ArticleMuscle & nerve2014
Rasch analysis of clinical outcome measures in spinal muscular atrophy.
Article in Muscle & nerve, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
27 citing papers in PubMed, 1 synthesis or guideline pooled it, 77 citations in OpenAlex.
- Systematic Literature Review to Assess Economic Evaluations in Spinal Muscular Atrophy (SMA).PharmacoEconomics · 2022Pooled it
- Trial
- Upper limb evaluation and one-year follow up of non-ambulant patients with spinal muscular atrophy: an observational multicenter trial.PloS one · 2015Trial
- Comorbidities in spinal muscular atrophy and their impact on the course of the underlying disease: a real-life observational study.BMC musculoskeletal disorders · 2025Observational
- Concomitant telomere attrition is associated with spinal muscular atrophy in highly inbred region of North India: unraveling the thread in Kashmir region.BMC medical genomics · 2024Article
- Taldefgrobep Alfa and the Phase 3 RESILIENT Trial in Spinal Muscular Atrophy.International journal of molecular sciences · 2024Review
- Design and Validation of a Clinical Outcome Measure for Adolescents and Adult Patients with Spinal Muscular Atrophy: SMA Life Study Protocol.Neurology and therapy · 2024Article
- Smartphone-Based Assessment of Mobility and Manual Dexterity in Adult People with Spinal Muscular Atrophy.Journal of neuromuscular diseases · 2024Article
- Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3.Journal of neuromuscular diseases · 2024Article
- A Mixed-method Approach to Develop an Ambulatory Module of the SMA Independence Scale.Journal of neuromuscular diseases · 2023Article
- Validation of motor and functional scales for the evaluation of adult patients with 5q spinal muscular atrophy.European journal of neurology · 2022Article
- Revised Hammersmith Scale for spinal muscular atrophy: Inter and intra-rater reliability and agreement.PloS one · 2022Article
- A Patient-Centered Evaluation of Meaningful Change on the 32-Item Motor Function Measure in Spinal Muscular Atrophy Using Qualitative and Quantitative Data.Frontiers in neurology · 2021Article
- A continuous repetitive task to detect fatigability in spinal muscular atrophy.Orphanet journal of rare diseases · 2018Article
- Patient reported outcome measures in rare diseases: a narrative review.Orphanet journal of rare diseases · 2018Review
- An Objective and Child-friendly Assessment of Arm Function by Using a 3-D Sensor.Journal of visualized experiments : JoVE · 2018Article
- Motor Function Test Reliability During the NeuroNEXT Spinal Muscular Atrophy Infant Biomarker Study.Journal of neuromuscular diseases · 2018Article
- Article
- Building the patient community.Gene therapy · 2017Review
- Longitudinal characterization of biomarkers for spinal muscular atrophy.Annals of clinical and translational neurology · 2017Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
25 authors at 18 institutions in 5 countries.
Funding
Abstract
introductionTrial design for SMA depends on meaningful rating scales to assess outcomes. In this study Rasch methodology was applied to 9 motor scales in spinal muscular atrophy (SMA).
methodsData from all 3 SMA types were provided by research groups for 9 commonly used scales. Rasch methodology assessed the ordering of response option thresholds, tests of fit, spread of item locations, residual correlations, and person separation index.
resultsEach scale had good reliability. However, several issues impacting scale validity were identified, including the extent that items defined clinically meaningful constructs and how well each scale measured performance across the SMA spectrum.
conclusionsThe sensitivity and potential utility of each SMA scale as outcome measures for trials could be improved by establishing clear definitions of what is measured, reconsidering items that misfit and items whose response categories have reversed thresholds, and adding new items at the extremes of scale ranges.
Indexed as
Identifiers
What OpenQuestion holds
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.