PDF(Pubmed)
Abstract：
Severe bronchopulmonary dysplasia (BPD) is a chronic lung disorder that primarily affects premature babies with extremely low birth weight and involves in multiple organ system; no effective pharmacotherapy for this disease exists, and mortality remains high. Based on the evidence from previous preclinical studies and phase I clinical trials, this study aims to test the safety of intravenous application of a single dose of human umbilical cord-derived mesenchymal stem cells (hUC-MSCs) in patients with severe BPD. The Mesenchymal Stem cells for Bronchopulmonary Dysplasia Treatment (MSBDT) trial is a single center, open-label, dose-escalation phase I clinical trial. Severe BPD patients were enrolled in Children Hospital of Chongqing Medical University, Chongqing, China. The first six patients were treated with low-dose hUC-MSCs (1 × 106 cells/kg) and the next seven patients were treated with high-dose hUC-MSCs (5 × 106 cells/kg). This study is registered with ClinicalTrials.gov, number NCT03558334. No prespecified infusion-associated adverse events, immediate complication, respiratory or cardiovascular compromise were observed during infusion and 24 h after infusion. No significant changes in safety laboratory values were observed. One death event occurred in the low-dose group on study day 10, and one death event occurred in the high-dose group on study day 24, while, after review in detail, the two cases are not believed to be infusion-associated events. In conclusion, intravenous application of a single dose of hUC-MSCs was tolerated in thirteen patients with severe BPD.
摘要：
严重支气管肺发育不良（BPD）是一种慢性肺部疾病，主要影响极低出生体重的早产儿，并累及多器官系统；目前尚无有效的药物治疗方法。死亡率仍然很高。根据以前的临床前研究和I期临床试验的证据，这项研究旨在测试在重度BPD患者中静脉应用单剂量人脐带间充质干细胞（hUC-MSCs）的安全性。间充质干细胞治疗支气管肺发育不良（MSBDT）试验是一个单中心，开放标签，剂量递增I期临床试验。重庆医科大学附属儿童医院收治的重症BPD患者,重庆,中国。前6例患者接受低剂量hUC-MSCs（1×106细胞/kg）治疗，后7例患者接受高剂量hUC-MSCs（5×106细胞/kg）治疗。这项研究在ClinicalTrials.gov注册，编号NCT03558334。无预先设定的输液相关不良事件,立即并发症，在输注期间和输注后24小时观察到呼吸或心血管损害。没有观察到安全性实验室值的显著变化。低剂量组在研究第10天发生1例死亡事件,高剂量组在研究第24天发生1例死亡事件,经过详细的审查，这两例被认为不是输液相关事件.总之,在13例重度BPD患者中,静脉应用单次剂量的hUC-MSCs是耐受的.
