Document Detail


Outcome measures for clinical trials in Parkinson's disease: achievements and shortcomings.
MedLine Citation:
PMID:  15853525     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Three areas of intense investigation in Parkinson's disease clinical trials include symptomatic treatment of Parkinsonism, disease-modifying therapy (or neuroprotection), and the prevention and treatment of motor complications of dopaminergic therapy. Difficulty interpreting the results of many studies in recent years has been attributed to problems with the chosen outcome measures. This article reviews the most common outcome measures used, assesses their positive attributes and proposes needs for future research. The Unified Parkinson's disease Rating Scale has been extensively validated and is by far the most common outcome measure used in trials of symptomatic therapy. Ambiguities in the response scale descriptors, poor inter-rater reliability of some items and a lack of items addressing nonmotor features of the disease are being addressed in a revision of the scale. Quality of life outcomes are being used in the minority of clinical trials, and no single generic or disease-specific quality of life measure is being used most frequently. Additional work validating several of the disease-specific instruments is needed. When a generic measure is used, its validity for use in Parkinson's disease must be critically assessed despite its previously established validity in other diseases. With respect to measuring motor complications, significant unmet needs include a consensus as to the best way to define the first motor complication and validating time to the first occurrence of motor complications as a surrogate of future disability and quality of life. Measuring the effectiveness of a potential neuroprotective agent presents unique challenges, particularly since symptomatic effects of the experimental agent or concomitant treatment can obscure any neuroprotective effects. Study designs and biomarkers are being developed that may overcome this problem. Currently, neuroimaging techniques that reflect function of the dopaminergic system are the most promising biomarkers but still require additional validation.
Authors:
Connie Marras; Antony E Lang
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Publication Detail:
Type:  Journal Article; Review    
Journal Detail:
Title:  Expert review of neurotherapeutics     Volume:  4     ISSN:  1744-8360     ISO Abbreviation:  Expert Rev Neurother     Publication Date:  2004 Nov 
Date Detail:
Created Date:  2005-04-27     Completed Date:  2006-04-14     Revised Date:  2007-11-15    
Medline Journal Info:
Nlm Unique ID:  101129944     Medline TA:  Expert Rev Neurother     Country:  England    
Other Details:
Languages:  eng     Pagination:  985-93     Citation Subset:  IM    
Affiliation:
Toronto Western Hospital, Movement Disorders Centre, 7 McLaughlin, 399 Bathurst Street, Toronto, ON M5T 2S8, Canada. connie.marras@utoronto.ca
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MeSH Terms
Descriptor/Qualifier:
Antiparkinson Agents / therapeutic use*
Clinical Trials as Topic*
Humans
Motor Neuron Disease / etiology,  prevention & control
Neuroprotective Agents / therapeutic use
Outcome Assessment (Health Care)*
Parkinson Disease / drug therapy*,  physiopathology
Practice Guidelines as Topic
Chemical
Reg. No./Substance:
0/Antiparkinson Agents; 0/Neuroprotective Agents

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