PMID- 23674289
OWN - NLM
STAT- MEDLINE
DCOM- 20131111
LR  - 20220318
IS  - 1097-4598 (Electronic)
IS  - 0148-639X (Print)
IS  - 0148-639X (Linking)
VI  - 48
IP  - 3
DP  - 2013 Sep
TI  - The 6-minute walk test and other clinical endpoints in duchenne muscular 
      dystrophy: reliability, concurrent validity, and minimal clinically important 
      differences from a multicenter study.
PG  - 357-68
LID - 10.1002/mus.23905 [doi]
AB  - INTRODUCTION: An international clinical trial enrolled 174 ambulatory males >/=5 
      years old with nonsense mutation Duchenne muscular dystrophy (nmDMD). 
      Pretreatment data provide insight into reliability, concurrent validity, and 
      minimal clinically important differences (MCIDs) of the 6-minute walk test (6MWT) 
      and other endpoints. METHODS: Screening and baseline evaluations included the 
      6-minute walk distance (6MWD), timed function tests (TFTs), quantitative strength 
      by myometry, the PedsQL, heart rate-determined energy expenditure index, and 
      other exploratory endpoints. RESULTS: The 6MWT proved feasible and reliable in a 
      multicenter context. Concurrent validity with other endpoints was excellent. The 
      MCID for 6MWD was 28.5 and 31.7 meters based on 2 statistical distribution 
      methods. CONCLUSIONS: The ratio of MCID to baseline mean is lower for 6MWD than 
      for other endpoints. The 6MWD is an optimal primary endpoint for Duchenne 
      muscular dystrophy (DMD) clinical trials that are focused therapeutically on 
      preservation of ambulation and slowing of disease progression.
CI  - Copyright (c) 2013 Wiley Periodicals, Inc.
FAU - McDonald, Craig M
AU  - McDonald CM
AD  - Department of Physical Medicine and Rehabilitation, Neuromuscular Medicine and 
      Rehabilitation Research Center, University of California Davis School of 
      Medicine, Davis, California, 95817, USA.
FAU - Henricson, Erik K
AU  - Henricson EK
FAU - Abresch, R Ted
AU  - Abresch RT
FAU - Florence, Julaine
AU  - Florence J
FAU - Eagle, Michelle
AU  - Eagle M
FAU - Gappmaier, Eduard
AU  - Gappmaier E
FAU - Glanzman, Allan M
AU  - Glanzman AM
CN  - PTC124-GD-007-DMD Study Group
FAU - Spiegel, Robert
AU  - Spiegel R
FAU - Barth, Jay
AU  - Barth J
FAU - Elfring, Gary
AU  - Elfring G
FAU - Reha, Allen
AU  - Reha A
FAU - Peltz, Stuart W
AU  - Peltz SW
LA  - eng
GR  - MR/K000608/1/MRC_/Medical Research Council/United Kingdom
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
PT  - Research Support, U.S. Gov't, Non-P.H.S.
DEP - 20130717
PL  - United States
TA  - Muscle Nerve
JT  - Muscle & nerve
JID - 7803146
RN  - 0 (Oxadiazoles)
RN  - K16AME9I3V (ataluren)
SB  - IM
CIN - Muscle Nerve. 2013 Sep;48(3):315-7. PMID: 24038058
MH  - Disease Progression
MH  - *Exercise Test
MH  - Heart Rate/drug effects/physiology
MH  - Humans
MH  - Male
MH  - Muscular Dystrophy, Duchenne/diagnosis/drug therapy/*physiopathology
MH  - *Outcome Assessment, Health Care
MH  - Oxadiazoles/therapeutic use
MH  - Reproducibility of Results
MH  - Time Factors
MH  - Walking/*physiology
PMC - PMC3826053
OTO - NOTNLM
OT  - 6-minute walk test
OT  - Duchenne muscular dystrophy
OT  - PedsQL
OT  - ambulation
OT  - energy expenditure index
OT  - muscular dystrophy
OT  - myometry
OT  - natural history
OT  - timed function test
FIR - Ryan, Monique
IR  - Ryan M
FIR - Jones, Kristi
IR  - Jones K
FIR - Goemans, Nathalie
IR  - Goemans N
FIR - Campbell, Craig
IR  - Campbell C
FIR - Mah, Jean
IR  - Mah J
FIR - Selby, Kathryn
IR  - Selby K
FIR - Voit, Thomas
IR  - Voit T
FIR - Chabrol, Brigitte
IR  - Chabrol B
FIR - Pereon, Yann
IR  - Pereon Y
FIR - Schara, Ulrike
IR  - Schara U
FIR - Kirschner, Janberndt
IR  - Kirschner J
FIR - Bertini, Enrico
IR  - Bertini E
FIR - Mercuri, Eugenio
IR  - Mercuri E
FIR - Comi, Giacomo
IR  - Comi G
FIR - Nevo, Yoram
IR  - Nevo Y
FIR - Vilchez, Juan
IR  - Vilchez J
FIR - Colomer, Jaume
IR  - Colomer J
FIR - Children, Sylvia
IR  - Children S
FIR - Tulinius, Mar
IR  - Tulinius M
FIR - Sejersen, Thomas
IR  - Sejersen T
FIR - Bushby, Katherine
IR  - Bushby K
FIR - Muntoni, Francesco
IR  - Muntoni F
FIR - Quinlivan, Rosaline Christina Mary
IR  - Quinlivan RC
FIR - Wong, Brenda
IR  - Wong B
FIR - Finkel, Richard S
IR  - Finkel RS
FIR - Sampson, Jacinda B
IR  - Sampson JB
FIR - Flanigan, Kevin M
IR  - Flanigan KM
FIR - Butterfield, Russell
IR  - Butterfield R
FIR - Day, John W
IR  - Day JW
FIR - Mathews, Katherine
IR  - Mathews K
FIR - Darras, Basil T
IR  - Darras BT
FIR - Apkon, Susan D
IR  - Apkon SD
FIR - Parsons, Julie
IR  - Parsons J
FIR - Barohn, Richard
IR  - Barohn R
FIR - Connolly, Anne
IR  - Connolly A
FIR - Iannaccone, Susan
IR  - Iannaccone S
FIR - Sproule, Douglas M
IR  - Sproule DM
FIR - Kaufman, Petra
IR  - Kaufman P
FIR - Han, Jay J
IR  - Han JJ
FIR - Joyce, Nanette C
IR  - Joyce NC
FIR - Renfroe, J Ben
IR  - Renfroe J
FIR - Russman, Barry S
IR  - Russman BS
FIR - Burns-Wechsler, Stephanie
IR  - Burns-Wechsler S
FIR - Moore, Steven A
IR  - Moore SA
FIR - Sweeney, H Lee
IR  - Sweeney HL
FIR - Coleman, Kim
IR  - Coleman K
EDAT- 2013/05/16 06:00
MHDA- 2013/11/12 06:00
CRDT- 2013/05/16 06:00
PHST- 2013/05/07 00:00 [accepted]
PHST- 2013/05/16 06:00 [entrez]
PHST- 2013/05/16 06:00 [pubmed]
PHST- 2013/11/12 06:00 [medline]
AID - 10.1002/mus.23905 [doi]
PST - ppublish
SO  - Muscle Nerve. 2013 Sep;48(3):357-68. doi: 10.1002/mus.23905. Epub 2013 Jul 17.