Psychometric Characteristics of the Motor Function Measure in Neuromuscular Diseases: A Systematic Review1.

Outcome assessment natural history neuromuscular diseases systematic review validation study

Journal

Journal of neuromuscular diseases
ISSN: 2214-3602
Titre abrégé: J Neuromuscul Dis
Pays: Netherlands
ID NLM: 101649948

Informations de publication

Date de publication:
2023
Historique:
medline: 9 5 2023
pubmed: 1 5 2023
entrez: 1 5 2023
Statut: ppublish

Résumé

Recent pharmaceutical breakthroughs in neuromuscular diseases may considerably change the prognosis and natural history these diseases. The ability to measure clinically relevant outcomes such as motor function is critical for the assessment of therapeutics and the follow up of individuals. The Motor Function Measure (MFM) is a quantitative scale designed to measure motor function in adult and children with neuromuscular disease (NMD). The objective of this study is to assess the quality and level of evidence of the MFM's published measurement properties by completing a systematic review of the validation and responsiveness studies of the MFM20 (a 20-item version of MFM adapted for children 2 to 6 years of age) and the MFM32 (the original 32 item version), in all NMDs and in specific diseases. A search for MFM responsiveness and MFM validation studies was completed in February 2023 in EMBASE, MEDLINE, SCOPUS and Web of Science databases. The PRISMA guidelines and the COSMIN manual for systematic reviews were followed for databases searches, articles screening and selection, study quality and measurement properties evaluation. 49 studies were included in analysis. In studies including individuals with all NMDs, MFM's internal consistency, reliability, convergent validity, construct validity and responsiveness were rated as sufficient with a high quality of evidence. Structural validity was rated sufficient with a moderate quality of evidence In SMA in particular, MFM's reliability, internal consistency, convergent validity, discriminant validity and responsiveness are sufficient with a high quality of evidence. More studies would be required to assess specific measurement properties in different diseases. MFM32's minimal clinically relevant difference has been defined between 2 and 6%. MFM's structural validity, internal consistency, reliability, construct validity, convergent validity and responsiveness have been verified with moderate to high level of evidence.

Sections du résumé

BACKGROUND BACKGROUND
Recent pharmaceutical breakthroughs in neuromuscular diseases may considerably change the prognosis and natural history these diseases. The ability to measure clinically relevant outcomes such as motor function is critical for the assessment of therapeutics and the follow up of individuals. The Motor Function Measure (MFM) is a quantitative scale designed to measure motor function in adult and children with neuromuscular disease (NMD).
OBJECTIVE OBJECTIVE
The objective of this study is to assess the quality and level of evidence of the MFM's published measurement properties by completing a systematic review of the validation and responsiveness studies of the MFM20 (a 20-item version of MFM adapted for children 2 to 6 years of age) and the MFM32 (the original 32 item version), in all NMDs and in specific diseases.
METHODS METHODS
A search for MFM responsiveness and MFM validation studies was completed in February 2023 in EMBASE, MEDLINE, SCOPUS and Web of Science databases. The PRISMA guidelines and the COSMIN manual for systematic reviews were followed for databases searches, articles screening and selection, study quality and measurement properties evaluation.
RESULTS RESULTS
49 studies were included in analysis. In studies including individuals with all NMDs, MFM's internal consistency, reliability, convergent validity, construct validity and responsiveness were rated as sufficient with a high quality of evidence. Structural validity was rated sufficient with a moderate quality of evidence In SMA in particular, MFM's reliability, internal consistency, convergent validity, discriminant validity and responsiveness are sufficient with a high quality of evidence. More studies would be required to assess specific measurement properties in different diseases. MFM32's minimal clinically relevant difference has been defined between 2 and 6%.
CONCLUSION CONCLUSIONS
MFM's structural validity, internal consistency, reliability, construct validity, convergent validity and responsiveness have been verified with moderate to high level of evidence.

Identifiants

pubmed: 37125561
pii: JND230001
doi: 10.3233/JND-230001
pmc: PMC10408216
mid: NIHMS1912257
doi:

Types de publication

Systematic Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

301-314

Subventions

Organisme : Intramural NIH HHS
ID : Z99 CL999999
Pays : United States

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Auteurs

Shams Ribault (S)

Hospices Civils de Lyon, Hôpital Femme Mère Enfant, Service de médecine physique et réadaptation pédiatrique, Bron cedex F-69677.
Pathophysiology and Genetics of Neuron and Muscle, CNRS UMR 5261, INSERM U1315, Université Lyon1, Faculté de Médecine Lyon Est, Lyon, France.

Pascal Rippert (P)

Hospices Civils de Lyon, Hôpital Femme Mère Enfant, Service de médecine physique et réadaptation pédiatrique, Bron cedex F-69677.
Hospices Civils de Lyon, Pôle Santé Publique, Service Recherche et Epidémiologie Clinique, Lyon F-69003.

Minal Jain (M)

Rehabilitation Medicine Department, National Institutes of Health, Bethesda, MD, US.

Laure Le Goff (L)

Hospices Civils de Lyon, Hôpital Femme Mère Enfant, Service de médecine physique et réadaptation pédiatrique, Bron cedex F-69677.

Dominique Vincent Genod (DV)

Hospices Civils de Lyon, Hôpital Femme Mère Enfant, Service de médecine physique et réadaptation pédiatrique, Bron cedex F-69677.

Aurélie Barriere (A)

Hospices Civils de Lyon, Hôpital Femme Mère Enfant, Service de médecine physique et réadaptation pédiatrique, Bron cedex F-69677.

Anne Berruyer (A)

Hospices Civils de Lyon, Hôpital Femme Mère Enfant, Service de médecine physique et réadaptation pédiatrique, Bron cedex F-69677.

Camille Garde (C)

Hospices Civils de Lyon, Hôpital Femme Mère Enfant, Service de médecine physique et réadaptation pédiatrique, Bron cedex F-69677.

Marie Tinat (M)

Hospices Civils de Lyon, Hôpital Femme Mère Enfant, Service de médecine physique et réadaptation pédiatrique, Bron cedex F-69677.

Christelle Pons (C)

SSR pédiatrique, fondation Ildys, rue Alain-Colas, 29200 Brest, France; Service de médecine physique et de réadaptation, CHRU de Brest, Brest, France; Laboratoire de traitement de l'information médicale (LaTIM), inserm U1101, université Bretagne Occidentale, France.

Carole Vuillerot (C)

Hospices Civils de Lyon, Hôpital Femme Mère Enfant, Service de médecine physique et réadaptation pédiatrique, Bron cedex F-69677.
Pathophysiology and Genetics of Neuron and Muscle, CNRS UMR 5261, INSERM U1315, Université Lyon1, Faculté de Médecine Lyon Est, Lyon, France.

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Classifications MeSH