Division of Nephrology, Department of Pediatrics, All India Institute of Medical Sciences, Ansari Nagar, New Delhi, 110029, India.
CEN Case Rep. 2020 Aug;9(3):232-236. doi: 10.1007/s13730-020-00464-y. Epub 2020 Mar 17.
Identification of a monogenic etiology is possible in a proportion of patients with childhood-onset nephrolithiasis or nephrocalcinosis. Bartter syndrome (BS), a hereditary tubulopathy characterized by polyuria, hypokalemic alkalosis and growth retardation that rarely presents with isolated nephrocalcinosis. Patients with defect in renal outer medullary potassium channel, encoded by the KCNJ1 gene causing BS type 2, typically present during the neonatal period. We describe a 14-year-old girl with mild late-onset BS type 2 with reported pathogenic compound heterozygous variations in exon 2 of KCNJ1 (c.146G > A and c.657C > G). This patient presented with isolated medullary nephrocalcinosis due to hypercalciuria; absence of hypokalemia and metabolic alkalosis was unique. This case highlights the importance of screening the KCNJ1 gene in patients with hypercalciuria and nephrocalcinosis, even in older children.
在一部分儿童期起病的肾结石或肾钙质沉着症患者中,可能存在单基因病因。巴特综合征(Bartter syndrome,BS)是一种遗传性肾小管病,以多尿、低钾性碱中毒和生长迟缓为特征,很少表现为孤立性肾钙质沉着症。由 KCNJ1 基因编码的肾脏外髓质钾通道缺陷导致 BS 型 2,通常在新生儿期出现。我们描述了一名 14 岁女孩患有轻度晚发型 BS 型 2,该患者在 KCNJ1 基因的外显子 2中存在报道的致病性复合杂合变异(c.146G>A 和 c.657C>G)。该患者由于高钙尿症而出现孤立性髓质肾钙质沉着症;低钾血症和代谢性碱中毒的缺失是独特的。该病例强调了在高钙尿症和肾钙质沉着症患者中筛查 KCNJ1 基因的重要性,即使是在年龄较大的儿童中。