Movement Disorders (revue)

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Determination of Minimal Clinically Important Change in Early and Advanced Parkinson's Disease

Identifieur interne : 000612 ( PascalFrancis/Corpus ); précédent : 000611; suivant : 000613

Determination of Minimal Clinically Important Change in Early and Advanced Parkinson's Disease

Auteurs : Robert A. Hauser ; Peggy Auinger

Source :

RBID : Pascal:11-0228426

Descripteurs français

English descriptors

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.

Notice en format standard (ISO 2709)

Pour connaître la documentation sur le format Inist Standard.

pA  
A01 01  1    @0 0885-3185
A03   1    @0 Mov. disord.
A05       @2 26
A06       @2 5
A08 01  1  ENG  @1 Determination of Minimal Clinically Important Change in Early and Advanced Parkinson's Disease
A11 01  1    @1 HAUSER (Robert A.)
A11 02  1    @1 AUINGER (Peggy)
A14 01      @1 Departments of Neurology, Molecular Pharmacology, and Physiology, University of South Florida @2 Tampa, Florida @3 USA @Z 1 aut.
A14 02      @1 Department of Neurology, Center for Human Experimental Therapeutics, University of Rochester School of Medicine and Dentistry @2 Rochester, New York @3 USA @Z 2 aut.
A20       @1 813-818
A21       @1 2011
A23 01      @0 ENG
A43 01      @1 INIST @2 20953 @5 354000189767620070
A44       @0 0000 @1 © 2011 INIST-CNRS. All rights reserved.
A45       @0 15 ref.
A47 01  1    @0 11-0228426
A60       @1 P
A61       @0 A
A64 01  1    @0 Movement disorders
A66 01      @0 USA
C01 01    ENG  @0 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.
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C03 01  X  FRE  @0 Maladie de Parkinson @2 NM @5 01
C03 01  X  ENG  @0 Parkinson disease @2 NM @5 01
C03 01  X  SPA  @0 Parkinson enfermedad @2 NM @5 01
C03 02  X  FRE  @0 Pathologie du système nerveux @5 02
C03 02  X  ENG  @0 Nervous system diseases @5 02
C03 02  X  SPA  @0 Sistema nervioso patología @5 02
C03 03  X  FRE  @0 Stade avancé @5 09
C03 03  X  ENG  @0 Advanced stage @5 09
C03 03  X  SPA  @0 Estadio avanzado @5 09
C03 04  X  FRE  @0 Traitement @5 10
C03 04  X  ENG  @0 Treatment @5 10
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C03 05  X  FRE  @0 Essai clinique @5 11
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C03 05  X  SPA  @0 Ensayo clínico @5 11
C07 01  X  FRE  @0 Pathologie de l'encéphale @5 37
C07 01  X  ENG  @0 Cerebral disorder @5 37
C07 01  X  SPA  @0 Encéfalo patología @5 37
C07 02  X  FRE  @0 Syndrome extrapyramidal @5 38
C07 02  X  ENG  @0 Extrapyramidal syndrome @5 38
C07 02  X  SPA  @0 Extrapiramidal síndrome @5 38
C07 03  X  FRE  @0 Maladie dégénérative @5 39
C07 03  X  ENG  @0 Degenerative disease @5 39
C07 03  X  SPA  @0 Enfermedad degenerativa @5 39
C07 04  X  FRE  @0 Pathologie du système nerveux central @5 40
C07 04  X  ENG  @0 Central nervous system disease @5 40
C07 04  X  SPA  @0 Sistema nervosio central patología @5 40
N21       @1 150
N44 01      @1 OTO
N82       @1 OTO

Format Inist (serveur)

NO : PASCAL 11-0228426 INIST
ET : Determination of Minimal Clinically Important Change in Early and Advanced Parkinson's Disease
AU : HAUSER (Robert A.); AUINGER (Peggy)
AF : Departments of Neurology, Molecular Pharmacology, and Physiology, University of South Florida/Tampa, Florida/Etats-Unis (1 aut.); Department of Neurology, Center for Human Experimental Therapeutics, University of Rochester School of Medicine and Dentistry/Rochester, New York/Etats-Unis (2 aut.)
DT : Publication en série; Niveau analytique
SO : Movement disorders; ISSN 0885-3185; Etats-Unis; Da. 2011; Vol. 26; No. 5; Pp. 813-818; Bibl. 15 ref.
LA : Anglais
EA : 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.
CC : 002B17; 002B17G
FD : Maladie de Parkinson; Pathologie du système nerveux; Stade avancé; Traitement; Essai clinique
FG : Pathologie de l'encéphale; Syndrome extrapyramidal; Maladie dégénérative; Pathologie du système nerveux central
ED : Parkinson disease; Nervous system diseases; Advanced stage; Treatment; Clinical trial
EG : Cerebral disorder; Extrapyramidal syndrome; Degenerative disease; Central nervous system disease
SD : Parkinson enfermedad; Sistema nervioso patología; Estadio avanzado; Tratamiento; Ensayo clínico
LO : INIST-20953.354000189767620070
ID : 11-0228426

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Pascal:11-0228426

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