Queensland Fertility Group, Virtus Genetics, Brisbane, Australia.
Department of Nephrology, The Queensland Children's Hospital, Brisbane, Australia.
Pediatr Nephrol. 2022 Oct;37(10):2369-2374. doi: 10.1007/s00467-022-05421-8. Epub 2022 Feb 24.
Intronic WT1 mutations are usually causative of Frasier syndrome with focal segmental glomerulosclerosis as the characteristic nephropathy. Membranoproliferative glomerulonephritis is not commonly associated with disorders of sex development but has been recently identified as a WT1-associated nephropathy, but usually in cases of exonic mutations in either isolated Wilms tumor or Denys-Drash syndrome.
The clinical and genetic data from 3 individuals are reported.
This report describes the kidney manifestations in 3 individuals from 2 unrelated families with Frasier syndrome intronic WT1 mutations, noting that 2 of the 3 individuals have histologically confirmed membranoproliferative glomerulonephritis.
These case reports support expansion of the clinical spectrum of the kidney phenotypes associated with Frasier syndrome providing evidence of an association between WT1 mutation and an immune complex-related membranoproliferative glomerulonephritis. A higher resolution version of the Graphical abstract is available as Supplementary information.
内含子 WT1 突变通常是 Fraser 综合征的病因,其特征性肾病为局灶节段性肾小球硬化。膜增殖性肾小球肾炎通常与性发育障碍无关,但最近已被确定为一种与 WT1 相关的肾病,但通常见于孤立性肾母细胞瘤或 Denys-Drash 综合征中存在外显子突变的病例。
报告了 3 名个体的临床和遗传数据。
本报告描述了来自 2 个无关家系的 3 名 Fraser 综合征内含子 WT1 突变个体的肾脏表现,注意到 3 名个体中有 2 名的肾脏组织学检查证实为膜增殖性肾小球肾炎。
这些病例报告支持 Fraser 综合征相关肾脏表型的临床谱扩展,为 WT1 突变与免疫复合物相关膜增殖性肾小球肾炎之间的关联提供了证据。更清晰的图表版本可以作为补充信息获取。