ArticleJournal of rehabilitation medicine2025
An exploratory qualitative assessment of patient and clinician perspectives on patient-reported outcome measures and disease-modifying therapies in adults with spinal muscular atrophy.
Article in Journal of rehabilitation medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
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
3 citing papers in PubMed.
- Risdiplam treatment in adults with spinal muscular atrophy: a single-center, real-world study.BMC neurology · 2026Article
- A Psychometric Evaluation of Maximum Phonation Time and S/Z Ratio as Pragmatic Outcome Measures of Bulbar Function in Adults With Spinal Muscular Atrophy.Muscle & nerve · 2026Observational
- Italian validation of the SMA independence scale-upper limb module.European journal of pediatrics · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveTo understand patient, caregiver, and clinician perspectives on patient-reported outcome measures, critical functional domains, and disease-modifying therapies in adult spinal muscular atrophy.
designAn exploratory qualitative single-site study. PATIENTS: Ten adults with spinal muscular atrophy and two clinicians participated in semi-structured interviews.
methodsSemi-structured interviews were conducted virtually or in person with participants after they completed outcome measures at a routine clinic visit. Two researchers analysed transcripts concurrently using a thematic approach to determine themes.
resultsTen themes were identified among partici-pants. Patient-reported outcome measure preference varied between functional groups and was under-responsive, although it captured meaningful data. Motor stability was most frequently expected with disease-modifying therapy, but participants also reported improved fatigue and respiratory status.
conclusionAfter considering patient goals, functional status, and preferences, patient-reported outcome measures represent a valuable adjunct to standard clinical and research tools. Optimal selection of patient-reported outcome measures requires careful consideration of multiple patient factors. Collaborative development of modified patient-reported outcome measures may yield a responsive, meaningful, and acceptable tool that can be used across a broad functional spectrum.
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.