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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Review of patient-reported outcome measures for use in myotonic dystrophy type 1 patients
Tara Symonds1, Jason A Randall1, Patrick Campbell2
1Clinical Outcomes Solutions, Folkestone, Kent, United Kingdom.
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.
