PMID- 34151852
OWN - NLM
STAT- MEDLINE
DCOM- 20220203
LR  - 20240404
IS  - 2214-3602 (Electronic)
IS  - 2214-3599 (Print)
IS  - 2214-3599 (Linking)
VI  - 8
IP  - 6
DP  - 2021
TI  - The Minimal Clinical Important Difference (MCID) in Annual Rate of Change of 
      Timed Function Tests in Boys with DMD.
PG  - 939-948
LID - 10.3233/JND-210646 [doi]
AB  - BACKGROUND: Duchenne muscular dystrophy (DMD) is a rare x-linked recessive 
      genetic disorder affecting 1 in every 5000-10000 [1, 2]. This disease leads to a 
      variable but progressive sequential pattern of muscle weakness that eventually 
      causes loss of important functional milestones such as the ability to walk. With 
      promising drugs in development to ameliorate the effects of muscle weakness, 
      these treatments must be associated with a clinically meaningful functional 
      change. OBJECTIVE: The objective of this analysis is to determine both 
      distribution, minimal detectable change (MDC), and anchor-based, minimal 
      clinically important difference, (MCID) of 12 month change values in standardized 
      time function tests (TFT) used to monitor disease progression in DMD. METHOD: 
      This is a retrospective analysis of prospectively collected data from a 
      multi-center prospective natural history study with the Cooperative International 
      Neuromuscular Research Group (CINRG). This study calculated MDC and MCID values 
      for 3 commonly used timed function tests typically used to monitor disease 
      progression; supine to stand (STS), 10 meter walk/run (10MWT), and 4 stair climb 
      (4SC). MDC used standard error of measurement (SEM) while MCID measurements used 
      the Vignos scale as an anchor to determine clinical change in functional status. 
      RESULTS: All 3 TFT were significantly important clinical endpoints to detect MDC 
      and MCID changes. MDC and MCID 12 month changes were significant in 10MWT 
      (-0.138, -0.212), Supine to Stand (-0.026, -0.023) and 4 stair climb (-0.034, 
      -0.035) with an effect size greater or close to 0.2. CONCLUSION: The 3 TFT are 
      clinically meaningful endpoints used to establish change in DMD. MCID values were 
      higher than MDC values indicating that an anchor-based approach using Vignos as a 
      clinically meaningful loss of lower extremity abilities is appropriate to assess 
      change in boys with DMD.
FAU - Duong, Tina
AU  - Duong T
AD  - Department of Neurology, Stanford University School of Medicine, Stanford, CA, 
      USA.
FAU - Canbek, Jennifer
AU  - Canbek J
AD  - Physical Therapy Department, Nova Southeastern University, Fort Lauderdale, FL, 
      USA.
FAU - Birkmeier, Marisa
AU  - Birkmeier M
AD  - Department of Health, Human Function, and Rehabilitation Sciences, The George 
      Washington University School of Medicine and Health Sciences, Washington, DC, 
      USA.
FAU - Nelson, Leslie
AU  - Nelson L
AD  - Department of Physical Therapy, University of Texas Southwestern Medical Center, 
      Dallas, TX, USA.
FAU - Siener, Catherine
AU  - Siener C
AD  - Department of Neurology, Washington University, St. Louis, MO, USA.
FAU - Fernandez-Fernandez, Alicia
AU  - Fernandez-Fernandez A
AD  - Physical Therapy Department, Nova Southeastern University, Fort Lauderdale, FL, 
      USA.
FAU - Henricson, Erik
AU  - Henricson E
AD  - University of California Davis Health, Department of Physical Medicine and 
      Rehabilitation, Sacramento CA, USA.
FAU - McDonald, Craig M
AU  - McDonald CM
AD  - University of California Davis Health, Department of Physical Medicine and 
      Rehabilitation, Sacramento CA, USA.
FAU - Gordish-Dressman, Heather
AU  - Gordish-Dressman H
AD  - Children's National Medical Center, Department of Biostatistics, Washington DC, 
      USA.
CN  - CINRG-DNHS Investigators
LA  - eng
PT  - Journal Article
PT  - Multicenter Study
PL  - Netherlands
TA  - J Neuromuscul Dis
JT  - Journal of neuromuscular diseases
JID - 101649948
SB  - IM
MH  - Child
MH  - Disease Progression
MH  - Humans
MH  - Male
MH  - *Minimal Clinically Important Difference
MH  - Muscular Dystrophy, Duchenne/*physiopathology
MH  - Prospective Studies
MH  - Retrospective Studies
PMC - PMC8673528
OTO - NOTNLM
OT  - Duchenne muscular dystrophy
OT  - MCID
OT  - time function tests
COIS- TD: Serves on advisory boards or consultant for Pfizer, Inc., Dyne Therapeutics, 
      F. Hoffman-La Roche, LTD, Sarepta Therapeutics, Edgewise Therapeutics. JC: none. 
      MB: none. LN: none. CS: none. AF: none. EH: Serves on advisory boards for 
      Sarepta, Inc. and Santhera Pharmaceuticals. CM: Serves as a consultant on 
      clinical trials of DMD and serves on advisory boards for Astellas, Capricor 
      Therapeutics, Catabasis, Edgewise Therapeutics, Epirium Bio (formerly Cardero 
      Therapeutics), FibroGen, Italfarmaco, Pfizer, PTC Therapeutics, Roche, 
      SantheraPharmaceuticals and Sarepta Therapeutics. He has received research 
      support for clinical trials from Capricor Therapeutics, Catabasis, Italfarmaco, 
      Pfizer, PTC Therapeutics, Santhera Pharmaceuticals and Sarepta Therapeutics. HG: 
      none.
EDAT- 2021/06/22 06:00
MHDA- 2022/02/04 06:00
PMCR- 2021/12/15
CRDT- 2021/06/21 08:51
PHST- 2021/06/22 06:00 [pubmed]
PHST- 2022/02/04 06:00 [medline]
PHST- 2021/06/21 08:51 [entrez]
PHST- 2021/12/15 00:00 [pmc-release]
AID - JND210646 [pii]
AID - 10.3233/JND-210646 [doi]
PST - ppublish
SO  - J Neuromuscul Dis. 2021;8(6):939-948. doi: 10.3233/JND-210646.