Matsukura Hiro, Fuchizawa Tatsuya, Ohtsuki Akio, Higashiyama Hiroyuki, Higuchi Osamu, Higuchi Akira, Miyawaki Toshio
Department of Pediatrics, Saiseikai Toyama Hospital, 33-1 Kusunoki, 931-8533, Toyama, Japan.
Pediatr Nephrol. 2003 Mar;18(3):297-300. doi: 10.1007/s00467-002-1042-8. Epub 2003 Feb 7.
We describe an 8-year-old boy who presented with steroid-resistant nephrotic syndrome (SRNS) associated with X-linked ichthyosis (XLI). At birth, the patient exhibited scaly skin, cryptorchidism, and steroid sulfatase (STS) deficiency. DNA analysis showed deletion of exons 1-10 of the STS gene. Proteinuria developed at 6 years and was resistant to steroid therapy. Kidney biopsy findings prior to steroid therapy were compatible with minimal change nephrotic syndrome. By immunofluorescence, glomerular basement membranes exhibited diffuse linear staining for the alpha5 chain of collagen IV, making X-linked Alport syndrome an unlikely explanation for the association of SRNS and ichthyosis. Despite immunosuppressive therapy together with oral prednisolone, no clinical response was achieved. He rapidly reached end-stage renal failure and finally underwent renal transplantation. We propose that SRNS should be considered as one of the highly variable phenotypes associated with XLI.
我们描述了一名8岁男孩,他患有与X连锁鱼鳞病(XLI)相关的类固醇抵抗性肾病综合征(SRNS)。出生时,该患者表现出皮肤鳞屑、隐睾症和类固醇硫酸酯酶(STS)缺乏。DNA分析显示STS基因外显子1 - 10缺失。蛋白尿在6岁时出现,对类固醇治疗耐药。类固醇治疗前的肾活检结果与微小病变肾病综合征相符。通过免疫荧光检查,肾小球基底膜对IV型胶原α5链呈弥漫性线性染色,这使得X连锁Alport综合征不太可能解释SRNS与鱼鳞病的关联。尽管联合口服泼尼松龙进行免疫抑制治疗,但未取得临床缓解。他迅速发展为终末期肾衰竭,最终接受了肾移植。我们提出,SRNS应被视为与XLI相关的高度可变表型之一。