Responsiveness of the Motor Function Measure in Patients With Spinal Muscular Atrophy

Vuillerot, Carole; Payán, C.; Iwaz, Jean; Écochard, René; Bérard, C.

doi:10.1016/j.apmr.2013.01.014

Cited by 77 publications

(76 citation statements)

References 31 publications

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“…Vuillerot presented a discussion on the MFM and highlighted how the items captured as part of the domains D1, D2 and D3 are relevant also for SMA patients with types 2 and 3 [36]. The MFM appears to be less appropriate for type 1.…”

Section: Natural History Studies and Relevant Outcome Measuresmentioning

confidence: 95%

209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7–9 November 2014, Heemskerk, The Netherlands

Finkel

Bertini²,

Muntoni

et al. 2015

Neuromuscular Disorders

166

187

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Section: Natural History Studies and Relevant Outcome Measuresmentioning

confidence: 95%

209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7–9 November 2014, Heemskerk, The Netherlands

Finkel

Bertini²,

Muntoni

et al. 2015

Neuromuscular Disorders

166

187

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“…Responsiveness was higher in the months preceding ambulation loss 16 . The authors mentioned that fatigue interfered in testing.…”

mentioning

confidence: 89%

“…Cano et al 28 recommended that the ES should be interpreted with caution and combined with other statistical methods to avoid misinterpretation 28 . Only six studies investigated the responsiveness of one or more tests for neuromuscular diseases 2,16,17,18,19,29 . Most studies used the ES 18 and SRM 2,16,17 in isolation or in combination 19,29 .…”

mentioning

confidence: 99%

How often should sitting and rising from a chair be evaluated in patients with Duchenne muscular dystrophy?

Hukuda

Caromano

Escórcio

et al. 2017

Arq. Neuro-Psiquiatr.

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Objective: To determine how often sitting/rising from a chair should be assessed in Duchenne muscular dystrophy (DMD) patients to avoid redundant/missing data. Methods: Sitting/rising from a chair was evaluated in 26 DMD children (5-12 yrs), in three-month intervals, over twelve months, with the Functional Evaluation Scale (domain sitting/rising from a chair). Scores were compared by effect sizes (ES) and standardized response means (SRM) (responsiveness analysis). Results: Sit-to-stand showed low-to-moderate responsiveness in three-month intervals

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“…The HFMS is validated in children with spinal muscular atrophy (SMA) and functional impairments [12]; the NSAA is validated in ambulatory children with Duchenne muscular dystrophy (DMD) and SMA type III [14,24]. The MFM32, validated in individuals with various neuromuscular disorders, is a scale that spans the spectrum of abilities from non-ambulatory to ambulatory [11,25,26]. Other scales gather information about different aspects of function.…”

Section: Introductionmentioning

confidence: 99%

Results of a two-year pilot study of clinical outcome measures in collagen VI- and laminin alpha2-related congenital muscular dystrophies

Meilleur

Jain²,

Hynan

et al. 2015

Neuromuscular Disorders

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Potential therapies are currently under development for two congenital muscular dystrophy (CMD) subtypes: Collagen VI Related Myopathy (COL6-RM) and Laminin α2-related dystrophy (LAMA2-RD). However, appropriate clinical outcome measures to be used in clinical trials have not been validated in CMDs. We conducted a two-year pilot study to evaluate feasibility, reliability, and validity of various outcome measures, particularly the Motor Function Measure 32, in 33 subjects with COL6-RM and LAMA2-RD. In the first year, outcome measures tested included: Motor Function Measure 32 (MFM32), forced vital capacity (FVC) percent predicted sitting, myometry, goniometry, 10-meter walk, Egen Klassification 2, and PedsQL™ Generic and Neuromuscular Cores. In the second year, we added the North Star Ambulatory Assessment (NSAA), Hammersmith Functional Motor Scale (HFMS), timed functional tests, Measure of Activity Limitations (ACTIVLIM), Quality of Upper Extremity Skills Test (QUEST), and Patient-Reported Outcomes Measurement Information System (PROMIS) fatigue subscale. The MFM32 showed strong inter-rater (0.92) and internal consistency (0.96) reliabilities. Concurrent validity for the MFM32 was supported by large correlations (range 0.623–0.936) with the following: FVC, NSAA, HFMS, timed functional tests, ACTIVLIM, and QUEST. Significant correlations of the MFM32 were also found with select myometry measurements, mainly of the proximal extremities and domains of the PedsQL™ scales focusing on physical health and neuromuscular disease. Goniometry measurements were less reliable. The Motor Function Measure is reliable and valid in the two specific subtypes of CMD evaluated, COL6-RM and LAMA2-RD. The NSAA is useful as a complementary outcome measure in ambulatory individuals. Preliminary concurrent validity of several other clinical outcome measures was also demonstrated for these subtypes.

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Responsiveness of the Motor Function Measure in Patients With Spinal Muscular Atrophy

Cited by 77 publications

References 31 publications

209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7–9 November 2014, Heemskerk, The Netherlands

209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7–9 November 2014, Heemskerk, The Netherlands

How often should sitting and rising from a chair be evaluated in patients with Duchenne muscular dystrophy?

Results of a two-year pilot study of clinical outcome measures in collagen VI- and laminin alpha2-related congenital muscular dystrophies

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