依珠单抗治疗儿童致密沉积病1例报告及文献综述

Clinical Nephrology. Case Studies Pub Date : 2020-12-10 eCollection Date: 2020-01-01 DOI:10.5414/CNCS110309
Katsuaki Kasahara, Yoshimitsu Gotoh, Hisakazu Majima, Asami Takeda, Masashi Mizuno
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引用次数: 2

摘要

致密沉积病(DDD)是补体成分3 (C3)肾小球病(C3G)的一种亚型,是补体替代途径过度活跃导致膜攻击复合物形成的结果。DDD的治疗策略是有限的。我们报告一例13岁女孩在9岁时被诊断为DDD,伴有肾病和肾病综合征和C3肾病因子阴性替代补体途径激活。最初用强的松龙、甲基强的松龙脉冲(MPs)和米佐利滨治疗有效3年,之后复发。尽管在MP治疗后使用强的松龙和霉酚酸酯(MMF),她的肾功能和蛋白尿恶化,伴有高可溶性(s)C5b-9水平;同时出现呼吸困难和胸腔积液。第一次ECZ输注后3天,尿量增加,呼吸状况改善,PE消退,1个月内蛋白尿减少。血清肌酐水平下降,肾功能在7周内完全恢复正常。sC5b-9水平正常化,尽管蛋白尿减少,但在MMF治疗ECZ期间,肾病范围蛋白尿持续了53周,即使延长了治疗间隔。因此,补体激活途径靶向治疗可能对快速进展的DDD有用。我们的数据支持补体通路异常在C3G伴DDD中的作用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Eculizumab for pediatric dense deposit disease: A case report and literature review.

Eculizumab for pediatric dense deposit disease: A case report and literature review.

Eculizumab for pediatric dense deposit disease: A case report and literature review.

Dense deposit disease (DDD), a subtype of complement component 3 (C3) glomerulopathy (C3G), results from alternative complement pathway hyperactivity leading to membrane attack complex formation. DDD treatment strategies are limited. We report a case of a 13-year-old girl diagnosed with DDD at 9 years of age, with nephritic and nephrotic syndrome and C3 nephritic factor-negative alternative complement pathway activation. Initial treatment with prednisolone, methylprednisolone pulses (MPs), and mizoribines was effective for 3 years, after which she relapsed. Despite MP treatment followed by prednisolone and mycophenolate mofetil (MMF), her kidney function and proteinuria deteriorated with a high soluble (s)C5b-9 level; she also developed dyspnea and pleural effusion (PE). Three days after the first eculizumab (ECZ) infusion, urine volume increased, respiratory condition improved, PE resolved, and proteinuria decreased in 1 month. Serum creatinine level decreased, and kidney function completely normalized within 7 weeks. The sC5b-9 level normalized, and although proteinuria decreased, nephrotic range proteinuria persisted during ECZ treatment with MMF for 53 weeks, even with increased treatment interval. Thus, complement activation pathway-targeted therapy may be useful for rapidly progressing DDD. Our data support the role of complement pathway abnormalities in C3G with DDD.

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