Phase I trial of human umbilical cord-derived mesenchymal stem cells for treatment of severe bronchopulmonary dysplasia

作者全名："Xia, Yunqiu; Lang, Tingyuan; Niu, Yuqin; Wu, Xian; Zhou, Ou; Dai, Jihong; Bao, Lei; Yang, Ke; Zou, Lin; Fu, Zhou; Geng, Gang"

作者地址："[Xia, Yunqiu] Chongqing Med Univ, Natl Clin Res Ctr Child Hlth & Disorders, China Int Sci & Technol Cooperat Base Child Dev &, Minist Educ,Key Lab Child Dev & Disorders,China In, Chongqing 400014, Peoples R China; [Lang, Tingyuan] Chongqing Univ, Canc Hosp, Sch Med, Dept Gynecol Oncol, Chongqing 400030, Peoples R China; [Niu, Yuqin] Shihezi Univ, Affiliated Hosp 1, Sch Med, Shihezi 832008, Xinjiang, Peoples R China; [Wu, Xian] Sichuan Univ, West China Univ Hosp 2, Dept Resp Med, Chengdu 610041, Sichuan, Peoples R China; [Zhou, Ou; Dai, Jihong; Fu, Zhou; Geng, Gang] Chongqing Med Univ, Childrens Hosp, Dept Resp Med, Chongqing 400014, Peoples R China; [Bao, Lei] Chongqing Med Univ, Dept Neonatol, Childrens Hosp, Chongqing 400014, Peoples R China; [Yang, Ke; Fu, Zhou] Chongqing Med Univ, Childrens Hosp, Chongqing Engn Technol Res Ctr Stem Cell Therapy, Chongqing 400014, Peoples R China; [Zou, Lin] Chongqing Med Univ, Childrens Hosp, Ctr Clin Mol Med, Chongqing 400014, Peoples R China"

通信作者："Fu, Z; Geng, G (通讯作者)，Chongqing Med Univ, Childrens Hosp, Dept Resp Med, Chongqing 400014, Peoples R China."

来源：GENES & DISEASES

ESI学科分类：MOLECULAR BIOLOGY & GENETICS

WOS号：WOS:000994792700001

JCR分区：Q1

影响因子：6.8

年份：2023

卷号：10

期号：2

开始页：521

结束页：530

文献类型：Article

关键词：Bronchopulmonary dysplasia; Dose escalation; Human umbilical cord-derived mesenchymal stem cells; Intravenous treatment; Phase I trial

摘要："Severe bronchopulmonary dysplasia (BPD) is a chronic lung disorder that primarily affects premature babies with extremely low birth weight and involves in multiple organ sys-tem; 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 totest 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 x 106 cells/kg) and the next seven patients were treated with high -dose hUC-MSCs (5 x 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 infu-sion. 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.(c) 2022 The Authors. Publishing services by Elsevier B.V. on behalf of KeAi Communications Co., Ltd. This is an open access article under the CC BY-NC-ND license (http://creativecommons. org/licenses/by-nc-nd/4.0/)."

基金机构：Science and Technology Research Program of Chongqing Municipality Education Commision [KJQN201800407]

基金资助正文：Funding This work was partially supported by the Science and Technology Research Program of Chongqing Municipality Education Commision (No. KJQN201800407) .