Motor function and safety after allogeneic cord blood and cord tissue‐derived mesenchymal stromal cells in cerebral palsy: An open‐label, randomized trial

医学 脐带 脑瘫 随机对照试验 外科 麻醉 物理疗法 解剖
作者
Jessica Sun,Laura E. Case,Colleen McLaughlin,Alicia Burgess,Natalie Skergan,Sydney Crane,Joan Jasien,Mohamad A. Mikati,Jesse D. Troy,Joanne Kurtzberg
出处
期刊:Developmental Medicine & Child Neurology [Wiley]
卷期号:64 (12): 1477-1486 被引量:29
标识
DOI:10.1111/dmcn.15325
摘要

Abstract Aim To evaluate safety and motor function after treatment with allogeneic umbilical cord blood (AlloCB) or umbilical cord tissue‐derived mesenchymal stromal cells (hCT‐MSC) in children with cerebral palsy (CP). Method Ninety‐one children (52 males, 39 females; median age 3 years 7 months [range 2–5 years]) with CP due to hypoxic–ischemic encephalopathy, stroke, or periventricular leukomalacia were randomized to three arms: (1) the AlloCB group received 10 × 10 7 AlloCB total nucleated cells (TNC) per kilogram at baseline ( n = 31); (2) the hCT‐MSC group received 2 × 10 6 hCT‐MSC at baseline, 3 months, and 6 months ( n = 28); (3) the natural history control group received 10 × 10 7 AlloCB TNC per kilogram at 12 months ( n = 31). Motor function was assessed with the Gross Motor Function Measure‐66 (GMFM‐66) and Peabody Developmental Motor Scale, Second Edition. Results Infusions ( n = 143) were well tolerated, with eight infusion reactions (three in the AlloCB group, five in hCT‐MSC) and no other safety concerns. At 12 months, the mean differences (95% confidence intervals [CI]) between actual and expected changes in GMFM‐66 score were AlloCB 5.8 points (3.4–8.2), hCT‐MSC 4.3 (2.2–6.4), and natural history 3.1 (1.4–5.0). In exploratory, post hoc analysis, the mean GMFM‐66 score (95% CI) of the hCT‐MSC group was 1.4 points higher than natural history (−1.1 to 4.0; p = 0.27), and the AlloCB group was 3.3 points higher than natural history (0.59–5.93; p = 0.02) after adjustment for baseline Gross Motor Function Classification System level, GMFM‐66 score, and etiology. Interpretation High‐dose AlloCB is a potential cell therapy for CP and should be further tested in a randomized, blinded, placebo‐controlled trial. What this paper adds Unrelated donor allogeneic umbilical cord blood (AlloCB) and human umbilical cord tissue‐derived mesenchymal stromal cell infusion is safe in young children with cerebral palsy. Significant changes in motor function were not observed 6 months after treatment. One year later, treatment with AlloCB was associated with greater increases in Gross Motor Function Measure‐66 scores.
最长约 10秒,即可获得该文献文件

科研通智能强力驱动
Strongly Powered by AbleSci AI
更新
PDF的下载单位、IP信息已删除 (2025-6-4)

科研通是完全免费的文献互助平台,具备全网最快的应助速度,最高的求助完成率。 对每一个文献求助,科研通都将尽心尽力,给求助人一个满意的交代。
实时播报
kingripple发布了新的文献求助10
1秒前
kebing发布了新的文献求助30
1秒前
Owen应助Cici采纳,获得10
2秒前
wanci应助安静复天采纳,获得10
2秒前
冷萃咖啡发布了新的文献求助10
3秒前
4秒前
Mr.Reese完成签到,获得积分10
4秒前
4秒前
luo关注了科研通微信公众号
5秒前
Hevesy完成签到,获得积分10
5秒前
5秒前
6秒前
了111发布了新的文献求助10
6秒前
细心沛山完成签到,获得积分10
6秒前
7秒前
感动葵阴完成签到,获得积分10
7秒前
7秒前
量子星尘发布了新的文献求助10
8秒前
充电宝应助专注的含蕊采纳,获得10
8秒前
宋阳完成签到,获得积分10
9秒前
9秒前
9秒前
一一发布了新的文献求助10
10秒前
11秒前
11秒前
科研通AI2S应助tongxiner采纳,获得10
11秒前
夕禾发布了新的文献求助10
12秒前
T拐拐发布了新的文献求助10
12秒前
12秒前
12秒前
xu完成签到,获得积分10
12秒前
王家腾发布了新的文献求助10
13秒前
13秒前
xml完成签到,获得积分10
13秒前
了111完成签到,获得积分20
14秒前
尔尔发布了新的文献求助30
14秒前
14秒前
Viikey完成签到,获得积分0
14秒前
小半发布了新的文献求助10
14秒前
Zpear完成签到 ,获得积分10
15秒前
高分求助中
A new approach to the extrapolation of accelerated life test data 1000
Cognitive Neuroscience: The Biology of the Mind 1000
Technical Brochure TB 814: LPIT applications in HV gas insulated switchgear 1000
ACSM’s Guidelines for Exercise Testing and Prescription, 12th edition 500
Picture Books with Same-sex Parented Families: Unintentional Censorship 500
Nucleophilic substitution in azasydnone-modified dinitroanisoles 500
不知道标题是什么 500
热门求助领域 (近24小时)
化学 材料科学 医学 生物 工程类 有机化学 生物化学 物理 内科学 纳米技术 计算机科学 化学工程 复合材料 遗传学 基因 物理化学 催化作用 冶金 细胞生物学 免疫学
热门帖子
关注 科研通微信公众号,转发送积分 3970048
求助须知:如何正确求助?哪些是违规求助? 3514739
关于积分的说明 11175783
捐赠科研通 3250115
什么是DOI,文献DOI怎么找? 1795198
邀请新用户注册赠送积分活动 875630
科研通“疑难数据库(出版商)”最低求助积分说明 804951