Motor Function Measure: Validation of a Short Form for Young Children With Neuromuscular Diseases

Lattre, Capucine de; Payán, C.; Vuillerot, Carole; Rippert, Pascal; Castro, Denis de; Bérard, C.; Poirot, I.

doi:10.1016/j.apmr.2013.04.001

Cited by 70 publications

(72 citation statements)

References 25 publications

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“…Although validated for children older than 6 years old, children between 4 and 5 years old were able to complete all items on the MFM32 [27]. The olesoxime (Trophos) MFM data were presented for type 2 and non-ambulant type 3 SMA patients from 3 to 25 years old (see below).…”

Section: Natural History Studies and Relevant Outcome Measuresmentioning

confidence: 99%

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: 99%

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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“…Concurrent validity was assessed for the Hammersmith expanded version (O'Hagen et al, ) , reporting the Pearson Rank Correlation for scores compared to relevant items from the gross motor function measure. For convergent validity, scores from functional outcome measures aligned with scores from comparator scales (e.g., functional reaching scales and Hammersmith Functional Motor Scales) or clinical presentation (O'Hagen et al, ; Mazzone et al, ; de Lattre et al, ) . Two functional outcome measures were also able to discriminate between disease type (different neuromuscular disorders including CMT vs. DMD, SMA, and others) (de Lattre et al, ) or subtype (SMA II vs. SMA III) (Krosschell et al, ) .…”

Section: Resultsmentioning

confidence: 99%

“…None were specifically for CMT; however, several items across scales might be appropriate for infants with CMT. Most of the outcome measures were designed for patients with SMA, while one was for neuromuscular disorders in general and included a mixed cohort comprising 13 infants with CMT (de Lattre et al, ) . The validity and reliability of the functional outcome measures was generally acceptable, and the methodological quality of the included papers was high.…”

Section: Discussionmentioning

confidence: 99%

“…The only outcome measure validated with CMT (n = 13/88 aged 2–7 years, 15% of study sample CMT, types not reported) was the MFM‐20 (de Lattre et al, ) . The MFM‐20 includes items suitable for infants with CMT, particularly with regards to the range of gross motor items included in the standing position/transfers domain and axial/proximal motor function domain.…”

Section: Discussionmentioning

confidence: 99%

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Functional outcome measures for infantile Charcot‐Marie‐Tooth disease: a systematic review

Mandarakas

Rose

Sanmaneechai

et al. 2018

J Peripheral Nervous Sys

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A functional outcome measure for infants (aged 0-3 years) with Charcot-Marie-Tooth (CMT) disease is needed for upcoming disease-modifying trials. A systematic review of outcome measures for infants with neuromuscular disorders was completed to determine if validated measures were available for the CMT infant population. We assessed 20,375 papers and identified seven functional outcome measures for infants with neuromuscular disorders. Six were developed and validated for spinal muscular atrophy (SMA). There were no CMT-specific outcome measures identified; however, one (motor function measure) assessed a range of neuromuscular disorders including 13 infants and children with CMT. The included studies exhibited "good" face, discriminant, convergent and concurrent validity, and reported excellent intra- and inter-rater reliability. No outcome measure was subjected to item response theory. Studies reported outcome measures comprising of 51 different items assessing six domains of function: reflexive movement, axial movement, limb movement, positioning, gross motor, and fine-motor skills. Scoring of items ranged from 2- to 7-point rating scales; and none were scaled to normative reference values to account for changes in growth and development. The SMA focus of most items is likely to produce ceiling effects and lack sensitivity and responsiveness for within and between types of CMT in infants. Nevertheless, several items across scales assessing distal strength, gross- and fine-motor function, could be included in the development of a composite functional outcome measure for infants with CMT to assess disease-modifying interventions.

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“…In contrast, functional motor scales, like the NSAA are not appropriate for children below the age of 4 and have only been validated for boys above the age of 5. Other scales, like the MFM have also been validated from the age of 5 years [17]. In order to fill this gap, we adapted the NSAA to make it suitable for boys younger than 5 years.…”

Section: Discussionmentioning

confidence: 99%

Revised North Star ambulatory assessment for young boys with Duchenne muscular dystrophy

Coratti

Messina

Ricotti

et al. 2016

Neuromuscular Disorders

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The advent of therapeutic approaches for Duchenne muscular dystrophy (DMD) has highlighted the need to identify reliable outcome measures for young boys with DMD. The aim of this study was to develop a revised version of the North Star Ambulatory Assessment (NSAA) suitable for boys between the age of 3 and 5 years by identifying age appropriate items and revising the scoring system accordingly. Using the scale in 171 controls between the age of 2.9 and 4.8 years, we identified items that were appropriate at different age points. An item was defined as age appropriate if it was completed, achieving a full score, by at least 85% of the typically developing boys at that age. At 3 years (±3months) there were only 8 items that were age appropriate, at 3 years and 6 months there were 13 items while by the age of 4 years all 17 items were appropriate. A revised version of the scale was developed with items ordered according to the age when they could be reliably performed. The application of the revised version of the scale to data collected in young DMD boys showed that very few of the DMD boys were able to complete with a full score all the age appropriate items. In conclusion, our study suggests that a revised version of the NSAA can be used in boys from the age of 3 years to obtain information on how young DMD boys acquire new abilities and how this correlates with their peers.

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Motor Function Measure: Validation of a Short Form for Young Children With Neuromuscular Diseases

Cited by 70 publications

References 25 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

Functional outcome measures for infantile Charcot‐Marie‐Tooth disease: a systematic review

Revised North Star ambulatory assessment for young boys with Duchenne muscular dystrophy

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