ArticleOrphanet journal of rare diseases2020
Fatigability in spinal muscular atrophy: validity and reliability of endurance shuttle tests.
Article in Orphanet journal of rare diseases, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05632666 (Respiratory Muscle Training in Patients With Spinal Muscular Atrophy), which is not on this map. Cited by 19 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA).
Who cites it
19 citing papers in PubMed, 1 synthesis or guideline pooled it, 41 citations in OpenAlex.
- Walking test outcomes in adults with genetic neuromuscular diseases: a systematic literature review of their measurement properties.European journal of physical and rehabilitation medicine · 2024Pooled it
- Article
- Utility of Far-Field Potentials as a Biomarker of Neurodegeneration in Spinal Muscular Atrophy.Muscle & nerve · 2026Article
- 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
- Fatigue in Spinal Muscular Atrophy: a fundamental open issue.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2024Review
- Assessing the Assisted Six-Minute Cycling Test as a Measure of Endurance in Non-Ambulatory Patients with Spinal Muscular Atrophy (SMA).Journal of clinical medicine · 2023Article
- Translating fatigability in spinal muscular atrophy to clinical trials and management.Muscle & nerve · 2023Article
- Measuring Fatigue and Fatigability in Spinal Muscular Atrophy (SMA): Challenges and Opportunities.Journal of clinical medicine · 2023Review
- Objective measurement of oral function in adults with spinal muscular atrophy.Orphanet journal of rare diseases · 2023Article
- Article
- The RESISTANT study (Respiratory Muscle Training in Patients with Spinal Muscular Atrophy): study protocol for a randomized controlled trial.BMC neurology · 2023Article
- Pathophysiology and Management of Fatigue in Neuromuscular Diseases.International journal of molecular sciences · 2023Review
- Measuring objective fatigability and autonomic dysfunction in clinical populations: How and why?Frontiers in sports and active living · 2023Review
- Randomized double-blind placebo-controlled crossover trial with pyridostigmine in spinal muscular atrophy types 2-4.Brain communications · 2023Article
- Respiratory muscle fatigability in patients with spinal muscular atrophy.Pediatric pulmonology · 2022Article
- Mastication in Patients with Spinal Muscular Atrophy Types 2 and 3 is Characterized by Abnormal Efficiency, Reduced Endurance, and Fatigue.Dysphagia · 2022Review
- Magnetic resonance reveals mitochondrial dysfunction and muscle remodelling in spinal muscular atrophy.Brain : a journal of neurology · 2022Article
- Short-term effect of air stacking and mechanical insufflation-exsufflation on lung function in patients with neuromuscular diseases.Chronic respiratory diseaseArticle
- Development of the SMA EFFORT: A new approach to characterize perceived physical fatigability in spinal muscular atrophy.Journal of neuromuscular diseasesArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundTo determine construct validity and test-retest reliability of Endurance Shuttle Tests as outcome measures for fatigability of remaining motor functions in children and adults with Spinal Muscular Atrophy (SMA) across the severity spectrum.
resultsWe assessed the Endurance Shuttle - Nine Hole Peg Test (ESNHPT), - Box and Block Test (ESBBT) and - Walk Test (ESWT) in 61 patients with SMA types 2-4, 25 healthy controls (HC) and 15 disease controls (DC). Convergent validity, discriminative validity and test-retest reliability were investigated. Additionally, we compiled the Endurance Shuttle Combined Score (ESTCS) by selecting the most relevant endurance test of each individual. 54, 70 and 73% of patients with SMA demonstrated increased fatigability on the ESNHPT, ESBBT and the ESWT. Endurance response in SMA was characterized by a decrease in muscle strength, an increase in muscle fatigue and an increase in motor adaptions, thereby confirming convergent validity. Patients with SMA showed increased drop-out rates and a shorter endurance time compared to HC and DC demonstrating good discriminative validity. Test-retest reliability was moderate to excellent (ICC's ranging from .78 to .91) with a trend towards better performance on retest. The ESTCS increased sample size and drop-out rate up to 100 and 85%.
conclusionsFatigability is an important additional dimension of physical impairments across the severity spectrum in children and adults with SMA. The EST's are reliable and valid to document fatigability of walking, proximal- and distal arm function in SMA and thus are promising outcome measures for use in clinical trials.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.