ArticleJournal of neuromuscular diseases
Development of the SMA EFFORT: A new approach to characterize perceived physical fatigability in spinal muscular atrophy.
Article in Journal of neuromuscular diseases. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
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Who cites it
3 citing papers in PubMed.
- A qualitative, mixed-method approach to reaching consensus on function, fatigue, and fatigability outcomes in teens and adults living with spinal muscular atrophy.Orphanet journal of rare diseases · 2025Article
- Italian validation of the SMA independence scale-upper limb module.European journal of pediatrics · 2025Article
- Biomarkers in spinal muscular atrophy.Frontiers in neurology · 2025Review
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Authors and funding
14 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundFatigue and fatigability are commonly reported in spinal muscular atrophy (SMA). Physical fatigability, proposed to be the most relevant to SMA pathophysiology, encompasses performance-based and perceived physical fatigability (PPF) assessments. While performance-based measures have highlighted physical fatigability as an SMA hallmark, PPF is not well characterized due to the lack of disease- and construct-specific scales.
objectiveOur aim was to create a patient-reported outcome measure tailored for SMA, named the SMA EFFORT, to improve PPF assessment. Here, we describe the scale development process, assess scale properties, and discuss future research and application.
methodsSMA and scale development experts organized a comprehensive physical activity item bank, relevant across the SMA phenotypic spectrum. Activities were systematically categorized by varying intensities and durations. The SMA EFFORT was completed by an international cohort of individuals with all types of SMA. To compare PPF across demographic and clinical variables, SMA EFFORT PPF percent (PPF%) composite scoring was established.
resultsOne hundred eighteen participants completed the SMA EFFORT. Total PPF% scores were broadly distributed within functional groups, with differences between non-sitters (35.1 ± 21.0) and sitters (24.9 ± 15.1) (
conclusionsThe novel SMA EFFORT standardizes PPF ratings by anchoring activity to intensity and duration. Item and scale data insights will inform the next iteration, which will undergo additional investigation. The SMA EFFORT aims to improve upon current measures to better assess treatment impact on physical well-being across the SMA spectrum.
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