Department of Paediatric Endocrinology, Valluvanad Hospital Complex Limited, Palakkad, Kerala, India.
West Midlands Deanery, Edgbaston, Birmingham, UK.
BMJ Case Rep. 2024 Sep 19;17(9):e261512. doi: 10.1136/bcr-2024-261512.
The first and rate-limiting step of steroid hormone biosynthesis is catalysed by mitochondrial cytochrome P450 side-chain cleavage enzyme (CYP11A1). CYP11A1 deficiency is commonly associated with adrenal insufficiency and, in 46 XY individuals, with variable degrees of differences in sex development (DSD). Here, we present a case report of a preadolescent male who presented to our emergency outpatient department in a state of decompensated shock necessitating ionotropic support. Further evaluation confirmed primary adrenal insufficiency. Subsequent clinical exome sequencing uncovered a compound heterozygous mutation in exons one and five of the CYP11A1 gene. This case highlights the varied presentation of CYP11A1 deficiency, showing that it can present as adrenal insufficiency without DSD.
甾体激素生物合成的第一步和限速步骤是由线粒体细胞色素 P450 侧链裂解酶(CYP11A1)催化的。CYP11A1 缺乏通常与肾上腺功能不全有关,在 46 XY 个体中,与性别发育(DSD)的不同程度差异有关。在这里,我们报告了一例青春期前男性的病例,他因失代偿性休克到我们的急诊门诊就诊,需要离子支持。进一步的评估证实了原发性肾上腺功能不全。随后的临床外显子组测序发现 CYP11A1 基因的第一和第五外显子存在复合杂合突变。这个病例突出了 CYP11A1 缺乏的不同表现,表明它可以表现为没有 DSD 的肾上腺功能不全。