An exploratory qualitative assessment of patient and clinician perspectives on patient-reported outcome measures and disease-modifying therapies in adults with spinal muscular atrophy.
Jeremy Slayter, Lauren Casey, Shane McCullum and 3 others
PMID 39810476WHAT IT FOUND
Adults with spinal muscular atrophy found standard outcome measures discouraging and insensitive to meaningful change.
Walkers preferred broad questionnaires, while non-sitters found them depressing and preferred disease-specific scales. Clinicians should select measures based on functional status to avoid psychological harm.
Key findings
01Patient acceptability of outcome measures varied by functional group, with walkers preferring broad scales and non-sitters preferring disease-specific ones.
02Participants reported that outcome measures were under-responsive to meaningful functional changes and failed to capture how tasks were completed with assistance.
03Clinicians identified gaps in assessing mental health, fatigue, and bulbar function, noting that physical scores often did not reflect high life satisfaction.
STILL TO COME
How it was doneWhat they foundWhat it means for PTsWhat it means for OTsWhat it means for SLPs
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What it does not show
The study had a very small sample size (10 patients, 2 clinicians), limiting the generalizability of themes. There was a delay of approximately 2.8 months between completing the outcome measures and the interviews, which may have affected recall. The qualitative design cannot quantify psychometric properties like reliability or validity, only perceptions of them. Participants were excluded if they had additional physical or cognitive comorbidities, so findings may not apply to those with complex health profiles.
Declared interests
The study received funding from ResearchNB, Dalhousie University, and Hoffman-La Roche Limited (a manufacturer of a disease-modifying therapy for SMA).
The easy way to misread this
Do not interpret the finding that PROMs are 'discouraging' as a reason to stop using outcome measures entirely. The authors suggest refining and selecting measures carefully to match the patient's functional group, not abandoning assessment.