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我们做对了吗?一项关于脑瘫细胞治疗临床研究报告结果的范围综述。

Are We Getting It Right? A Scoping Review of Outcomes Reported in Cell Therapy Clinical Studies for Cerebral Palsy.

作者信息

Finch-Edmondson Megan, Paton Madison C B, Honan Ingrid, Karlsson Petra, Stephenson Candice, Chiu Darryl, Reedman Sarah, Griffin Alexandra R, Morgan Catherine, Novak Iona

机构信息

Cerebral Palsy Alliance Research Institute, Speciality of Child and Adolescent Health, Sydney Medical School, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2050, Australia.

Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2050, Australia.

出版信息

J Clin Med. 2022 Dec 9;11(24):7319. doi: 10.3390/jcm11247319.

Abstract

Cell therapies are an emergent treatment for cerebral palsy (CP) with promising evidence demonstrating efficacy for improving gross motor function. However, families value improvements in a range of domains following intervention and the non-motor symptoms, comorbidities and complications of CP can potentially be targeted by cell therapies. We conducted a scoping review to describe all outcomes that have been reported in cell therapy studies for CP to date, and to examine what instruments were used to capture these. Through a systematic search we identified 54 studies comprising 2066 participants that were treated with a range of cell therapy interventions. We categorized the reported 53 unique outcome instruments and additional descriptive measures into 10 categories and 12 sub-categories. Movement and Posture was the most frequently reported outcome category, followed by Safety, however Quality of Life, and various prevalent comorbidities and complications of CP were infrequently reported. Notably, many outcome instruments used do not have evaluative properties and thus are not suitable for measuring change following intervention. We provide a number of recommendations to ensure that future trials generate high-quality outcome data that is aligned with the priorities of the CP community.

摘要

细胞疗法是一种新兴的治疗脑瘫(CP)的方法,有证据表明其在改善粗大运动功能方面具有疗效。然而,家庭重视干预后多个领域的改善,而脑瘫的非运动症状、合并症和并发症可能是细胞疗法的潜在治疗靶点。我们进行了一项范围综述,以描述迄今为止细胞疗法治疗脑瘫的研究中报告的所有结果,并研究用于获取这些结果的工具。通过系统检索,我们确定了54项研究,包括2066名接受了一系列细胞疗法干预的参与者。我们将报告的53种独特的结果工具和其他描述性指标分为10类和12个子类别。运动和姿势是最常报告的结果类别,则是安全性,然而生活质量以及脑瘫的各种常见合并症和并发症报告较少。值得注意的是,许多使用的结果工具没有评估属性,因此不适合测量干预后的变化。我们提出了一些建议,以确保未来的试验能够产生与脑瘫群体的优先事项相一致的高质量结果数据。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5cb3/9786692/fdb3bebbd36b/jcm-11-07319-g001.jpg

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