Lee Ji Young, Kim Jimin, Choi Soo-Han, Kim Dong Hyun, Yun Ki Wook, Kim Yae-Jean, Cao Giang Pham Ha, Choi Miyoung, Ahn Jong Gyun
Department of Pediatrics, Severance Children's Hospital, Yonsei University College of Medicine, Seoul 03722, Republic of Korea.
Division for Healthcare Technology Assessment Research, National Evidence-Based Healthcare Collaborating Agency, Seoul 04933, Republic of Korea.
Children (Basel). 2024 Sep 27;11(10):1180. doi: 10.3390/children11101180.
Although there is consensus to use immunoglobulins and corticosteroids as first-line treatments for multisystem inflammatory syndrome in children (MIS-C), the effectiveness of biological immunomodulators in patients refractory to standard therapy remains unclear. We aimed to outline real-world data on biological immunomodulators.
A literature search using Ovid-Medline, EMBASE, Cochrane CDSR, and KMBASE was conducted from September 2021 to August 2022; certainty of evidence was assessed via GRADE.
Among 258 studies, 10 were selected for analysis, of which 2 were observational studies (with control groups receiving standard therapy of either intravenous immunoglobulins and/or glucocorticoids) and 8 were single-arm studies. In all, 145 patients were treated with biological immunomodulators (anakinra (72; 49%) or infliximab (65; 44%)). In the first observational study, patients in the anakinra group initially exhibited a lower left ventricular ejection fraction than those in the control group. In the second study, patients in the infliximab group required less additional therapy and showed lower newly developed left ventricular dysfunction rate and reduced C-reactive protein levels. The clinical outcomes associated with each biological agent in single-arm studies were not reported individually.
Biological immunomodulators are feasible therapeutic options for refractory MIS-C. Nevertheless, further research is warranted to demonstrate clinical efficacy.
尽管对于将免疫球蛋白和皮质类固醇作为儿童多系统炎症综合征（MIS-C）的一线治疗方法已达成共识，但生物免疫调节剂在对标准治疗无效的患者中的有效性仍不明确。我们旨在概述关于生物免疫调节剂的真实世界数据。
于2021年9月至2022年8月使用Ovid-Medline、EMBASE、Cochrane临床对照试验注册库和韩国医学数据库进行文献检索；通过GRADE评估证据的确定性。
在258项研究中，选择了10项进行分析，其中2项为观察性研究（对照组接受静脉注射免疫球蛋白和/或糖皮质激素的标准治疗），8项为单臂研究。共有145例患者接受了生物免疫调节剂治疗（阿那白滞素（72例；49%）或英夫利昔单抗（65例；44%））。在第一项观察性研究中，阿那白滞素组患者最初的左心室射血分数低于对照组。在第二项研究中，英夫利昔单抗组患者需要的额外治疗较少，新发生的左心室功能障碍率较低，C反应蛋白水平降低。单臂研究中与每种生物制剂相关的临床结果未单独报告。
生物免疫调节剂是难治性MIS-C可行的治疗选择。然而，仍需进一步研究以证明其临床疗效。
