ArticleBMC neurology2019
Assessment of fatigability in patients with spinal muscular atrophy: development and content validity of a set of endurance tests.
Article in BMC neurology, 2019. 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 24 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
24 citing papers in PubMed, 1 synthesis or guideline pooled it, 43 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
- Temperature-induced symptoms in adolescents and adults with spinal muscular atrophy.Journal of neuromuscular diseases · 2026Article
- Oral function tests in spinal muscular atrophy: closing the diagnostic gap in severely affected adult patients : A prospective observational study.Journal of orofacial orthopedics = Fortschritte der Kieferorthopadie : Organ/official journal Deutsche Gesellschaft fur Kieferorthopadie · 2026Observational
- Investigating the orthotic effect of a passive gravity-compensated exoskeletal device on upper-limb function in people with multiple sclerosis: a pilot cross-sectional study.Journal of neuroengineering and rehabilitation · 2025Article
- Fatigue in Spinal Muscular Atrophy: a fundamental open issue.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2024Review
- Natural History of Mandibular Function in Spinal Muscular Atrophy Types 2 and 3.Journal of neuromuscular diseases · 2024Observational
- 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
- One episode of low intensity aerobic exercise prior to systemic AAV9 administration augments transgene delivery to the heart and skeletal muscle.Journal of translational medicine · 2023Article
- Objective measurement of oral function in adults with spinal muscular atrophy.Orphanet journal of rare diseases · 2023Article
- The RESISTANT study (Respiratory Muscle Training in Patients with Spinal Muscular Atrophy): study protocol for a randomized controlled trial.BMC neurology · 2023Article
- Randomized double-blind placebo-controlled crossover trial with pyridostigmine in spinal muscular atrophy types 2-4.Brain communications · 2023Article
- Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes.Journal of neuromuscular diseases · 2023Article
- Respiratory muscle fatigability in patients with spinal muscular atrophy.Pediatric pulmonology · 2022Article
- Spinal muscular atrophy.Nature reviews. Disease primers · 2022Review
- Magnetic resonance reveals mitochondrial dysfunction and muscle remodelling in spinal muscular atrophy.Brain : a journal of neurology · 2022Article
- Diminished muscle oxygen uptake and fatigue in spinal muscular atrophy.Annals of clinical and translational neurology · 2021Observational
- Persistent neuromuscular junction transmission defects in adults with spinal muscular atrophy treated with nusinersen.BMJ neurology open · 2021Article
- Quality of life data for individuals affected by spinal muscular atrophy: a baseline dataset from the Cure SMA Community Update Survey.Orphanet journal of rare diseases · 2020Article
- Fatigue in adults with spinal muscular atrophy under treatment with nusinersen.Scientific reports · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 1 institution in 1 country.
Funding
Abstract
backgroundFatigability has emerged as an important dimension of physical impairment in patients with Spinal Muscular Atrophy (SMA). At present reliable and valid outcome measures for both mildly and severely affected patients are lacking. Therefore the primary aim of this study is the development of clinical outcome measures for fatigability in patients with SMA across the range of severity.
methodsWe developed a set of endurance tests using five methodological steps as recommended by the 'COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN). In this iterative process, data from multiple sources were triangulated including a scoping review of scientific literature, input from a scientific and clinical multidisciplinary expert panel and three pilot studies including healthy persons (N = 9), paediatric patients with chronic disorders (N = 10) and patients with SMA (N = 15).
resultsFatigability in SMA was operationalised as the decline in physical performance. The following test criteria were established; one method of testing for patients with SMA type 2-4, a set of outcome measures that mimic daily life activities, a submaximal test protocol of repetitive activities over a longer period; external regulation of pace. The scoping review did not generate suitable outcome measures. We therefore adapted the Endurance Shuttle Walk Test for ambulatory patients and developed the Endurance Shuttle Box and Block Test and the - Nine Hole Peg Test for fatigability testing of proximal and distal arm function. Content validity was established through input from experts and patients. Pilot testing showed that the set of endurance tests are comprehensible, feasible and meet all predefined test criteria.
conclusionsThe development of this comprehensive set of endurance tests is a pivotal step to address fatigability in patients with SMA.
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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.