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

Article type: Systematic Review

Authors: Ribault, Shams^{a; b; *} | Rippert, Pascal^{a; c} | Jain, Minal^d | Le Goff, Laure^a | Genod, Dominique Vincent^a | Barriere, Aurélie^a | Berruyer, Anne^a | Garde, Camille^a | Tinat, Marie^a | Pons, Christelle^e | Vuillerot, Carole^{a; b}

Affiliations: [a] Hospices Civils de Lyon, Hôpital Femme Mère Enfant, Service de médecine physique et réadaptation pédiatrique, Bron cedex F-69677 | [b] Pathophysiology and Genetics of Neuron and Muscle, CNRS UMR 5261, INSERM U1315, Université Lyon1, Faculté de Médecine Lyon Est, Lyon, France | [c] Hospices Civils de Lyon, Pôle Santé Publique, Service Recherche et Epidémiologie Clinique, Lyon F-69003 | [d] Rehabilitation Medicine Department, National Institutes of Health, Bethesda, MD, US | [e] 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

Correspondence: [*] Correspondence to: Shams Ribault, Hôpital Femme Mère Enfant, Service l’Escale – Médecine Physique et de Réadaptation Pédiatrique 59 Boulevard Pinel, 69500 Bron, France. Tel.: +33 4 72 12 95 04; Fax: +33 4 27 85 67 90; E-mail: [email protected].

Note: [1] The study was registered on PROSPERO (registration number: CRD42021261491)

Abstract: 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: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: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: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:MFM’s structural validity, internal consistency, reliability, construct validity, convergent validity and responsiveness have been verified with moderate to high level of evidence.

Keywords: Outcome assessment, validation study, natural history, neuromuscular diseases, systematic review

DOI: 10.3233/JND-230001

Journal: Journal of Neuromuscular Diseases, vol. 10, no. 3, pp. 301-314, 2023