A Canadian Adult Spinal Muscular Atrophy Outcome Measures Toolkit: Results of a National Consensus using a Modified Delphi Method
| Autor: | 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 |
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| Rok vydání: | 2021 |
| Předmět: |
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 |
| Zdroj: | Journal of Neuromuscular Diseases |
| ISSN: | 2214-3602 2214-3599 |
| DOI: | 10.3233/jnd-200617 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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