PMID- 37099511
OWN - NLM
STAT- MEDLINE
DCOM- 20230428
LR  - 20230511
IS  - 1932-6203 (Electronic)
IS  - 1932-6203 (Linking)
VI  - 18
IP  - 4
DP  - 2023
TI  - Determining minimal clinically important differences in the North Star Ambulatory 
      Assessment (NSAA) for patients with Duchenne muscular dystrophy.
PG  - e0283669
LID - 10.1371/journal.pone.0283669 [doi]
LID - e0283669
AB  - The North Star ambulatory assessment (NSAA) is a functional motor outcome measure 
      in Duchenne muscular dystrophy (DMD), widely used in clinical trials and natural 
      history studies, as well as in clinical practice. However, little has been 
      reported on the minimal clinically important difference (MCID) of the NSAA. The 
      lack of established MCID estimates for NSAA presents challenges in interpreting 
      the significance of the results of this outcome measure in clinical trials, 
      natural history studies and clinical practice. Combining statistical approaches 
      and patient perspectives, this study estimated MCID for NSAA using 
      distribution-based estimates of 1/3 standard deviation (SD) and standard error of 
      measurement (SEM), an anchor-based approach, with six-minute walk distance (6MWD) 
      as the anchor, and evaluation of patient and parent perception using 
      participant-tailored questionnaires. The MCID for NSAA in boys with DMD aged 7 to 
      10 years based on 1/3 SD ranged from 2.3-2.9 points, and that on SEM ranged from 
      2.9-3.5 points. Anchored on the 6MWD, the MCID for NSAA was estimated as 3.5 
      points. When the impact on functional abilities was considered using participant 
      response questionnaires, patients and parent perceived a complete loss of 
      function in a single item or deterioration of function in one to two items of the 
      assessment as an important change. Our study examines MCID estimates for total 
      NSAA scores using multiple approaches, including the impact of patient and parent 
      perspective on within scale changes in items based on complete loss of function 
      and deterioration of function, and provides new insight on evaluation of 
      differences in these widely used outcome measure in DMD.
CI  - Copyright: (c) 2023 Ayyar Gupta et al. This is an open access article distributed 
      under the terms of the Creative Commons Attribution License, which permits 
      unrestricted use, distribution, and reproduction in any medium, provided the 
      original author and source are credited.
FAU - Ayyar Gupta, Vandana
AU  - Ayyar Gupta V
AUID- ORCID: 0000-0003-3022-4307
AD  - UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, 
      Great Ormond Street Hospital for Children, London, United Kingdom.
FAU - Pitchforth, Jacqueline M
AU  - Pitchforth JM
AD  - UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, 
      Great Ormond Street Hospital for Children, London, United Kingdom.
FAU - Domingos, Joana
AU  - Domingos J
AD  - UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, 
      Great Ormond Street Hospital for Children, London, United Kingdom.
FAU - Ridout, Deborah
AU  - Ridout D
AD  - Population, Policy & Practice Research and Teaching Department, UCL Great Ormond 
      Street (GOS) Institute of Child Health, London, United Kingdom.
AD  - NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United 
      Kingdom.
FAU - Iodice, Mario
AU  - Iodice M
AD  - UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, 
      Great Ormond Street Hospital for Children, London, United Kingdom.
FAU - Rye, Catherine
AU  - Rye C
AD  - UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, 
      Great Ormond Street Hospital for Children, London, United Kingdom.
FAU - Chesshyre, Mary
AU  - Chesshyre M
AD  - UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, 
      Great Ormond Street Hospital for Children, London, United Kingdom.
FAU - Wolfe, Amy
AU  - Wolfe A
AD  - UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, 
      Great Ormond Street Hospital for Children, London, United Kingdom.
FAU - Selby, Victoria
AU  - Selby V
AD  - UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, 
      Great Ormond Street Hospital for Children, London, United Kingdom.
FAU - Mayhew, Anna
AU  - Mayhew A
AD  - The John Walton Muscular Dystrophy Research Centre, Translational and Clinical 
      Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation 
      Trust, Newcastle upon Tyne, United Kingdom.
FAU - Mazzone, Elena S
AU  - Mazzone ES
AD  - Child Neurology Unite Centro Nemo, IRCCS Fondazione Policlinico Gemelli, 
      Universita Cattolica del Sacro Cuore, Rome, Italy.
FAU - Ricotti, Valeria
AU  - Ricotti V
AD  - UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, 
      Great Ormond Street Hospital for Children, London, United Kingdom.
AD  - NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United 
      Kingdom.
FAU - Hogrel, Jean-Yves
AU  - Hogrel JY
AD  - Institute of Myology, Paris, France.
FAU - Niks, Erik H
AU  - Niks EH
AUID- ORCID: 0000-0001-5892-5143
AD  - Department of Neurology, Leiden University Medical Center, Leiden, The 
      Netherlands.
AD  - European Reference Network for Rare Neuromuscular Diseases - ERN EURO NMD.
FAU - de Groot, Imelda
AU  - de Groot I
AD  - Department of Rehabilitation, Donders Center for Medical Neuroscience, Radboud 
      University Medical Center, Nijmegen, The Netherlands.
FAU - Servais, Laurent
AU  - Servais L
AD  - Institute of Myology, Paris, France.
