甲基强的松龙与静脉注射免疫球蛋白治疗儿童炎症性多系统综合征-与SARS-CoV-2暂时相关（PIMS-TS）的多中心随机试验：瑞士儿科复苏试验方案
Multicenter Randomized Trial of Methylprednisolone vs. Intravenous Immunoglobulins to Treat the Pediatric Inflammatory Multisystem Syndrome-Temporally Associated With SARS-CoV-2 (PIMS-TS): Protocol of the Swissped RECOVERY Trial.
作者信息
Welzel Tatjana, Schöbi Nina, André Maya C, Bailey Douggl G N, Blanchard-Rohner Geraldine, Buettcher Michael, Grazioli Serge, Koehler Henrik, Perez Marie-Helene, Trück Johannes, Vanoni Federica, Zimmermann Petra, Atkinson Andrew, Sanchez Carlos, Whittaker Elizabeth, Faust Saul N, Bielicki Julia A, Schlapbach Luregn J
机构信息
Paediatric Research Center, University Children's Hospital Basel, University of Basel, Basel, Switzerland.
Paediatric Pharmacology and Pharmacometrics, University Children's Hospital Basel, University of Basel, Basel, Switzerland.
出版信息
Front Pediatr. 2022 May 20;10:905046. doi: 10.3389/fped.2022.905046. eCollection 2022.
INTRODUCTION
In 2020, a new disease entitled Pediatric Inflammatory Multisystem Syndrome temporally associated with COVID-19 (PIMS-TS), or Multisystem Inflammatory Syndrome in Children (MIS-C), emerged, with thousands of children affected globally. There is no available evidence based on randomized controlled trials (RCT) to date on the two most commonly used immunomodulatory treatments, intravenous immunoglobulins (IVIG) and corticosteroids. Therefore, the Swissped RECOVERY trial was conducted to assess whether intravenous (IV) methylprednisolone shortens hospital length of stay compared with IVIG.
METHODS AND ANALYSIS
Swissped RECOVERY is an ongoing investigator-initiated, open-label, multicenter two-arm RCT in children and adolescents <18 years hospitalized with a diagnosis of PIMS-TS. The trial is recruiting at 10 sites across Switzerland. Patients diagnosed with PIMS-TS are randomized 1:1 to methylprednisolone IV (10 mg/kg/day for 3 days) or IVIG (2 g/kg as a single dose). The primary outcome is hospital length of stay censored at day 28, death, or discharge (whichever is first). The target total sample size is ~80 patients 1:1 randomized to each study arm. Ancillary and exploratory studies on inflammation, vaccination acceptance and coverage, long-term outcomes, and healthcare costs are pre-planned.
SIGNIFICANCE
Currently, robust trial evidence for the treatment of PIMS-TS is lacking, with a controversy surrounding the use of corticosteroids vs. IVIG. This trial will provide evidence for the effectiveness and safety of these two treatments.
ETHICS AND DISSEMINATION
The study protocol, which was designed based on the U.K. RECOVERY trial, the patient information and consent forms, and other study-specific study documents were approved by the local ethics committees (Project ID: 2021-00362).
REGISTRATION DETAILS
The study is registered on the Swiss National Clinical Trials Portal (SNCTP000004720) and Clinicaltrials.gov (NCT04826588).
引言
2020年，一种名为儿童炎症性多系统综合征（与新冠病毒 temporally 相关，简称PIMS-TS）或儿童多系统炎症综合征（MIS-C）的新疾病出现，全球数千名儿童受到影响。迄今为止，尚无基于随机对照试验（RCT）的证据来证明两种最常用的免疫调节治疗方法，即静脉注射免疫球蛋白（IVIG）和皮质类固醇的效果。因此，开展了瑞士儿童多系统炎症综合征康复试验（Swissped RECOVERY），以评估静脉注射甲泼尼龙与IVIG相比是否能缩短住院时间。
方法与分析
瑞士儿童多系统炎症综合征康复试验是一项正在进行的、由研究者发起的、开放标签、多中心双臂随机对照试验，针对18岁以下因PIMS-TS诊断而住院的儿童和青少年。该试验在瑞士的10个地点招募患者。被诊断为PIMS-TS的患者按1:1随机分为静脉注射甲泼尼龙组（10mg/kg/天，共3天）或IVIG组（2g/kg单剂量）。主要结局是在第28天、死亡或出院（以先发生者为准）时截尾的住院时间。目标总样本量约为80名患者，按1:1随机分配到每个研究组。预先计划了关于炎症、疫苗接种接受度和覆盖率、长期结局以及医疗成本的辅助和探索性研究。
意义
目前，缺乏关于PIMS-TS治疗的有力试验证据，关于皮质类固醇与IVIG的使用存在争议。该试验将为这两种治疗方法的有效性和安全性提供证据。
伦理与传播
该研究方案基于英国康复试验设计，患者信息和同意书以及其他特定研究文件已获得当地伦理委员会批准（项目编号：2021-00362）。
注册详情
该研究已在瑞士国家临床试验门户网站（SNCTP000004720）和美国国立医学图书馆临床试验数据库（Clinicaltrials.gov，编号NCT04826588）注册。
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