帕金森病研究中的替代终点

Surrogate endpoints in Parkinson's disease research.

作者信息

Biglan Kevin M, Holloway Robert G

机构信息

Department of Neurology, University of Rochester, 1351 Mt. Hope Avenue, Rochester, NY 14620, USA.

出版信息

Curr Neurol Neurosci Rep. 2003 Jul;3(4):314-20. doi: 10.1007/s11910-003-0008-y.

DOI:10.1007/s11910-003-0008-y

PMID:12930701

Abstract

Biomarkers are important tools in understanding the underlying mechanisms of causation, progression, and treatment effects in Parkinson's disease (PD). In addition, these biomarkers may be utilized as surrogate endpoints that, when used appropriately, can lead to important advances in therapeutics in a timely and cost-effective manner. This paper outlines the definition, role, validity process, and risks associated with surrogate endpoints. The use of biomarkers in recent PD clinical trials is discussed and potential shortcomings and unanswered questions related to interpreting these outcomes are reviewed. Finally, the significant challenges that lie ahead for validating and interpreting surrogate endpoints in PD are addressed.

摘要

生物标志物是理解帕金森病（PD）病因、进展及治疗效果潜在机制的重要工具。此外，这些生物标志物可用作替代终点，若使用得当，能够以及时且具成本效益的方式推动治疗学取得重大进展。本文概述了替代终点的定义、作用、验证过程及相关风险。讨论了生物标志物在近期PD临床试验中的应用，并回顾了与解读这些结果相关的潜在不足和未解决的问题。最后，阐述了在PD中验证和解读替代终点面临的重大挑战。