An exploratory qualitative assessment of patient and clinician perspectives on patient-reported outcome measures and disease-modifying therapies in adults with spinal muscular atrophy
Objective: To understand patient, caregiver, and clinician perspectives on patient-reported outcome measures, critical functional domains, and disease-modifying therapies in adult spinal muscular atrophy. Design: An exploratory qualitative single-site study. Patients: Ten adults with spinal muscul...
Saved in:
| Main Authors: | , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Medical Journals Sweden
2025-01-01
|
| Series: | Journal of Rehabilitation Medicine |
| Subjects: | |
| Online Access: | https://medicaljournalssweden.se/jrm/article/view/41254 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1841527818236723200 |
|---|---|
| author | Jeremy Slayter Lauren Casey Shane McCullum Dorothy Drost Allison Banks Colleen O'Connell |
| author_facet | Jeremy Slayter Lauren Casey Shane McCullum Dorothy Drost Allison Banks Colleen O'Connell |
| author_sort | Jeremy Slayter |
| collection | DOAJ |
| description | Objective: To understand patient, caregiver, and clinician perspectives on patient-reported outcome measures, critical functional domains, and disease-modifying therapies in adult spinal muscular atrophy.
Design: An exploratory qualitative single-site study.
Patients: Ten adults with spinal muscular atrophy and two clinicians participated in semi-structured interviews.
Methods: Semi-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.
Results: Ten 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.
Conclusion: After 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.
|
| format | Article |
| id | doaj-art-41a09505ce8d4871ab9073dc67914a31 |
| institution | Kabale University |
| issn | 1651-2081 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Medical Journals Sweden |
| record_format | Article |
| series | Journal of Rehabilitation Medicine |
| spelling | doaj-art-41a09505ce8d4871ab9073dc67914a312025-01-15T07:05:03ZengMedical Journals SwedenJournal of Rehabilitation Medicine1651-20812025-01-015710.2340/jrm.v57.41254An exploratory qualitative assessment of patient and clinician perspectives on patient-reported outcome measures and disease-modifying therapies in adults with spinal muscular atrophyJeremy Slayter0Lauren Casey1Shane McCullum2Dorothy Drost3Allison Banks4Colleen O'Connell5Division of Physical Medicine & Rehabilitation, Dalhousie University, Halifax, Nova Scotia, Canada; Stan Cassidy Centre for Rehabilitation, Horizon Health Network, Fredericton, New Brunswick, CanadaStan Cassidy Centre for Rehabilitation, Horizon Health Network, Fredericton, New Brunswick, CanadaStan Cassidy Centre for Rehabilitation, Horizon Health Network, Fredericton, New Brunswick, CanadaStan Cassidy Centre for Rehabilitation, Horizon Health Network, Fredericton, New Brunswick, CanadaStan Cassidy Centre for Rehabilitation, Horizon Health Network, Fredericton, New Brunswick, CanadaStan Cassidy Centre for Rehabilitation, Horizon Health Network, Fredericton, New Brunswick, Canada; Dalhousie Medicine New Brunswick, Dalhousie University, Saint John, New Brunswick, CanadaObjective: To understand patient, caregiver, and clinician perspectives on patient-reported outcome measures, critical functional domains, and disease-modifying therapies in adult spinal muscular atrophy. Design: An exploratory qualitative single-site study. Patients: Ten adults with spinal muscular atrophy and two clinicians participated in semi-structured interviews. Methods: Semi-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. Results: Ten 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. Conclusion: After 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. https://medicaljournalssweden.se/jrm/article/view/41254adult spinal muscular atrophyneuromuscular diseasepatient-centred carepatient-reported outcome measureQualitative ResearchActivities of Daily Living |
