Management of Multisystem Inflammatory Syndrome in Children: Decision-Making Regarding a New Condition in the Absence of Clinical Trial Data.
Management of Multisystem Inflammatory Syndrome in Children: Decision-Making Regarding a New Condition in the Absence of Clinical Trial Data.
复制标题
DOI：
10.1016/j.cjca.2022.11.011
复制
发表时间：
2023-06
影响因子：
6.2
通讯作者：
McCrindle, Brian W.
中科院分区：
文献类型：
--
作者：
Harahsheh, Ashraf S.;Portman, Michael A.;Khoury, Michael;Elias, Matthew D.;Lee, Simon;Lin, Justin;McCrindle, Brian W.
儿童多系统炎症综合征（MIS-C）是一种新的疾病，在COVID-19大流行期间演变而来，最初报告的严重疾病包括使用体外膜氧合和死亡。各机构迅速组建了工作队，以制定治疗方案。在国家/国际一级，合作者和协会组成共识编写小组起草准则。这些准则和算法最初是基于专家意见和小案例系列。一些研究小组使用了德尔菲方法，由此产生的指南通常模仿其他类似MIS-C的疾病，如川崎（KD）。例如，静脉注射免疫球蛋白（IVIG），一种已知的KD有效治疗方法，被推荐用于MIS-C。在流感大流行的早期，许多人更喜欢IVIG而不是类固醇作为一线治疗。随着证据的发展，一些指南也在发展，现在支持IVIG与类固醇的双重使用作为一线治疗。相比之下，对于一些症状轻微的MIS-C患者，停止免疫治疗成为一种选择。在此，我们回顾了指南，并讨论了早期建议的证据，这是如何演变的，专家意见和观察数据的作用和局限性，以及利用现有研究基础设施的重要性，如重症监护室合作（克服COVID-19监测登记）和国际川崎登记。最后，我们讨论了快速开发、部署和调整临床试验的策略，这些临床试验评估了儿童罕见疾病的治疗，其中可能包括传统临床试验设计的替代方案。新型冠状病毒大流行期间MIS-C的出现凸显了对新病症研究的未满足需求。
Multisystem inflammatory syndrome in children (MIS-C) is a new illness that evolved during the COVID-19 pandemic with initial reports of severe disease including use of extracorporeal membrane oxygenation and death. Institutions rapidly assembled task forces to develop treatment algorithms. At the national/international levels, collaboratives and associations assembled consensus writing groups to draft guidelines. These guidelines and algorithms were initially on the basis of expert opinion and small case series. Some groups used the Delphi approach, and the resultant guidelines often mimicked those for other conditions that resembled MIS-C, like Kawasaki disease (KD). For instance, intravenous immunoglobulin (IVIG), a known effective treatment for KD, was recommended for MIS-C. Early in the pandemic many favoured IVIG over steroids as first-line therapy. As evidence evolved so did some guidelines, which now endorse the dual use of IVIG with steroids as first-line therapy. In contrast, withholding immunotherapy became an option for some MIS-C patients with mild symptoms. Herein, we review guidelines and discuss the evidence informing early recommendations, how this has evolved, the role and limitations of expert opinion and observational data, and the importance of leveraging existing research infrastructures, such as the intensive care unit collaborative (Overcoming COVID-19 surveillance registry), and the International Kawasaki Disease Registry. Finally, we discuss strategies to rapidly develop, deploy, and adapt clinical trials evaluating the treatment of such rare conditions in children, which might include alternatives to conventional clinical trial design. The emergence of MIS-C during the COVID-19 pandemic has highlighted unmet needs regarding research of a new condition.
登录
查看更多内容
影响因子：
3.2
作者：
Ankola, Ashish A.;Bradford, Victoria R.;VanderPluym, Christina J.
通讯作者：
VanderPluym, Christina J.
影响因子：
120.7
作者：
Feldstein, Leora R.;Tenforde, Mark W.;Randolph, Adrienne G.
通讯作者：
Randolph, Adrienne G.
影响因子：
--
作者：
DOWNING, SE;LEE, JC;TAYLOR, JFN
通讯作者：
TAYLOR, JFN
影响因子：
3.6
作者：
Cattalini M;Taddio A;Bracaglia C;Cimaz R;Paolera SD;Filocamo G;La Torre F;Lattanzi B;Marchesi A;Simonini G;Zuccotti G;Zunica F;Villani A;Ravelli A;Rheumatology Study Group of the Italian Society of Pediatrics
通讯作者：
Rheumatology Study Group of the Italian Society of Pediatrics
基于共识的临床建议和研究优先级，用于住院的儿童抗凝血栓预防症，因1909999名相关疾病而住院。
DOI：
10.1111/jth.15073
发表时间：
2020-11
期刊：
Journal of thrombosis and haemostasis : JTH
影响因子：
--
作者：
通讯作者：
--