Mendiratta Meenal S, Yang Yaping, Balazs Andrea E, Willis Alecia S, Eng Christine M, Karaviti Lefkothea P, Potocki Lorraine
Endocrinology section-Department of Pediatrics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, USA.
Int J Pediatr Endocrinol. 2011;2011(1):5. doi: 10.1186/1687-9856-2011-5. Epub 2011 Jul 6.
Isolated hypocortisolism due to ACTH deficiency is a rare condition that can be caused by homozygous or compound heterozygous mutations in the gene encoding proopiomelanocortin (POMC). Loss of function mutations of POMC gene typically results in adrenal insufficiency, obesity and red hair. We describe an 18 month old Hispanic female with congenital adrenal insufficiency, a novel POMC mutation and atypical clinical features. The patient presented at the age of 9 months with hypoglycemia and the endocrine evaluation resulted in a diagnosis of ACTH deficiency. She developed extreme weight gain prompting sequence analysis of POMC, which revealed a homozygous c.231C > A change which is predicted to result in a premature termination codon. The case we report had obesity, hypocortisolism but lacked red hair which is typical for subjects with POMC mutations. Mutations of POMC should be considered in individuals with severe early onset obesity and adrenal insufficiency even when they lack the typical pigmentary phenotype.
由于促肾上腺皮质激素(ACTH)缺乏导致的孤立性皮质醇减少症是一种罕见疾病,可由编码阿黑皮素原(POMC)的基因中的纯合或复合杂合突变引起。POMC基因的功能丧失突变通常会导致肾上腺功能不全、肥胖和红发。我们描述了一名18个月大的西班牙裔女性,患有先天性肾上腺功能不全、一种新的POMC突变和非典型临床特征。该患者9个月大时因低血糖就诊,内分泌评估诊断为ACTH缺乏。她体重极度增加,促使对POMC进行序列分析,结果显示存在纯合的c.231C>A变化,预计会导致提前终止密码子。我们报告的这个病例有肥胖、皮质醇减少症,但没有POMC突变患者典型的红发。即使没有典型的色素沉着表型,对于严重早发性肥胖和肾上腺功能不全的个体也应考虑POMC突变。