| spellingShingle | Jeremy Slayter Lauren Casey Shane McCullum Dorothy Drost Allison Banks Colleen O'Connell An exploratory qualitative assessment of patient and clinician perspectives on patient-reported outcome measures and disease-modifying therapies in adults with spinal muscular atrophy Journal of Rehabilitation Medicine adult spinal muscular atrophy neuromuscular disease patient-centred care patient-reported outcome measure Qualitative Research Activities of Daily Living |
| title | An exploratory qualitative assessment of patient and clinician perspectives on patient-reported outcome measures and disease-modifying therapies in adults with spinal muscular atrophy |
| title_full | An exploratory qualitative assessment of patient and clinician perspectives on patient-reported outcome measures and disease-modifying therapies in adults with spinal muscular atrophy |
| title_fullStr | An exploratory qualitative assessment of patient and clinician perspectives on patient-reported outcome measures and disease-modifying therapies in adults with spinal muscular atrophy |
| title_full_unstemmed | An exploratory qualitative assessment of patient and clinician perspectives on patient-reported outcome measures and disease-modifying therapies in adults with spinal muscular atrophy |
| title_short | An exploratory qualitative assessment of patient and clinician perspectives on patient-reported outcome measures and disease-modifying therapies in adults with spinal muscular atrophy |
| title_sort | exploratory qualitative assessment of patient and clinician perspectives on patient reported outcome measures and disease modifying therapies in adults with spinal muscular atrophy |
| topic | adult spinal muscular atrophy neuromuscular disease patient-centred care patient-reported outcome measure Qualitative Research Activities of Daily Living |
| url | https://medicaljournalssweden.se/jrm/article/view/41254 |
| work_keys_str_mv | AT jeremyslayter anexploratoryqualitativeassessmentofpatientandclinicianperspectivesonpatientreportedoutcomemeasuresanddiseasemodifyingtherapiesinadultswithspinalmuscularatrophy AT laurencasey anexploratoryqualitativeassessmentofpatientandclinicianperspectivesonpatientreportedoutcomemeasuresanddiseasemodifyingtherapiesinadultswithspinalmuscularatrophy AT shanemccullum anexploratoryqualitativeassessmentofpatientandclinicianperspectivesonpatientreportedoutcomemeasuresanddiseasemodifyingtherapiesinadultswithspinalmuscularatrophy AT dorothydrost anexploratoryqualitativeassessmentofpatientandclinicianperspectivesonpatientreportedoutcomemeasuresanddiseasemodifyingtherapiesinadultswithspinalmuscularatrophy AT allisonbanks anexploratoryqualitativeassessmentofpatientandclinicianperspectivesonpatientreportedoutcomemeasuresanddiseasemodifyingtherapiesinadultswithspinalmuscularatrophy AT colleenoconnell anexploratoryqualitativeassessmentofpatientandclinicianperspectivesonpatientreportedoutcomemeasuresanddiseasemodifyingtherapiesinadultswithspinalmuscularatrophy AT jeremyslayter exploratoryqualitativeassessmentofpatientandclinicianperspectivesonpatientreportedoutcomemeasuresanddiseasemodifyingtherapiesinadultswithspinalmuscularatrophy AT laurencasey exploratoryqualitativeassessmentofpatientandclinicianperspectivesonpatientreportedoutcomemeasuresanddiseasemodifyingtherapiesinadultswithspinalmuscularatrophy AT shanemccullum exploratoryqualitativeassessmentofpatientandclinicianperspectivesonpatientreportedoutcomemeasuresanddiseasemodifyingtherapiesinadultswithspinalmuscularatrophy AT dorothydrost exploratoryqualitativeassessmentofpatientandclinicianperspectivesonpatientreportedoutcomemeasuresanddiseasemodifyingtherapiesinadultswithspinalmuscularatrophy AT allisonbanks exploratoryqualitativeassessmentofpatientandclinicianperspectivesonpatientreportedoutcomemeasuresanddiseasemodifyingtherapiesinadultswithspinalmuscularatrophy AT colleenoconnell exploratoryqualitativeassessmentofpatientandclinicianperspectivesonpatientreportedoutcomemeasuresanddiseasemodifyingtherapiesinadultswithspinalmuscularatrophy |