Determination of minimal clinically important change in early and advanced Parkinson's disease
- 24 March 2011
- journal article
- research article
- Published by Wiley in Movement Disorders
- Vol. 26 (5), 813-818
- https://doi.org/10.1002/mds.23638
Abstract
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 SocietyThis publication has 16 references indexed in Scilit:
- Irreversible motor impairment in young addicts ? ephedrone, manganism or both?Acta Neurologica Scandinavica, 2007
- Minimal clinically important change on the unified Parkinson's disease rating scaleMovement Disorders, 2006
- Defining a minimal clinically relevant difference for the unified Parkinson's rating scale: an important but still unmet need.Movement Disorders, 2006
- Double‐blind, randomized, controlled trial of rasagiline as monotherapy in early Parkinson's disease patientsMovement Disorders, 2004
- Efficacy of piribedil as early combination to levodopa in patients with stable Parkinson's disease: A 6‐month, randomized, placebo‐controlled studyMovement Disorders, 2003
- Pallidotomy: A Comparison of Responders and NonrespondersNeurosurgery, 2001
- Pergolide monotherapy in the treatment of early PDNeurology, 1999
- A 3-year randomized trial of ropinirole and bromocriptine in early Parkinson’s diseaseNeurology, 1999
- Ropinirole in the treatment of early Parkinson's disease: A 6‐month interim report of a 5‐year levodopa‐controlled studyMovement Disorders, 1998
- Safety and efficacy of pramipexole in early Parkinson disease. A randomized dose-ranging study. Parkinson Study GroupPublished by American Medical Association (AMA) ,1997