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患有皮尔逊综合征的儿童肾移植

Kidney transplantation in a child with Pierson syndrome.

作者信息

Guler Sanem, Cimen Sertac, Acott Phillip, Whelan Kathy, Molinari Michele

机构信息

Department of Surgery, QEII HSC, Dalhousie University, Halifax, NS, Canada.

Department of Pediatrics, IWK Health Centre, Dalhousie University, Halifax, NS, Canada.

出版信息

Pediatr Transplant. 2017 Dec;21(8). doi: 10.1111/petr.13076. Epub 2017 Nov 1.

Abstract

Congenital nephrotic syndrome is commonly associated with mutations in genes that encode podocyte and slit diaphragm proteins or the structural and regulatory proteins of the GBM. These mutations lead to the formation of dysfunctional proteins, which account for the resistance of the renal manifestations to conventional treatment methods. Consequently, patients become renal replacement therapy dependent. Mutation of the LAMB2 gene is associated with Pierson syndrome, which is an autosomal recessive disorder characterized by congenital nephrotic syndrome and ocular abnormalities. In this report, a 2-year-old male patient who was diagnosed with Pierson syndrome is presented. He had bilateral microcoria and developmental delay in addition to nephrotic syndrome. His renal function deteriorated rapidly, and he underwent a deceased donor kidney transplantation. He showed dramatic improvement after kidney transplantation; in addition to having good renal function, he started to catch up to his peers in terms of growth. Pierson syndrome should be considered during the diagnostic investigations of children with renal manifestations and ocular abnormalities. Children with Pierson syndrome must be evaluated in terms of kidney transplantation as soon as they are diagnosed.

摘要

先天性肾病综合征通常与编码足细胞和裂孔隔膜蛋白或肾小球基底膜(GBM)的结构和调节蛋白的基因突变有关。这些突变导致功能失调的蛋白质形成,这解释了肾脏表现对传统治疗方法的抵抗性。因此,患者依赖肾脏替代治疗。LAMB2基因突变与皮尔逊综合征相关,这是一种常染色体隐性疾病,其特征为先天性肾病综合征和眼部异常。在本报告中,介绍了一名被诊断为皮尔逊综合征的2岁男性患者。除肾病综合征外,他还有双侧小瞳孔和发育迟缓。他的肾功能迅速恶化,接受了已故供体肾移植。肾移植后他显示出显著改善;除肾功能良好外,他在生长方面开始赶上同龄人。在对有肾脏表现和眼部异常的儿童进行诊断检查时应考虑皮尔逊综合征。一旦诊断出患有皮尔逊综合征的儿童,必须尽快对其进行肾脏移植评估。

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