异体人脐带间充质干细胞治疗儿童重度支气管肺发育不良的随机对照研究（MSC-BPD 试验）方案。
Allogeneic human umbilical cord-derived mesenchymal stem cells for severe bronchopulmonary dysplasia in children: study protocol for a randomized controlled trial (MSC-BPD trial).
机构信息
Pediatric Research Institute, Children's Hospital of Chongqing Medical University, Ministry of Education Key Laboratory of Child Development and Disorders, No 136, 2nd Zhongshan Rd, Yuzhong District, Chongqing, 400014, China.
Chongqing Key Laboratory of Pediatrics, Chongqing, China.
出版信息
Trials. 2020 Jan 31;21(1):125. doi: 10.1186/s13063-019-3935-x.
BACKGROUND
Bronchopulmonary dysplasia (BPD) is a complex lung pathological lesion secondary to multiple factors and one of the most common chronic lung diseases. It has a poor prognosis, especially in preterm infants. However, effective therapies for this disease are lacking. Stem-cell therapy is a promising way to improve lung injury and abnormal alveolarization, and the human umbilical cord (hUC) is a good source of mesenchymal stem cells (MSCs), which have demonstrated efficacy in other diseases. We hypothesized that intravenously administered allogeneic hUC-MSCs are safe and effective for severe BPD.
METHODS
The MSC-BPD trial is a randomized, single-center, open-label, dose-escalation, phase-II trial designed to investigate the safety and efficacy of hUC-MSCs in children with severe BPD. In this study, 72 patients will be enrolled and randomly divided into two intervention groups and one control group. Patients in the intervention groups will receive a low dose of hUC-MSCs (n = 24; 2.5 million cells/kg) or a high dose of hUC-MSCs (n = 24; 5 million cells/kg) in combination with traditional supportive treatments for BPD. The patients in the control group (n = 24) will be treated with traditional supportive treatments alone without hUC-MSCs. The primary outcome measures will be cumulative duration of oxygen therapy. Follow-up assessments will be performed at 1, 3, 6, 12, and 24 months post intervention, and the key outcome during follow-up will be changes on chest radiography. Statistical analyses will evaluate the efficacy of the hUC-MSC treatment.
DISCUSSION
This will be the first randomized controlled trial to evaluate the safety and efficacy of intravenously administered hUC-MSCs in children with severe BPD. Its results should provide a new evidence-based therapy for severe BPD.
TRIAL REGISTRATION
ClinicalTrials.gov, ID: NCT03601416. Registered on 26 July 2018.
背景
支气管肺发育不良（BPD）是一种由多种因素引起的复杂肺部病理病变，是早产儿最常见的慢性肺部疾病之一。其预后较差，但目前针对该病缺乏有效的治疗方法。干细胞治疗是改善肺损伤和异常肺泡化的一种很有前途的方法，人脐带（hUC）是间充质干细胞（MSCs）的良好来源，在其他疾病中已证明其具有疗效。我们假设静脉内给予同种异体 hUC-MSCs 对严重 BPD 是安全且有效的。
方法
MSC-BPD 试验是一项随机、单中心、开放标签、剂量递增、二期试验，旨在研究 hUC-MSCs 治疗严重 BPD 患儿的安全性和疗效。本研究共纳入 72 例患者，并随机分为两组干预组和一组对照组。干预组患者将接受低剂量 hUC-MSCs（n = 24；250 万细胞/kg）或高剂量 hUC-MSCs（n = 24；500 万细胞/kg）联合传统 BPD 支持治疗。对照组患者（n = 24）将仅接受传统 BPD 支持治疗而不接受 hUC-MSCs 治疗。主要结局指标为吸氧时间的累积时间。干预后 1、3、6、12 和 24 个月进行随访评估，随访期间的关键结局为胸部 X 线变化。统计分析将评估 hUC-MSC 治疗的疗效。
讨论
这将是第一项评估静脉内给予 hUC-MSCs 治疗严重 BPD 患儿安全性和疗效的随机对照试验。其结果应可为严重 BPD 提供一种新的循证治疗方法。
试验注册
ClinicalTrials.gov，ID：NCT03601416。于 2018 年 7 月 26 日注册。
Zotero 插件