PMID- 23770101
OWN - NLM
STAT- MEDLINE
DCOM- 20140305
LR  - 20220318
IS  - 1873-2364 (Electronic)
IS  - 0960-8966 (Linking)
VI  - 23
IP  - 8
DP  - 2013 Aug
TI  - Ambulatory capacity and disease progression as measured by the 
      6-minute-walk-distance in Duchenne muscular dystrophy subjects on daily 
      corticosteroids.
PG  - 618-23
LID - S0960-8966(13)00148-X [pii]
LID - 10.1016/j.nmd.2013.05.006 [doi]
AB  - In order to understand contemporary natural history of Duchenne muscular 
      dystrophy (DMD), we report 6-minute walk distance (6MWD) and its change over time 
      from a large single centre population of corticosteroid treated DMD boys. 
      Sixty-five boys on daily corticosteroid treatment were identified with a mean 
      (SD) age of 9.5 (2.3) years at first observation. 6MWD was described for 1year 
      age groupings. In addition, changes in 6MWD at 1, 1.5 and 2years (+/-12weeks) of 
      follow-up were evaluated. The same evaluations were applied to 6MWD data 
      converted to percent predicted values based on the Geiger equation. 6MWD showed 
      an increase from age group 4.5-5.5years to age group 6.5-7.5years, followed by a 
      decline, which became precipitous from 12.5years onwards. From 15.5years, all 
      boys were unable to perform the 6-min test. Changes in 6MWD demonstrated a mean 
      (median, SD) decline of -43 (-14, 90) m at 1year (N=25, mean baseline age 
      9.5years), -64 (-56, 99) m at 1.5years (N=18, mean baseline age 9.6years), -125 
      (-106, 139) m at 2years (N=14, mean baseline age 10.0years). Conversion to 
      percent predicted values showed the same pattern of evolution.This study provides 
      data on the ambulatory capacity and its changes over time in a homogenous cohort 
      of 65 DMD boys on daily corticosteroids. The variability, the age-related aspects 
      and the slope of decline of the 6MWD should be considered in the design and 
      interpretation of therapeutic trials in ambulant DMD patients.
CI  - Copyright (c) 2013 Elsevier B.V. All rights reserved.
FAU - Goemans, Nathalie
AU  - Goemans N
AD  - Child Neurology, University Hospitals Leuven, Leuven, Belgium. 
      nathalie.goemans@uzleuven.be
FAU - van den Hauwe, Marleen
AU  - van den Hauwe M
FAU - Wilson, Rosamund
AU  - Wilson R
FAU - van Impe, Annelies
AU  - van Impe A
FAU - Klingels, Katrijn
AU  - Klingels K
FAU - Buyse, Gunnar
AU  - Buyse G
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20130613
PL  - England
TA  - Neuromuscul Disord
JT  - Neuromuscular disorders : NMD
JID - 9111470
RN  - 0 (Adrenal Cortex Hormones)
SB  - IM
MH  - Adolescent
MH  - Adrenal Cortex Hormones/*therapeutic use
MH  - Child
MH  - Child, Preschool
MH  - Disease Progression
MH  - *Exercise Test
MH  - Female
MH  - Humans
MH  - Longitudinal Studies
MH  - Male
MH  - Muscular Dystrophy, Duchenne/*drug therapy/*physiopathology
MH  - *Outcome Assessment, Health Care
MH  - Retrospective Studies
MH  - Time Factors
OTO - NOTNLM
OT  - 6-Min walk test
OT  - Ambulatory capacity
OT  - Corticosteroids
OT  - Disease evolution
OT  - Duchenne muscular dystrophy
OT  - Variability
EDAT- 2013/06/19 06:00
MHDA- 2014/03/07 06:00
CRDT- 2013/06/18 06:00
PHST- 2013/03/15 00:00 [received]
PHST- 2013/05/03 00:00 [revised]
PHST- 2013/05/09 00:00 [accepted]
PHST- 2013/06/18 06:00 [entrez]
PHST- 2013/06/19 06:00 [pubmed]
PHST- 2014/03/07 06:00 [medline]
AID - S0960-8966(13)00148-X [pii]
AID - 10.1016/j.nmd.2013.05.006 [doi]
PST - ppublish
SO  - Neuromuscul Disord. 2013 Aug;23(8):618-23. doi: 10.1016/j.nmd.2013.05.006. Epub 
      2013 Jun 13.