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利妥昔单抗用于治疗儿童肾病综合征的棘手病例。

Rituximab for troublesome cases of childhood nephrotic syndrome.

作者信息

Safdar Osama Y, Aboualhameael Adila, Kari Jameela A

机构信息

Osama Y Safdar, Adila Aboualhameael, Jameela A Kari, Department of Pediatrics, King Abdulaziz University Hospital, Jeddah 21589, Saudi Arabia.

出版信息

World J Clin Pediatr. 2014 Nov 8;3(4):69-75. doi: 10.5409/wjcp.v3.i4.69.

Abstract

Nephrotic syndrome (NS) is the most common glomerular disease of childhood. Steroid-dependent and steroid-resistant nephrotic syndrome present challenges in their pharmaceutical management; patients may need several immunosuppressive medication for optimum control, each of which medication has its own safety profile. Rituximab (RTX) is a monoclonal antibody that targets B cells and has been used successfully for management of lymphoma and rheumatoid arthritis. Recent clinical studies showed that rituximab may be an efficacious and safe alternative for the treatment of complicated nephrotic syndrome. In this review article, we aim to review the efficacy and safety of RTX therapy in nephrotic syndrome. We reviewed the literature pertaining to this topic by searching for relevant studies on PubMed and Medline using specific keywords. The initial search yielded 452 articles. These articles were then examined to ensure their relevance to the topic of research. We focused on multicenter randomized controlled trials with relatively large numbers of patients. A total of 29 articles were finally identified and will be summarized in this review. The majority of clinical studies of RTX in complicated pediatric NS showed that rituximab is effective in approximately 80% of patients with steroid-dependent NS, as it decreases the number of relapses and steroid dosage. However, RTX is less effective at achieving remission in steroid-resistant NS. RTX use was generally safe, and most side effects were transient and infusion-related. More randomized, double-blinded clinical studies are needed to assess the role of RTX in children with nephrotic syndrome.

摘要

肾病综合征(NS)是儿童最常见的肾小球疾病。激素依赖型和激素抵抗型肾病综合征在药物治疗方面存在挑战;患者可能需要几种免疫抑制药物来实现最佳控制,每种药物都有其自身的安全性。利妥昔单抗(RTX)是一种靶向B细胞的单克隆抗体,已成功用于治疗淋巴瘤和类风湿性关节炎。最近的临床研究表明,利妥昔单抗可能是治疗复杂性肾病综合征的一种有效且安全的替代药物。在这篇综述文章中,我们旨在回顾利妥昔单抗治疗肾病综合征的疗效和安全性。我们通过使用特定关键词在PubMed和Medline上搜索相关研究,对与该主题相关的文献进行了综述。初步搜索得到452篇文章。然后对这些文章进行审查,以确保它们与研究主题相关。我们重点关注了患者数量相对较多的多中心随机对照试验。最终确定了29篇文章,并将在本综述中进行总结。大多数关于利妥昔单抗治疗复杂性儿童肾病综合征的临床研究表明,利妥昔单抗对大约80%的激素依赖型肾病综合征患者有效,因为它减少了复发次数和激素用量。然而,利妥昔单抗在激素抵抗型肾病综合征中实现缓解的效果较差。使用利妥昔单抗总体上是安全的,大多数副作用是短暂的且与输液相关。需要更多的随机、双盲临床研究来评估利妥昔单抗在儿童肾病综合征中的作用。

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本文引用的文献

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Rituximab in children with resistant idiopathic nephrotic syndrome.利妥昔单抗治疗耐药性特发性肾病综合征患儿。
J Am Soc Nephrol. 2012 Jun;23(6):1117-24. doi: 10.1681/ASN.2011080775. Epub 2012 May 10.

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