简明综述:针对脑瘫患者的干细胞干预措施:系统评价与荟萃分析

Concise Review: Stem Cell Interventions for People With Cerebral Palsy: Systematic Review With Meta-Analysis.

作者信息

Novak Iona, Walker Karen, Hunt Rod W, Wallace Euan M, Fahey Michael, Badawi Nadia

机构信息

Cerebral Palsy Alliance, The University of Sydney, Sydney, New South Wales, Australia University of Notre Dame Australia, Fremantle, Western Australia, Australia

Cerebral Palsy Alliance, The University of Sydney, Sydney, New South Wales, Australia Grace Centre for Newborn Care, Children's Hospital at Westmead, The University of Sydney, Sydney, New South Wales, Australia.

出版信息

Stem Cells Transl Med. 2016 Aug;5(8):1014-25. doi: 10.5966/sctm.2015-0372. Epub 2016 May 31.

Abstract

UNLABELLED

: Evidence for stem cells as a potential intervention for cerebral palsy is emerging. Our objective was to determine the efficacy and safety of stem cells for improving motor and cognitive function of people with cerebral palsy. Searches were conducted in October 2015 in CENTRAL, EMBASE, MEDLINE, and Cochrane Libraries. Randomized controlled trials and controlled clinical trials of stem cells for cerebral palsy were included. Two authors independently decided upon included trials, extracted data, quality, and risk of bias. The primary outcome was gross motor function. Secondary outcomes were cognitive function and adverse events (AEs). Effects were expressed as standardized mean differences (SMD) with 95% confidence intervals (CI), using a random-effects model. Five trials comprising 328 participants met inclusion criteria. Four cell types were studied: olfactory ensheathing, neural, neural progenitors, and allogeneic umbilical cord blood (UCBs). Transplantation procedures differed from central nervous system neurosurgical transplantation to intravenous/arterial infusion. Participants were followed short-term for only 6 months. Evidence of variable quality indicated a small statistically significant intervention effect from stem cells on gross motor skills (SMD 1.27; 95% CI 0.22, 2.33), with UCBs most effective. There were insufficient and heterogeneous data to compare cognitive effects. Serious AEs were rare (n = 4/135 [3%] stem cells; n = 3/139 [2%] controls). Stem cells appeared to induce short-term improvements in motor skills. Different types of stem cell interventions were compared, meaning the data were heterogeneous and are a study limitation. Further randomized controlled trials are warranted, using rigorous methodologies.

SIGNIFICANCE

Stem cells are emerging as a scientifically plausible treatment and possible cure for cerebral palsy, but are not yet proven. The lack of valid animal models has significantly hampered the scope of clinical trials. Despite the state of current treatment evidence, parents remain optimistic about the potential improvements from stem cell intervention and feel compelled to exhaust all therapeutic options, including stem cell tourism. Receiving unproven therapies from unvalidated sources is potentially dangerous. Thus it is essential that researchers and clinicians stay up to date. A systematic review and meta-analysis summarizing and aggregating current research data may provide more conclusive evidence to inform treatment decision making and help direct future research.

摘要

未标注

干细胞作为脑瘫潜在干预手段的证据正在不断涌现。我们的目的是确定干细胞改善脑瘫患者运动和认知功能的疗效及安全性。于2015年10月检索了CENTRAL、EMBASE、MEDLINE和Cochrane图书馆。纳入了干细胞治疗脑瘫的随机对照试验和对照临床试验。两位作者独立决定纳入的试验,提取数据、质量和偏倚风险。主要结局是粗大运动功能。次要结局是认知功能和不良事件(AE)。效应以标准化均数差(SMD)及95%置信区间(CI)表示,采用随机效应模型。五项试验共328名参与者符合纳入标准。研究了四种细胞类型:嗅鞘细胞、神经细胞、神经祖细胞和异体脐带血(UCB)。移植程序从中枢神经系统神经外科移植到静脉/动脉输注各不相同。参与者仅短期随访6个月。质量参差不齐的证据表明,干细胞对粗大运动技能有小的统计学显著干预效应(SMD 1.27;95%CI 0.22,2.33),其中UCB最有效。比较认知效应的数据不足且存在异质性。严重AE很少见(干细胞组n = 4/135 [3%];对照组n = 3/139 [2%])。干细胞似乎能在短期内改善运动技能。比较了不同类型的干细胞干预措施,这意味着数据存在异质性,是本研究的一个局限。需要采用严格方法进行进一步的随机对照试验。

意义

干细胞正成为一种在科学上看似合理的脑瘫治疗方法及可能的治愈手段,但尚未得到证实。缺乏有效的动物模型严重阻碍了临床试验的范围。尽管现有治疗证据的状况如此,但家长们仍对干细胞干预可能带来的改善持乐观态度,并感到有必要用尽所有治疗选择,包括干细胞旅游。从未经验证的来源接受未经证实的治疗可能很危险。因此,研究人员和临床医生必须与时俱进。一项总结和汇总当前研究数据的系统评价和荟萃分析可能会提供更具决定性的证据,为治疗决策提供信息,并有助于指导未来的研究。

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索