INTRODUCTION: The heterogeneity of symptoms experienced by myotonic dystrophy type 1 (DM1) patients means patient-reported outcome (PRO) assessments are uniquely suited to address this through questionnaires. METHODS: A structured literature review of PRO measures used in DM1 populations, comparing psychometric data from this population was undertaken. RESULTS: One health status measure, 3 activities of daily living (ADL) scales, 3 health-related quality of life (HRQOL) assessments, and 5 sleep and fatigue measures have validity and reliability information from DM1 populations. The Myotonic Dystrophy Health Index and DM1 Activity and Participation Scale (DM1-Activ) have the strongest validity and reliability evidence. The DM1-Activc has been published recently and builds on the DM1-Activ by adding more relevant items. CONCLUSIONS: The PRO instruments we identified have varying psychometric evidence in DM1 populations; all require further testing to be confident of their ability to make accurate and valid measurements of symptoms, HRQOL, and ADL in a DM1 population. Muscle Nerve 56: 86-92, 2017.
INTRODUCTION: The heterogeneity of symptoms experienced by myotonic dystrophy type 1 (DM1) patients means patient-reported outcome (PRO) assessments are uniquely suited to address this through questionnaires. METHODS: A structured literature review of PRO measures used in DM1 populations, comparing psychometric data from this population was undertaken. RESULTS: One health status measure, 3 activities of daily living (ADL) scales, 3 health-related quality of life (HRQOL) assessments, and 5 sleep and fatigue measures have validity and reliability information from DM1 populations. The Myotonic Dystrophy Health Index and DM1 Activity and Participation Scale (DM1-Activ) have the strongest validity and reliability evidence. The DM1-Activc has been published recently and builds on the DM1-Activ by adding more relevant items. CONCLUSIONS: The PRO instruments we identified have varying psychometric evidence in DM1 populations; all require further testing to be confident of their ability to make accurate and valid measurements of symptoms, HRQOL, and ADL in a DM1 population. Muscle Nerve 56: 86-92, 2017.
Authors: Bettine A H Vosse; Charlotte Seijger; Nicolle Cobben; Baziel van Engelen; Sander M J van Kuijk; Catharina Faber; Peter Wijkstra Journal: Respiration Date: 2021-05-07 Impact factor: 3.580
Authors: Erik Landfeldt; Nikoletta Nikolenko; Cecilia Jimenez-Moreno; Sarah Cumming; Darren G Monckton; Catharina G Faber; Ingemar S J Merkies; Grainne Gorman; Chris Turner; Hanns Lochmüller Journal: J Neurol Date: 2020-06-15 Impact factor: 4.849