Clinical outcome study for dysferlinopathy: Sensitivity of outcome measures over one-year

Moore, Ursula; James, M.; Mayhew, A.; Jacobs, Marni; Feng, June; Harris, Elizabeth; Fernández‐Torrón, Roberto; Bettinson, K.; Hilsden, H.; Rufibach, Laura; Cnaan, Avital; Straub, V.; Bushby, Kate

doi:10.1016/s0960-8966(17)30241-9

Cited by 1 publication

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“…The use of MFM was consistent with the administration methods developed by MFM study team. Misuses were mainly due to the administration to the incorrect age group, use of MFM32 in younger individuals [14,33] or the use of MFM20 in individuals older individuals [34]. We insist on the necessity to perform a total MFM including its 3 domains so its measurement properties are verified.…”

Section: Discussionmentioning

confidence: 99%

“…Schreiber et al showed a large responsiveness for MFM32 total score at 2 years (n = 74) [12]. For dysferlinopathy, mean change of MFM20 in individuals older than 6 years old at 1 year (median (range)) was evaluated at -1 (-13.0-6.0, p < 0.001) with showing low responsiveness for MFM total score (n = 160) [33]. One study about CMT using adequately MFM32 and MFM20 found no significant change at 1 except for CMT2 individuals (large responsiveness) [29].…”

Section: Natural History Studies: Responsiveness and Minimal Clinical...mentioning

confidence: 98%

“…Four studies adequately used MFM20 under 6 years old and MFM32 after 6 years old [29][30][31][32]. One study inappropriately used MFM20 for individuals older than 12 years old to reduce participant fatigue and duplication [33]. One study used MFM20 for patients older than 6 years old with no justification.…”

Section: Natural History Studies: Responsiveness and Minimal Clinical...mentioning

confidence: 99%

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

Ribault

Rippert

Jain

et al. 2023

JND

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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.

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Section: Discussionmentioning

confidence: 99%

Section: Natural History Studies: Responsiveness and Minimal Clinical...mentioning

confidence: 98%

Section: Natural History Studies: Responsiveness and Minimal Clinical...mentioning

confidence: 99%

See 1 more Smart Citation

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

Ribault

Rippert

Jain

et al. 2023

JND

View full text Add to dashboard Cite

show abstract

Clinical outcome study for dysferlinopathy: Sensitivity of outcome measures over one-year

Cited by 1 publication

References 0 publications

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

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

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