Outcome Assessment in Parkinson Disease Prevention Trials: Utility of Clinical and Digital Measures

Anat Mirelman, Andrew Siderowf*, Lana Chahine

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

Abstract

Background and ObjectivesThe prodromal phase of Parkinson disease (PD) is accompanied by subtle clinical signs that are not sufficient for diagnosis but could potentially be measured in the context of clinical trials of therapies intended to delay or prevent more definitive clinical features. The objective of this study was to review the available literature on the presence and time course of subtle motor features in prodromal PD in the context of planning for possible clinical trials.MethodsWe reviewed the available literature based on expert opinion. We considered a range of outcomes including measurement of clinical features, patient-reported outcomes, digital markers, and clinical diagnosis.ResultsWe considered these features and measures in the context of patient stratification, intermediate outcomes, and clinically relevant end points, including phenoconversion.DiscussionSubstantial progress has been made in understanding how motor features evolve in the period immediately before a PD diagnosis. Digital measures hold substantial progress for measurement precision and may be additionally relevant because they can be used in naturalistic environments outside the clinic. Future studies should focus on advancing digital sensor technology and analysis and developing methods to implement available methods, particularly determination of a clinical diagnosis of PD, in a clinical trial context.

Original languageEnglish
Pages (from-to)S52-S60
JournalNeurology
Volume99
Issue number7
DOIs
StatePublished - 16 Aug 2022

Funding

FundersFunder number
Prilenia Therapeutics
National Institute of Neurological Disorders and Stroke
Michael J. Fox Foundation for Parkinson's Research
National Parkinson Foundation
Merck
Massachusetts General Hospital
Biogen
Parkinson's Foundation
Voyager Therapeutics

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