免疫球蛋白联合泼尼松龙对降低川崎病患者冠状动脉病变的影响：一项 III 期、多中心、开放标签、盲终点随机对照临床试验研究方案。
Effects of immunoglobulin plus prednisolone in reducing coronary artery lesions in patients with Kawasaki disease: study protocol for a phase III multicenter, open-label, blinded-endpoints randomized controlled trial.
机构信息
Heart Center, Children's Hospital of Fudan University, Shanghai, 201102, China.
Clinical Trial Unit, Children's Hospital of Fudan University, Shanghai, 201102, China.
出版信息
Trials. 2021 Dec 11;22(1):898. doi: 10.1186/s13063-021-05807-3.
BACKGROUND
Kawasaki disease (KD) is an acute systemic vasculitis of unclear etiology that mainly affects infants and young children. Strategies to reduce the incidence and severity of coronary artery lesions (CALs), the determinant factor in the long-term prognosis of KD, are currently a focus of studies on KD. Corticosteroids, preferred in the treatment of the majority of vasculitides, are controversial in the treatment of acute KD. In this trial, we will evaluate whether the addition of prednisolone to standard intravenous immunoglobulin (IVIG) plus aspirin therapy can reduce the occurrence of CAL in Chinese patients with KD.
METHODS
This is a multicenter, prospective, open-label, randomized controlled trial, which is expected to be conducted in more than 20 hospitals in China and aims to assess the efficacy and safety of IVIG + prednisolone treatment versus standard treatment. Patients with KD who fulfill the inclusion and exclusion criteria will be recruited and randomized (1:1) to receive either a large dose of IVIG (2 g/kg over 12-24 h with a maximum dose of 60 g) + aspirin 30 mg/kg/d or IVIG (2 g/kg over 12-24 h) + aspirin 30 mg/kg/d + prednisolone (2 mg/kg/d with a maximum dose of 60 mg tapered over 15 days after normalization of C-reactive protein concentration). The primary outcome will be the occurrence of CAL at 1 month of illness. The follow-up duration for each participant will be set as 1 year. Patients and treating physicians will be unmasked to group allocation.
DISCUSSION
This will be the first multicenter randomized controlled trial to evaluate the efficacy of IVIG + aspirin + prednisolone in Chinese pediatric patients with KD, which may provide high-level evidence for improving the initial treatment for acute KD.
TRIAL REGISTRATION
ClinicalTrials.gov NCT04078568 . Registered on 16 August 2018.
背景
川崎病（KD）是一种病因不明的急性全身性血管炎，主要影响婴儿和幼儿。降低冠状动脉病变（CALs）发生率和严重程度的策略是目前 KD 研究的重点，CALs 是 KD 长期预后的决定因素。皮质类固醇在大多数血管炎的治疗中被优先使用，但在急性 KD 的治疗中存在争议。在这项试验中，我们将评估在 KD 中国患者中，泼尼松龙联合标准静脉注射免疫球蛋白（IVIG）加阿司匹林治疗是否可以减少 CAL 的发生。
方法
这是一项多中心、前瞻性、开放性、随机对照试验，预计将在中国的 20 多家医院进行，旨在评估 IVIG + 泼尼松龙治疗与标准治疗的疗效和安全性。符合纳入和排除标准的 KD 患者将被招募并随机（1:1）接受大剂量 IVIG（2 g/kg 静脉滴注 12-24 小时，最大剂量 60 g）+阿司匹林 30 mg/kg/d 或 IVIG（2 g/kg 静脉滴注 12-24 小时）+阿司匹林 30 mg/kg/d+泼尼松龙（2 mg/kg/d，最大剂量 60 mg，在 CRP 浓度正常后 15 天逐渐减量）。主要结局是疾病 1 个月时发生 CAL。每位参与者的随访时间设定为 1 年。患者和治疗医生将对分组情况进行盲法评估。
讨论
这将是第一项评估 IVIG + 阿司匹林 + 泼尼松龙在中国儿科 KD 患者中的疗效的多中心随机对照试验，这可能为改善急性 KD 的初始治疗提供高级别证据。
试验注册
ClinicalTrials.gov NCT04078568，注册于 2018 年 8 月 16 日。
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