一例罕见的与PRKACA重复基因相关的儿童期原发性色素结节性肾上腺皮质疾病。

JCEM case reports Pub Date : 2025-03-10 eCollection Date: 2025-03-01 DOI:10.1210/jcemcr/luaf035
Padala Ravi Kumar, Bandana Dash, Deepak Kumar Dash, Debasish Patro, Jatin Kumar Majhi, Bhabani Sankar Dhal
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摘要

原发性色素结节性肾上腺皮质病(PPNAD)是促肾上腺皮质激素(ACTH)不依赖库欣综合征(CS)的一种罕见但重要的病因。它通常表现为周期性CS在年轻人。儿童发病的PPNAD极为罕见。约90%的PPNAD病例与卡尼复合体(CNC)有关。PPNAD和CNC都与PRKAR1A基因的多种致病变异有关,PRKAR1A基因编码蛋白激酶A (PKA)的调节亚基1型α。编码PKA催化亚基α的PRKACA基因的致病性变异在PPNAD中极为罕见。我们报告一例8岁零3个月的女童,她表现出提示CS的特征,包括肥胖、身材矮小、高血压、月亮相、痤疮和面部过多,但没有经典的条纹或CNC的迹象。激素评估证实acth非依赖性CS。然而,腹部影像学显示肾上腺形态正常。遗传分析在19p染色体上发现了PRKACA基因的重复,这与PPNAD有关。患者行双侧腹腔镜肾上腺切除术,组织病理学检查证实PPNAD的诊断。术后随访显示库欣样特征消退,高血压消失。据我们所知,这是首例报道的PRKACA重复的女童因PPNAD而表现为CS的病例。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
A Rare Case of PRKACA Duplication-Associated Childhood-Onset Primary Pigmented Nodular Adrenocortical Disease.

Primary pigmented nodular adrenocortical disease (PPNAD) is a rare but important cause of adrenocorticotropic hormone (ACTH)-independent Cushing syndrome (CS). It usually presents as cyclical CS in young adults. Childhood onset of PPNAD is exceedingly rare. About 90% of cases of PPNAD are associated with Carney complex (CNC). Both PPNAD and CNC are linked to diverse pathogenic variants of the PRKAR1A gene, which encodes the regulatory subunit type 1 alpha of protein kinase A (PKA). Pathogenic variants of PRKACA gene, which encodes the catalytic subunit alpha of PKA, are extremely rare in PPNAD. We report a case of a female child, aged 8 years and 3 months, who presented with features suggestive of CS, including obesity, short stature, hypertension, moon facies, acne, and facial plethora but without classical striae or signs of CNC. Hormonal evaluation confirmed ACTH-independent CS. However, abdominal imaging revealed normal adrenal morphology. Genetic analysis identified a duplication of the PRKACA gene on chromosome 19p, which is linked to PPNAD. The patient underwent bilateral laparoscopic adrenalectomy, and histopathological study confirmed the PPNAD diagnosis. Postoperative follow-up showed resolution of cushingoid features and hypertension. To our knowledge, this is the first reported case of a female child with PRKACA duplication presenting as CS due to PPNAD.

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