive and electrolyte abnormalities. Considering the clinical and biochemical findings, lipoid CAH was diagnosed and replacement therapy with standard doses of glucocorticoid and mineralocorticoid and sodium chloride was initiated. During follow-up, she had good clinical condition, but at 6 years of age, she refers with hypertension and adrenal insufficiency because of arbitrary drug discontinuation by mother. In ultrasonography an abdominal mass (the testicles) was reported. Chromosome study showed 46XY pattern. Orchiectomy was performed. We recommended that in cases with clinical presentation of adrenal insufficiency if there is not the facility to determine the karyotype, repeated ultrasonography perform during follow-up. In addition, investigating the genetic bases of the disorder would help us to determine the pathogenesis of lipoid CAH in our community. It would be helpful in prenatal diagnosis and treatment of the disorder to prevent its related comorbidities.
类脂性先天性肾上腺皮质增生症(类脂性CAH)是一种罕见的类固醇生物合成障碍疾病,是先天性肾上腺皮质增生症最严重的形式。在这种疾病中,糖皮质激素、盐皮质激素和性激素的合成受损,导致肾上腺功能衰竭、严重的失盐危机以及表型为女性的婴儿出现色素沉着,无论其遗传性别如何。在本报告中,我们介绍了一名28日龄的表型为女性的婴儿,她因嗜睡、生长发育迟缓及电解质异常前来就诊。根据临床和生化检查结果,诊断为类脂性CAH,并开始用标准剂量的糖皮质激素、盐皮质激素和氯化钠进行替代治疗。在随访期间,她的临床状况良好,但6岁时,由于母亲擅自停药,她出现了高血压和肾上腺功能不全。超声检查报告发现腹部有一肿块(睾丸)。染色体研究显示为46XY核型。遂进行了睾丸切除术。我们建议,对于临床表现为肾上腺功能不全的病例,如果没有条件确定核型,在随访期间应反复进行超声检查。此外,研究该疾病的基因基础将有助于我们确定本社区类脂性CAH的发病机制。这将有助于该疾病的产前诊断和治疗,以预防其相关合并症。