The treatment of atypical hemolytic uremic syndrome with eculizumab in pediatric patients: a systematic review,Pediatric Nephrology

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The treatment of atypical hemolytic uremic syndrome with eculizumab in pediatric patients: a systematic review
Pediatric Nephrology ( IF 2.6 ) Pub Date : 2022-07-21 , DOI: 10.1007/s00467-022-05683-2
Raquel Medeiros de Souza ₁ , Bernardo Henrique Mendes Correa ₁ , Paulo Henrique Moreira Melo ₁ , Pedro Antunes Pousa ₁ , Tamires Sara Campos de Mendonça ₁ , Lucas Gustavo Castelar Rodrigues ₁ , Ana Cristina Simões E Silva ₁

Affiliation

Background

The atypical hemolytic uremic syndrome (aHUS) is a rare form of thrombotic microangiopathy associated with high morbidity and high mortality. Eculizumab, a humanized anti-C5 monoclonal antibody, was the first medication approved for treating aHUS in 2011.

Objective

The objective of this study is to evaluate the efficacy and safety of eculizumab treatment in pediatric patients with aHUS.

Data sources

We consulted PubMed, Scopus, SciELO, and Cochrane Library databases in July 2021. The descriptors were as follows: “Atypical Hemolytic Uremic Syndrome,” “aHUS,” “eculizumab,” “Pediatrics,” “Pediatric,” “Child,” “Children,” “Adolescent.”

Study eligibility criteria

The study eligibility criteria are as follows: clinical trials and observational studies that included pediatric patients with aHUS diagnosis and who were treated with eculizumab.

Participants and interventions

The participants are pediatric patients, up to 18 years old, with aHUS. The intervention was eculizumab treatment.

Study appraisal

For quality assessment, we used the Newcastle–Ottawa Scale, the National Institutes of Health (NIH) quality assessment tool for case series studies, and the Risk of Bias In Non-Randomized Studies of Interventions (ROBINS-I) tool.

Results

The initial search retrieved 433 studies, from which 15 were selected after complete assessment: 9 cohorts, 4 case series, and 1 clinical trial. The publication date ranged from 2015 to 2021. In total, 940 pediatric patients were included, and 682 received eculizumab. All studies reported improvements in renal and hematological parameters in most of the patients treated with eculizumab. The mortality rate was 1.6% for all patients treated with eculizumab.

Limitations

The number of studies is limited, and the included studies were methodologically heterogeneous. The studies were mostly observational and many had small sample sizes.

Conclusions

Eculizumab appears to be safe and effective for the treatment of aHUS in pediatric patients. More research is necessary to establish long-term efficacy, safety, and time of discontinuation.

Systematic review registration number

CRD42021266255.

中文翻译：

依库珠单抗治疗儿童非典型溶血性尿毒症综合征：系统评价

背景

非典型溶血性尿毒症综合征 (aHUS) 是一种罕见的血栓性微血管病，具有高发病率和高死亡率。Eculizumab 是一种人源化抗 C5 单克隆抗体，是 2011 年批准用于治疗 aHUS 的第一种药物。

客观的

本研究的目的是评估依库珠单抗治疗 aHUS 儿科患者的疗效和安全性。

数据源

我们在 2021 年 7 月查阅了 PubMed、Scopus、SciELO 和 Cochrane 图书馆数据库。描述符如下：“非典型溶血性尿毒症综合征”、“aHUS”、“eculizumab”、“儿科”、“儿科”、“儿童”、“儿童”，“青少年”。

研究资格标准

研究资格标准如下：临床试验和观察性研究包括诊断为 aHUS 并接受依库珠单抗治疗的儿科患者。

参与者和干预措施

参与者是 18 岁以下患有 aHUS 的儿科患者。干预措施是依库珠单抗治疗。

学习评估

对于质量评估，我们使用了纽卡斯尔-渥太华量表、美国国立卫生研究院 (NIH) 用于案例系列研究的质量评估工具，以及非随机干预研究中的偏倚风险 (ROBINS-I) 工具。

结果

初始搜索检索到 433 项研究，在完成评估后从中选择了 15 项：9 个队列、4 个病例系列和 1 个临床试验。发表日期从 2015 年到 2021 年不等。总共纳入了 940 名儿科患者，其中 682 名接受了依库珠单抗治疗。所有研究均报告了大多数接受依库珠单抗治疗的患者的肾脏和血液学参数有所改善。所有接受依库珠单抗治疗的患者的死亡率为 1.6%。