Evidence map›Paper›PMID 33166523›Full record

Observational studyArchives of physical medicine and rehabilitation2021

Responsiveness and Minimal Clinically Important Difference of the Motor Function Measure in Collagen VI-Related Dystrophies and Laminin Alpha2-Related Muscular Dystrophy.

Laure Le Goff, Katherine G Meilleur, Gina Norato, Pascal Rippert, Minal Jain, Margaret Fink, A Reghan Foley, Melissa Waite, Sandra Donkervoort, Carsten G Bönnemann and 1 more

Open access · greenAbstract readObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in Archives of physical medicine and rehabilitation, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed, 1 pooled it
0.6field-weighted citation impact, top 34% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

7 citing papers in PubMed, 1 synthesis or guideline pooled it, 16 citations in OpenAlex.

  1. Pooled it
  2. Current clinical applications of AAV-mediated gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  3. Article
  4. Article
  5. Observational
  6. Article
  7. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors at 4 institutions in 2 countries.

Laure Le GoffDepartment of Pediatric Physical Medicine and Rehabilitation, Hospices Civils de Lyon, Bron, France. Electronic address: laure.le-goff@chu-lyon.fr.
Katherine G MeilleurNational Institute of Nursing Research, National Institutes of Health, Bethesda, MD; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD.
Gina NoratoOffice of Biostatistics, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD.
Pascal RippertPublic Health Center, Research and Clinical Epidemiology Department, Hospices Civils de Lyon, Lyon, France.
Minal JainMark O. Hatfield Clinical Research Center, National Institutes of Health, Bethesda, MD.
Margaret FinkNeuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD.
A Reghan FoleyNeuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD.
Melissa WaiteMark O. Hatfield Clinical Research Center, National Institutes of Health, Bethesda, MD.
Sandra DonkervoortNeuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD.
Carsten G BönnemannNeuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD.
Carole VuillerotDepartment of Pediatric Physical Medicine and Rehabilitation, Hospices Civils de Lyon, Bron, France; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Neuromyogen Institute, CNRS UMR 5310-INSERM, University of Lyon, Lyon, France; University of Lyon 1, F-69100, Villeurbanne, France.
National Institutes of Health · USHospices Civils de Lyon · FRNational Institutes of Health Clinical Center · USUniversité Claude Bernard Lyon 1 · FR

Funding

Intramural NIH HHS Z99 CL999999
6 · The paper itself

Abstract

objectivesTo investigate the responsiveness of the motor function measure (MFM) and determine the minimal clinically important difference (MCID) in individuals with 2 common types of congenital muscular dystrophy (CMD).

designObservational, prospective, single center, cohort study.

settingNational Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH).

participantsIndividuals (N=44) with collagen VI-related dystrophies (COL6-RD, n=23) and 21 individuals laminin alpha2-related muscular dystrophy (LAMA2-RD, n=21) enrolled in a 4-year longitudinal natural history study.

interventionsNot applicable.

main outcome measuresResponsiveness of the MFM-32 and the Rasch-scaled MFM-25 and the MCID of the MFM-32 determined from a patient-reported anchor with 2 different methods, within-patient and between-patient.

resultsThe original MFM-32 and Rasch-scaled MFM-25 performed similarly overall in both the COL6-RD and LAMA2-RD populations, with all subscores (D1, standing and transfers; D2, axial and proximal; D3, distal) showing a significant decrease over time, except MFM D1 and D3 for LAMA2-RD. The MFM D1 subscore was the most sensitive to change for ambulant individuals, whereas the MFM D2 subscore was the most sensitive to change for nonambulant individuals. The MCID for the MFM-32 total score was calculated as 2.5 and 3.9 percentage points according to 2 different methods.

conclusionsThe MFM showed strong responsiveness in individuals with LAMA2-RD and COL6-RD. Because a floor effect was identified more prominently with the Rasch-Scaled MFM-25, the use of the original MFM-32 as a quantitative variable with the assumption of scale linearity appears to be a good compromise. When designing clinical trials in congenital muscular dystrophies, the use of MCID for MFM should be considered to determine if a given intervention effects show not only a statistically significant change but also a clinically meaningful change.

Indexed as

Disability EvaluationMinimal Clinically Important DifferenceAdolescentAdultChildChild, PreschoolCohort StudiesFemaleHumansLongitudinal StudiesMaleMotor ActivityMuscular DystrophiesProspective StudiesYoung AdultDisability evaluationMuscular dystrophiesRehabilitation

Identifiers

PMID33166523
PMCPMC10363856
OpenAlexW3096783064

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.