抗肿瘤坏死因子治疗肾小球病:病例报告和文献综述。

Olcay Y Jones, Laura C Malone, Celina Brunson
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摘要

背景:肾小球病变是指由于多种原因导致肾小球滤过屏障受损,从而引起特发性肾病综合征(NS)。治疗方案有限,通常包括类固醇,但反应程度不一:病例介绍:一名 7 岁的男性患者在 2 岁时因呼吸道感染而患上了特发性肾病综合征,经检查发现其 COL4A4 和 TRPC6 基因存在意义不明的杂合子变异。活检结果包括荚膜细胞病变和基底膜变化。在对类固醇产生初步反应后,患者接受了一个疗程的阿纳金拉治疗,随后又接受了阿达木单抗治疗,作为节省类固醇的生物反应调节剂,持续时间超过两年。在逐渐减量后,患者的病情仍在缓解,在过去的12个月中没有接受过治疗:本病例表明,在肾小球疾病的出现过程中,环境诱因和遗传因素在生物学上决定了级联事件的复杂性。下调由肾小球微环境成分引起的体细胞组织驱动的促炎环境有助于从新出现的荚膜细胞病中恢复。在病程早期阻断肿瘤坏死因子-α,即使是暂时阻断,也能为新生再生过程留出时间。还需要进行更多的研究来验证这一假设,并尽量减少类固醇的使用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Anti-Tumor Necrosis Factor Treatment for Glomerulopathy: Case Report and Review of Literature.

Background: Glomerulopathy involves damage to the glomerular filtration barrier for several reasons, resulting in idiopathic nephrotic syndrome (NS). Treatment options are limited and often include steroids with varying levels of response.

Case presentation: A 7-year-old male with a history of NS at age 2 years that developed following a respiratory tract infection was found to have a heterozygous variant of uncertain significance in COL4A4 and TRPC6 genes. Biopsy findings included podocytopathy and changes in the basement membrane. Upon initial response to steroids, the patient was treated with a brief course of anakinra followed by adalimumab for > 2 years as steroid-sparing biological response modifiers. After a gradual taper, the patient remains in remission and has not received treatment in the last 12 months.

Conclusions: This case shows the complex nature of biologically predetermined cascading events in the emergence of glomerular disease with environmental triggers and genetic factors. Downregulation of somatic tissue-driven proinflammatory milieu originating from the constituents of the glomerular microenvironment can help in recovery from emerging podocytopathy. Blocking tumor necrosis factor-α early in the disease course, even temporarily, may allow time for the de novo regenerative process to prevail. Additional research is warranted to test this hypothesis and minimize steroid use.

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