评估细胞移植治疗帕金森病的疗效：以患者为中心的方法。
Assessing the Efficacy of Cell Transplantation for Parkinson's Disease: A Patient-Centered Approach.
机构信息
School of Allied Health, La Trobe University, Bundoora, Melbourne, Australia.
School of Psychology and Public Health, La Trobe University, Bundoora, Melbourne, Australia.
出版信息
J Parkinsons Dis. 2018;8(3):375-383. doi: 10.3233/JPD-181309.
BACKGROUND
Evidence from a growing number of preclinical studies indicate that recently discovered stem cell lines may be translated into viable cellular therapies for people with Parkinson's disease.
OBJECTIVES
In a brief but critical review, we examine the use of primary and secondary outcome measures currently used to evaluate the efficacy of cellular therapies.
METHODS
The current practice of relying on a single primary outcome measure does not appear to provide the evidence required for demonstrating the robust, life-changing recovery anticipated with the successful implementation of cellular therapies.
RESULTS
We propose a 360-degree assessment protocol, which includes co-primary and composite outcome measures to provide accurate and comprehensive evidence of treatment efficacy, from the perspectives of both the researchers and the patients.
背景
越来越多的临床前研究证据表明，最近发现的干细胞系可能被转化为帕金森病患者可行的细胞治疗方法。
目的
在一个简短但关键的综述中，我们检查了目前用于评估细胞治疗疗效的主要和次要结果测量的使用。
方法
目前依赖单一主要结果测量的做法似乎并不能提供成功实施细胞治疗所预期的稳健、改变生活的恢复所需的证据。
结果
我们提出了一个 360 度评估方案，包括共同主要和综合结果测量，从研究人员和患者的角度提供治疗效果的准确和全面的证据。
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