Minimal Clinically Important Difference in Parkinson’s Disease as Assessed in Pivotal Trials of Pramipexole Extended Release

Background. The minimal clinically important difference (MCID) is the smallest change in an outcome measure that is meaningful for patients. Objectives. To calculate the MCID for Unified Parkinson’s Disease Rating Scale (UPDRS) scores in early Parkinson’s disease (EPD) and for UPDRS scores and “OFF”...

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Main Authors: Robert A. Hauser, Mark Forrest Gordon, Yoshikuni Mizuno, Werner Poewe, Paolo Barone, Anthony H. Schapira, Olivier Rascol, Catherine Debieuvre, Mandy Fräßdorf
Format: Article
Language:English
Published: Wiley 2014-01-01
Series:Parkinson's Disease
Online Access:http://dx.doi.org/10.1155/2014/467131
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author Robert A. Hauser
Mark Forrest Gordon
Yoshikuni Mizuno
Werner Poewe
Paolo Barone
Anthony H. Schapira
Olivier Rascol
Catherine Debieuvre
Mandy Fräßdorf
author_facet Robert A. Hauser
Mark Forrest Gordon
Yoshikuni Mizuno
Werner Poewe
Paolo Barone
Anthony H. Schapira
Olivier Rascol
Catherine Debieuvre
Mandy Fräßdorf
author_sort Robert A. Hauser
collection DOAJ
description Background. The minimal clinically important difference (MCID) is the smallest change in an outcome measure that is meaningful for patients. Objectives. To calculate the MCID for Unified Parkinson’s Disease Rating Scale (UPDRS) scores in early Parkinson’s disease (EPD) and for UPDRS scores and “OFF” time in advanced Parkinson’s disease (APD). Methods. We analyzed data from two pivotal, double-blind, parallel-group trials of pramipexole ER that included pramipexole immediate release (IR) as an active comparator. We calculated MCID as the mean change in subjects who received active treatment and rated themselves “a little better” on patient global impression of improvement (PGI-I) minus the mean change in subjects who received placebo and rated themselves unchanged. Results. MCIDs in EPD (pramipexole ER, pramipexole IR) for UPDRS II were −1.8 and −2.0, for UPDRS III −6.2 and −6.1, and for UPDRS II + III −8.0 and −8.1. MCIDs in APD for UPDRS II were −1.8 and −2.3, for UPDRS III −5.2 and −6.5, and for UPDRS II + III −7.1 and −8.8. MCID for “OFF” time (pramipexole ER, pramipexole IR) was −1.0 and −1.3 hours. Conclusions. A range of MCIDs is emerging in the PD literature that provides the basis for power calculations and interpretation of clinical trials.
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spelling doaj-art-54d7f5bec96e4559aeda590d57da22b72025-08-20T03:34:20ZengWileyParkinson's Disease2090-80832042-00802014-01-01201410.1155/2014/467131467131Minimal Clinically Important Difference in Parkinson’s Disease as Assessed in Pivotal Trials of Pramipexole Extended ReleaseRobert A. Hauser0Mark Forrest Gordon1Yoshikuni Mizuno2Werner Poewe3Paolo Barone4Anthony H. Schapira5Olivier Rascol6Catherine Debieuvre7Mandy Fräßdorf8University of South Florida, Parkinson’s Disease and Movement Disorders Center, Byrd Institute, National Parkinson Foundation Center of Excellence, Tampa, FL 33613, USABoehringer Ingelheim Pharmaceuticals, Inc., Ridgefield, CT 06877, USADepartment of Neurology, Juntendo University School of Medicine, Tokyo 113-8421, JapanDepartment of Neurology, Innsbruck Medical University, Innsbruck, AustriaCenter for Neurodegenerative Diseases, Department of Medicine and Surgery, University of Salerno, Salerno, ItalyDepartment of Clinical Neurosciences, University College London Institute of Neurology, London, UKClinical Investigation Center, INSERM CIC-9302 and UMR-825 and Departments of Clinical Pharmacology and Neurosciences, Toulouse University Hospital, Toulouse, FranceBoehringer Ingelheim France S.A.S., Reims, FranceBoehringer Ingelheim Pharmaceuticals, GmbH & Co. KG, Ingelheim, GermanyBackground. The minimal clinically important difference (MCID) is the smallest change in an outcome measure that is meaningful for patients. Objectives. To calculate the MCID for Unified Parkinson’s Disease Rating Scale (UPDRS) scores in early Parkinson’s disease (EPD) and for UPDRS scores and “OFF” time in advanced Parkinson’s disease (APD). Methods. We analyzed data from two pivotal, double-blind, parallel-group trials of pramipexole ER that included pramipexole immediate release (IR) as an active comparator. We calculated MCID as the mean change in subjects who received active treatment and rated themselves “a little better” on patient global impression of improvement (PGI-I) minus the mean change in subjects who received placebo and rated themselves unchanged. Results. MCIDs in EPD (pramipexole ER, pramipexole IR) for UPDRS II were −1.8 and −2.0, for UPDRS III −6.2 and −6.1, and for UPDRS II + III −8.0 and −8.1. MCIDs in APD for UPDRS II were −1.8 and −2.3, for UPDRS III −5.2 and −6.5, and for UPDRS II + III −7.1 and −8.8. MCID for “OFF” time (pramipexole ER, pramipexole IR) was −1.0 and −1.3 hours. Conclusions. A range of MCIDs is emerging in the PD literature that provides the basis for power calculations and interpretation of clinical trials.http://dx.doi.org/10.1155/2014/467131
spellingShingle Robert A. Hauser
Mark Forrest Gordon
Yoshikuni Mizuno
Werner Poewe
Paolo Barone
Anthony H. Schapira
Olivier Rascol
Catherine Debieuvre
Mandy Fräßdorf
Minimal Clinically Important Difference in Parkinson’s Disease as Assessed in Pivotal Trials of Pramipexole Extended Release
Parkinson's Disease
title Minimal Clinically Important Difference in Parkinson’s Disease as Assessed in Pivotal Trials of Pramipexole Extended Release
title_full Minimal Clinically Important Difference in Parkinson’s Disease as Assessed in Pivotal Trials of Pramipexole Extended Release
title_fullStr Minimal Clinically Important Difference in Parkinson’s Disease as Assessed in Pivotal Trials of Pramipexole Extended Release
title_full_unstemmed Minimal Clinically Important Difference in Parkinson’s Disease as Assessed in Pivotal Trials of Pramipexole Extended Release
title_short Minimal Clinically Important Difference in Parkinson’s Disease as Assessed in Pivotal Trials of Pramipexole Extended Release
title_sort minimal clinically important difference in parkinson s disease as assessed in pivotal trials of pramipexole extended release
url http://dx.doi.org/10.1155/2014/467131
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