AD  - Division of Child Neurology, Reference Center for Neuromuscular Disease, Centre 
      Hospitalier Regional de References des Maladies Neuromusculaires, Department of 
      Paediatrics, University Hospital Liege & University of La Citadelle, Liege, 
      Belgium.
AD  - Department of Paediatrics, MDUK Neuromuscular Center, University of Oxford, 
      Oxford, United Kingdom.
FAU - Straub, Volker
AU  - Straub V
AUID- ORCID: 0000-0001-9046-3540
AD  - The John Walton Muscular Dystrophy Research Centre, Translational and Clinical 
      Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation 
      Trust, Newcastle upon Tyne, United Kingdom.
FAU - Mercuri, Eugenio
AU  - Mercuri E
AD  - Child Neurology Unite Centro Nemo, IRCCS Fondazione Policlinico Gemelli, 
      Universita Cattolica del Sacro Cuore, Rome, Italy.
AD  - Child Neurology Unit, Universita Cattolica del Sacro Cuore, Rome, Italy.
FAU - Manzur, Adnan Y
AU  - Manzur AY
AD  - UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, 
      Great Ormond Street Hospital for Children, London, United Kingdom.
FAU - Muntoni, Francesco
AU  - Muntoni F
AD  - UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, 
      Great Ormond Street Hospital for Children, London, United Kingdom.
AD  - NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United 
      Kingdom.
CN  - iMDEX Consortium and the U.K. NorthStar Clinical Network
LA  - eng
SI  - ClinicalTrials.gov/NCT02780492
PT  - Journal Article
DEP - 20230426
PL  - United States
TA  - PLoS One
JT  - PloS one
JID - 101285081
SB  - IM
MH  - Male
MH  - Humans
MH  - *Muscular Dystrophy, Duchenne
MH  - Minimal Clinically Important Difference
MH  - Walking/physiology
MH  - Physical Therapy Modalities
MH  - Surveys and Questionnaires
PMC - PMC10132589
COIS- E.H.Niks report grants from Spieren voor Spieren, Duchenne Parent Project, ZonMW, 
      AFM and PPMD. He has been site principal investigator for clinical trials 
      conducted by BioMarin, GSK, Eli Lilly, Santhera Pharmaceuticals, Italfarmaco SpA, 
      Roche Pharma, Reveragen, NS Pharma, Fibrogen, Sarepta, Alexion, Janssen and Argnx 
      outside the submitted work. He also reports ad hoc consultancies for BioMarin, 
      Summit, PTC therapeutics, WAVE Life Sciences, Edgewise, Epirium Bio, Janssen, 
      Sarepta and Regenxbio. All reimbursements were received by the LUMC. No personal 
      financial benefits were received. I.de Groot has received consulting and 
      education fees from PTC Therapeutics, Santhera, Biomarin/Prosensa. J-Y.Hogrel has 
      received consulting fees from Biogen, Sarepta, Minoryx and Roche. L.Servais has 
      received consulting fees from Roche, Biogen, Avexis, Cytokinetics, Sarepta, 
      Biomarin, Santhera, Servier, Biophytis and Dynacure. He is coordinating natural 
      history studies funded by Valerion, Dynacure and Roche. A.Mayhew has received 
      consulting fees from Roche, Novartis (Avexis), Biogen, Rehenxbio, PTC, BMS/Roche, 
      Sarepta, Italfarmaco, Pfizer, Summit, Catabasis, Santhera, Vision, Mallinckrodt, 
      Lysogen, Modis and Wave. V.Straub received speaker honoraria from Sanofi Genzyme 
      and has participated in advisory boards for Audentes Therapeutics, Biogen, 
      AveXis, Pharmaceuticals, Pfizer, Roche, Sanofi Genzyme, Sarepta Therapeutics, 
      Summit Therapeutics and Wave Therapeutics. V.Ricotti is co-founder, EVP, CMO of 
      DiNAQOR, and served as a consultant for Solid Biosciences and Antisense 
      Therapeutics. F.Muntoni reports grants from Sarepta, grants from Wave, grants 
      from PTC Therapeutics, personal fees from Avexis, Roche, Pfizer, Dyne 
      Therapeutics, Sarepta, outside the submitted work. M.Chesshyre has had the costs 
      associated with attending a conference (including travel, accommodation, 
      conference fee, food and drink) funded by PTC Therapeutics. This does not alter 
      our adherence to PLOS ONE policies on sharing data and materials.
EDAT- 2023/04/26 18:42
MHDA- 2023/04/28 06:42
PMCR- 2023/04/26
CRDT- 2023/04/26 13:34
PHST- 2022/06/20 00:00 [received]
PHST- 2023/03/14 00:00 [accepted]
PHST- 2023/04/28 06:42 [medline]
PHST- 2023/04/26 18:42 [pubmed]
PHST- 2023/04/26 13:34 [entrez]
PHST- 2023/04/26 00:00 [pmc-release]
AID - PONE-D-22-17578 [pii]
AID - 10.1371/journal.pone.0283669 [doi]
PST - epublish
SO  - PLoS One. 2023 Apr 26;18(4):e0283669. doi: 10.1371/journal.pone.0283669. 
      eCollection 2023.