The Impact of Umbilical Cord Mesenchymal Stem Cells on Motor Function in Children with Cerebral Palsy: Results of a Real-world, Compassionate use Study.

IF 4.5 3区医学 Q2 CELL & TISSUE ENGINEERING

Stem Cell Reviews and Reports Pub Date : 2024-08-01 Epub Date: 2024-06-15 DOI:10.1007/s12015-024-10742-2

Magdalena Chrościńska-Kawczyk, Izabela Zdolińska-Malinowska, Dariusz Boruczkowski

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Abstract

The aim of this study was to analyze the impact of human umbilical cord-derived MSCs (hUC-MSCs) on motor function in children with cerebral palsy (CP). The study enrolled 152 children with CP who received up to two courses of five hUC-MSCs injections. Children's motor functions were assessed with the Gross Motor Function Measure (GMFM), 6-Minute Walk Test (6-MWT), Timed Up and Go test (Up&Go test), and Lovett's test, and mental abilities were assessed with the Clinical Global Impression (CGI) scale. Data collected at visit 1 (baseline) and visit 5 (after four injections) were analyzed retrospectively. After four hUC-MSCs administrations, all evaluated parameters improved. The change in GMFM score, by a median of 1.9 points (IQR: 0.0-8.0), correlated with age. This change was observed in all GFMCS groups and was noticed in all assessed GMFM areas. A median increase of 75 m (IQR: 20.0-115.0) was noted on the 6-MWT, and this correlated with GMFM score change. Time on the Up&Go test was reduced by a median of 2 s (IQR: -3 to - 1) and the change correlated with age, GMFM score at baseline, and the difference observed on the 6-MWT. Results of Lovett's test indicated slight changes in muscle strength. According to the CGI, 75.5% (96/151) of children were seriously (level VI) or significantly ill (level V) at the 1^st visit, with any improvement observed in 63.6% (96/151) of patients at the 5^th visit, 23.8% (36/151) with improvement (level II) or great improvement (level I). In conclusion, the application of hUC-MSCs generally enhanced functional performance, but individual responses varied. The therapy also benefited children with high level of disability but not to the same extent as the initially less disabled children. Although younger patients responded better to the treatment, older children can also benefit. Trial Registration 152/2018/KB/VII and 119/2021/KB/VIII. Retrospective registration in ClinicalTrials: ongoing.

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脐带间充质干细胞对脑瘫儿童运动功能的影响：一项真实世界、同情使用研究的结果。

本研究旨在分析人脐源性间充质干细胞（hUC-MSCs）对脑瘫（CP）儿童运动功能的影响。研究共招募了152名患有CP的儿童，他们接受了最多两个疗程、共五次的hUC-间充质干细胞注射。儿童的运动功能通过粗大运动功能量表（GMFM）、6分钟步行测试（6-MWT）、定时起立行走测试（Up&Go test）和洛维特测试进行评估，心理能力通过临床整体印象量表（CGI）进行评估。对第 1 次（基线）和第 5 次（四次注射后）收集的数据进行了回顾性分析。注射四次后，所有评估指标均有所改善。GMFM评分的变化中位数为1.9分（IQR：0.0-8.0），与年龄相关。这一变化出现在所有的 GFMCS 组别中，并在所有的 GMFM 评估区域中均有体现。6-MWT 的中位数增加了 75 米（IQR：20.0-115.0），这与 GMFM 分数的变化相关。向上和向后运动测试时间的中位数减少了 2 秒（IQR：-3 到 -1），这一变化与年龄、基线时的 GMFM 分数以及 6-MWT 上观察到的差异相关。洛维特测试结果表明，肌肉力量略有变化。根据 CGI，75.5%（96/151）的患儿在第一次就诊时病情严重（VI 级）或显著恶化（V 级），63.6%（96/151）的患者在第五次就诊时病情有所改善，23.8%（36/151）的患者病情有所改善（II 级）或显著改善（I 级）。总之，应用 hUC 间充质干细胞普遍提高了功能表现，但个体反应各不相同。这种疗法也能使高度残疾的儿童受益，但受益程度不如最初残疾程度较轻的儿童。虽然年龄较小的患者对治疗反应较好，但年龄较大的儿童也能从中受益。试验注册号：152/2018/KB/VII 和 119/2021/KB/VIII。临床试验回顾性注册：正在进行中。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Stem Cell Reviews and Reports 医学-细胞生物学

CiteScore

9.30

自引率

4.20%

发文量

审稿时长

3 months

期刊介绍： The purpose of Stem Cell Reviews and Reports is to cover contemporary and emerging areas in stem cell research and regenerative medicine. The journal will consider for publication: i) solicited or unsolicited reviews of topical areas of stem cell biology that highlight, critique and synthesize recent important findings in the field. ii) full length and short reports presenting original experimental work. iii) translational stem cell studies describing results of clinical trials using stem cells as therapeutics. iv) papers focused on diseases of stem cells. v) hypothesis and commentary articles as opinion-based pieces in which authors can propose a new theory, interpretation of a controversial area in stem cell biology, or a stem cell biology question or paradigm. These articles contain more speculation than reviews, but they should be based on solid rationale. vi) protocols as peer-reviewed procedures that provide step-by-step descriptions, outlined in sufficient detail, so that both experts and novices can apply them to their own research. vii) letters to the editor and correspondence. In order to facilitate this exchange of scientific information and exciting novel ideas, the journal has created five thematic sections, focusing on: i) the role of adult stem cells in tissue regeneration; ii) progress in research on induced pluripotent stem cells, embryonic stem cells and mechanism governing embryogenesis and tissue development; iii) the role of microenvironment and extracellular microvesicles in directing the fate of stem cells; iv) mechanisms of stem cell trafficking, stem cell mobilization and homing with special emphasis on hematopoiesis; v) the role of stem cells in aging processes and cancerogenesis.