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脐带来源间充质干细胞治疗改善脑瘫患儿生活质量和自理能力:一项回顾性研究结果

Wharton's Jelly Mesenchymal Stem Cell Administration Improves Quality of Life and Self-Sufficiency in Children with Cerebral Palsy: Results from a Retrospective Study.

作者信息

Boruczkowski Dariusz, Zdolińska-Malinowska Izabela

机构信息

Polski Bank Komórek Macierzystych S.A./FamiCord Group (Polish Stem Cell Bank), Jana Pawła II 29, Warsaw, Poland.

出版信息

Stem Cells Int. 2019 May 2;2019:7402151. doi: 10.1155/2019/7402151. eCollection 2019.

Abstract

The aim of this paper was to describe the outcome of the therapeutic administration of allogenic mesenchymal stem cells obtained from Wharton's jelly (WJ-MSCs) in children with cerebral palsy (CP) during a medical therapeutic experiment. We retrospectively analyzed the records of 109 patients recruited in daily clinical practice. Each patient received 1-10 injections and was examined by the same neurologist (study investigator (SI)) on the day of each infusion. The SI used a 6-point Likert scale to assess the quality of life (QoL) and self-sufficiency of the patients on the basis of the neurological examination. Children with >50% follow-ups after this administration were included into the quantitative analysis. In addition, the assessments of the parents and other health care professionals were obtained for 23 patients and compared with those of the SI. Forty-eight of 54 analyzed patients (88.9%) achieved some improvement in health status. Forty-eight (88.9%) patients experienced an increase in their QoL, and 21 patients (38.9%) achieved an increase in their self-sufficiency level. Improvement was achieved in 17 areas. Adverse events were mild and temporary except one case of epilepsy deterioration leading to treatment discontinuation. Age, body mass, and cell dose were not significant predictors of QoL response, contrary to epilepsy; developmental breakthrough was dose-dependent.

摘要

本文旨在描述在一项医学治疗实验中,对患有脑瘫(CP)的儿童进行从脐带华通氏胶中获取的同种异体间充质干细胞(WJ-MSCs)治疗给药的结果。我们回顾性分析了日常临床实践中招募的109例患者的记录。每位患者接受了1至10次注射,并在每次输注当天由同一位神经科医生(研究调查员(SI))进行检查。SI基于神经学检查,使用6点李克特量表来评估患者的生活质量(QoL)和自理能力。此次给药后随访率超过50%的儿童被纳入定量分析。此外,还获取了23例患者的家长和其他医护人员的评估结果,并与SI的评估结果进行比较。在分析的54例患者中,有48例(88.9%)的健康状况有一定改善。48例(88.9%)患者的生活质量有所提高,21例(38.9%)患者的自理能力水平有所提高。在17个方面实现了改善。不良事件较轻且为暂时性,但有1例癫痫病情恶化导致治疗中断除外。与癫痫情况相反,年龄、体重和细胞剂量并非生活质量反应的显著预测因素;发育突破呈剂量依赖性。

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本文引用的文献

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