Determination of minimal clinically important change in early and advanced Parkinson's disease

Two common primary efficacy outcome measures in Parkinson's disease (PD) are change in Unified Parkinson's Disease Rating Scale (UPDRS) scores in early PD and change in “off” time in patients with motor fluctuations. Defining the minimal clinically important change (MCIC) in these outcome...

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Bibliographic Details
Published in:Movement disorders 2011-04, Vol.26 (5), p.813-818
Main Authors: Hauser, Robert A., Auinger, Peggy
Format: Article
Language:English
Subjects:
Antiparkinson Agents - therapeutic use
clinical global impression
clinical trial
Disability Evaluation
Dose-Response Relationship, Drug
Double-Blind Method
Female
Follow-Up Studies
Humans
Indans - therapeutic use
Levodopa
Male
minimal clinically important change
Neuroprotective Agents - therapeutic use
Parkinson Disease - classification
Parkinson Disease - drug therapy
Parkinson's disease
Placebo Effect
ROC Curve
Severity of Illness Index
Time Factors
treatment
Treatment Outcome
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Summary:Two common primary efficacy outcome measures in Parkinson's disease (PD) are change in Unified Parkinson's Disease Rating Scale (UPDRS) scores in early PD and change in “off” time in patients with motor fluctuations. Defining the minimal clinically important change (MCIC) in these outcome measures is important to interpret the clinical relevance of changes observed in clinical trials and other situations. We analyzed data from 2 multicenter, placebo‐controlled, randomized clinical trials of rasagiline; TEMPO studied 404 early PD subjects, and PRESTO studied 472 levodopa‐treated subjects with motor fluctuations. An anchor‐based approach using clinical global impression of improvement (CGI‐I) was used to determine MCIC for UPDRS scores and daily “off” time. MCIC was defined as mean change in actively treated subjects rated minimally improved on CGI‐I. Receiver operating characteristic (ROC) curves defined optimal cutoffs discriminating between changed and unchanged subjects. MCIC for improvement in total UPDRS score (parts I–III) in early PD was determined to be −3.5 points based on mean scores and −3.0 points based on ROC curves. In addition, we found an MCIC for reduction in “off” time of 1.0 hours as defined by mean reduction in “off” time in active treated subjects self‐rated as minimally improved on CGI‐I minus mean reduction in “off” time in placebo‐treated subjects self‐rated as unchanged (1.9–0.9 hours). We hypothesize that many methodological factors can influence determination of the MCIC, and a range of values is likely to emerge from multiple studies. © 2011 Movement Disorder Society
ISSN:0885-3185
1531-8257
DOI:10.1002/mds.23638