A Canadian Adult Spinal Muscular Atrophy Outcome Measures Toolkit: Results of a National Consensus using a Modified Delphi Method
| العنوان: | A Canadian Adult Spinal Muscular Atrophy Outcome Measures Toolkit: Results of a National Consensus using a Modified Delphi Method |
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| المؤلفون: | Monique Taillon, Jeremy Slayter, Angela Genge, Kristine M. Chapman, Hanns Lochmüller, Christen Shoesmith, Aaron Izenberg, Wendy Johnston, Christine Stables, Colleen O'Connell, Stephanie Plamondon, Jodi Warman-Chardon, Lawrence Korngut, Victoria Hodgkinson, Kerri Schellenberg, Bernard Brais, Erin K. O'Ferrall, Gerald Pfeffer, Josh Lounsberry, Xavier Rodrigue |
| المصدر: | Journal of Neuromuscular Diseases |
| بيانات النشر: | IOS Press, 2021. |
| سنة النشر: | 2021 |
| مصطلحات موضوعية: | Research Report, Canada, medicine.medical_specialty, Consensus, Delphi Technique, Standardization, delphi method, Delphi method, Modified delphi, neuromuscular diseases, Adult spinal muscular atrophy, Muscular Atrophy, Spinal, outcome measures, 03 medical and health sciences, 0302 clinical medicine, Outcome Assessment, Health Care, medicine, Humans, 030212 general & internal medicine, business.industry, Outcome measures, Spinal muscular atrophy, medicine.disease, SMA, Neurology, Data quality, Physical therapy, Neurology (clinical), business, 030217 neurology & neurosurgery |
| الوصف: | Background: Spinal Muscular Atrophy (SMA) is a rare disease that affects 1 in 11 000 live births. Recent developments in SMA treatments have included new disease-modifying therapies that require high quality data to inform decisions around initiation and continuation of therapy. In Canada, there are no nationally agreed upon outcome measures (OM) used in adult SMA. Standardization of OM is essential to obtain high quality data that is comparable among neuromuscular clinics. Objective: To develop a recommended toolkit and timing of OM for assessment of adults with SMA. Methods: A modified delphi method consisting of 2 virtual voting rounds followed by a virtual conference was utilized with a panel of expert clinicians treating adult SMA across Canada. Results: A consensus-derived toolkit of 8 OM was developed across three domains of function, with an additional 3 optional measures. Optimal assessment frequency is 12 months for most patients regardless of therapeutic access, while patients in their first year of receiving disease-modifying therapy should be assessed more frequently. Conclusions: The implementation of the consensus-derived OM toolkit will improve monitoring and assessment of adult SMA patients, and enrich the quality of real-world evidence. Regular updates to the toolkit must be considered as new evidence becomes available. |
| تدمد: | 2214-3602 2214-3599 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f5a8c69bfeac82753b239ff91fd5fe43Test https://doi.org/10.3233/jnd-200617Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....f5a8c69bfeac82753b239ff91fd5fe43 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 22143602 22143599 |
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