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Assessing the Efficacy of Cell Transplantation for Parkinson's Disease: A Patient-Centered Approach.

ABSTRACT: 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.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.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.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.

SUBMITTER: Polgar S

PROVIDER: S-EPMC6130410 | biostudies-other | 2018

REPOSITORIES: biostudies-other

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Assessing the Efficacy of Cell Transplantation for Parkinson's Disease: A Patient-Centered Approach.

Polgar Stephen S Karimi Leila L Buultjens Melissa M Morris Meg E ME Busse Monica M

Journal of Parkinson's disease 20180101 3

<h4>Background</h4>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.<h4>Objectives</h4>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.<h4>Methods</h4>The current practice of relying on a single primary outcome measure does not appear to provide the evi ...[more]

PMID: 29889080

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