Department of Pathology and Laboratory Medicine, Perelman School of Medicine at The University of Pennsylvania, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Pediatr Dev Pathol. 2024 Sep-Oct;27(5):426-458. doi: 10.1177/10935266241242669. Epub 2024 May 14.
The etiologies of podocyte dysfunction that lead to pediatric nephrotic syndrome (NS) are vast and vary with age at presentation. The discovery of numerous novel genetic podocytopathies and the evolution of diagnostic technologies has transformed the investigation of steroid-resistant NS while simultaneously promoting the replacement of traditional morphology-based disease classifications with a mechanistic approach. Podocytopathies associated with primary and secondary steroid-resistant NS manifest as diffuse mesangial sclerosis, minimal change disease, focal segmental glomerulosclerosis, and collapsing glomerulopathy. Molecular testing, once an ancillary option, has become a vital component of the clinical investigation and when paired with kidney biopsy findings, provides data that can optimize treatment and prognosis. This review focuses on the causes including selected monogenic defects, clinical phenotypes, histopathologic findings, and age-appropriate differential diagnoses of nephrotic syndrome in the pediatric population with an emphasis on podocytopathies.
导致小儿肾病综合征(NS)的足细胞功能障碍的病因广泛,且随发病年龄而变化。大量新的遗传足细胞病的发现和诊断技术的发展改变了对类固醇耐药性 NS 的研究,同时促进了用基于机制的方法替代传统的基于形态的疾病分类。与原发性和继发性类固醇耐药性 NS 相关的足细胞病表现为弥漫性系膜硬化、微小病变病、局灶节段性肾小球硬化和塌陷性肾小球病。分子检测曾经是一种辅助选择,现在已成为临床研究的重要组成部分,当与肾活检结果相结合时,可提供可优化治疗和预后的数据。本文重点介绍儿科人群肾病综合征的病因,包括选定的单基因缺陷、临床表型、组织病理学发现以及与足细胞病相关的年龄适宜的鉴别诊断。