de Souza Raquel Medeiros, Correa Bernardo Henrique Mendes, Melo Paulo Henrique Moreira, Pousa Pedro Antunes, de Mendonça Tamires Sara Campos, Rodrigues Lucas Gustavo Castelar, Simões E Silva Ana Cristina
Interdisciplinary Laboratory of Medical Investigation, Unit of Pediatric Nephrology, Faculty of Medicine, Federal University of Minas Gerais (UFMG), Alfredo Balena Avenue, 190, 2nd Floor, Room # 281, Belo Horizonte, MG, Brazil.
Pediatr Nephrol. 2023 Jan;38(1):61-75. doi: 10.1007/s00467-022-05683-2. Epub 2022 Jul 21.
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.
The objective of this study is to evaluate the efficacy and safety of eculizumab treatment in pediatric patients with aHUS.
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."
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.
The participants are pediatric patients, up to 18 years old, with aHUS. The intervention was eculizumab treatment.
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.
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.
The number of studies is limited, and the included studies were methodologically heterogeneous. The studies were mostly observational and many had small sample sizes.
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.
CRD42021266255.
非典型溶血尿毒综合征（aHUS）是一种罕见的血栓性微血管病，发病率和死亡率都很高。依库珠单抗是一种人源化抗C5单克隆抗体，于2011年成为首个获批用于治疗aHUS的药物。
本研究旨在评估依库珠单抗治疗儿童aHUS患者的疗效和安全性。
2021年7月，我们查阅了PubMed、Scopus、SciELO和Cochrane图书馆数据库。检索词如下：“非典型溶血尿毒综合征”“aHUS”“依库珠单抗”“儿科学”“儿科”“儿童”“青少年”。
研究纳入标准如下：纳入诊断为aHUS且接受依库珠单抗治疗的儿科患者的临床试验和观察性研究。
参与者为18岁及以下的aHUS儿科患者。干预措施为依库珠单抗治疗。
为进行质量评估，我们使用了纽卡斯尔-渥太华量表、美国国立卫生研究院（NIH）病例系列研究质量评估工具以及干预性非随机研究的偏倚风险（ROBINS-I）工具。
初步检索获得433项研究，经全面评估后筛选出15项：9项队列研究、4项病例系列研究和1项临床试验。发表日期为2015年至2021年。总共纳入940例儿科患者，其中682例接受了依库珠单抗治疗。所有研究均报告，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。接受依库珠单抗治疗的所有患者的死亡率为1.6%。
研究数量有限，且纳入研究在方法上存在异质性。这些研究大多为观察性研究，许多研究样本量较小。
依库珠单抗治疗儿童aHUS似乎安全有效。有必要开展更多研究以确定其长期疗效、安全性和停药时间。
CRD42021266255。
