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NPHS2 p.V290M 突变与迟发性激素抵抗性肾病综合征。

NPHS2 p.V290M mutation in late-onset steroid-resistant nephrotic syndrome.

机构信息

First Department of Pediatrics, Semmelweis University, Budapest, Bókay J. u. 53, Budapest, Hungary.

出版信息

Pediatr Nephrol. 2013 May;28(5):751-7. doi: 10.1007/s00467-012-2379-2. Epub 2012 Dec 14.

Abstract

BACKGROUND

The most frequently mutated gene of steroid-resistant nephrotic syndrome (SRNS) is NPHS2. Current guidelines propose the sequencing of all NPHS2 exons only in childhood-onset SRNS.

METHODS

A cohort of 38 Hungarian patients with childhood-onset nephrotic-range proteinuria was screened for NPHS2 mutations. The frequency of the p.V290M mutation in late-onset SRNS was examined in the French and PodoNet cohorts.

RESULTS

Of the 38 Hungarian patients screened, seven carried NPHS2 mutations on both alleles, of whom two-diagnosed with proteinuria through school screening programs at the age of 9.7 and 14 years, respectively-did not develop nephrotic syndrome in childhood. The first, an 18-year-old boy, homozygous for p.V290M, has never developed edema. The second, a 31-year-old woman-compound heterozygous for p.V290M and p.R138Q-was first detected with hypoalbuminemia (<30 g/l) and edema at the age of 24.3 and 27.5 years, respectively. Both patients currently have a normal glomerular filtration rate. The mutation p.V290M was carried by three of the 38 patients in the Hungarian cohort, by two of the 95 patients with late-onset SRNS in the PodoNet cohort and by none of the 83 patients in the French cohort.

CONCLUSIONS

We propose that not only the p.R229Q variant, but also the p.V290M mutation should be screened in Central and Eastern European patients with late-onset SRNS.

摘要

背景

激素耐药性肾病综合征(SRNS)最常发生突变的基因是 NPHS2。目前的指南建议仅在儿童期起病的 SRNS 中对所有 NPHS2 外显子进行测序。

方法

对 38 名匈牙利儿童期起病肾病范围蛋白尿患者进行 NPHS2 突变筛查。在法国和 PodoNet 队列中检查迟发性 SRNS 中 p.V290M 突变的频率。

结果

在筛查的 38 名匈牙利患者中,有 7 名患者两个等位基因均携带 NPHS2 突变,其中两名患者通过学校筛查计划在 9.7 岁和 14 岁时被诊断为蛋白尿-在儿童期未发展为肾病综合征。第一个是一个 18 岁的男孩,纯合子 p.V290M,从未出现水肿。第二位是一个 31 岁的女性,复合杂合子 p.V290M 和 p.R138Q-分别在 24.3 岁和 27.5 岁时首次被发现低白蛋白血症(<30 g/l)和水肿。这两位患者目前肾小球滤过率正常。突变 p.V290M 在匈牙利队列的 38 名患者中有 3 名携带,在 PodoNet 队列的 95 名迟发性 SRNS 患者中有 2 名携带,而在法国队列的 83 名患者中均未携带。

结论

我们建议,不仅应筛查 p.R229Q 变异体,还应筛查迟发性 SRNS 的中欧和东欧患者中的 p.V290M 突变。

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