Open AccessResponsiveness of outcome measures in myotonic dystrophy type 1
Author(s) -
Knak Kirsten L.,
Sheikh Aisha M.,
Witting Nanna,
Vissing John
Publication year - 2020
Publication title -
annals of clinical and translational neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.824
H-Index - 42
ISSN - 2328-9503
DOI - 10.1002/acn3.51129
Subject(s) - medicine , physical medicine and rehabilitation , myotonic dystrophy , balance (ability) , balance test , step test , physical therapy , ceiling effect , timed up and go test , isometric exercise , muscle strength , population , significant difference , alternative medicine , environmental health , pathology
Objective As myotonic dystrophy type 1(DM1) evolves slowly and interventional trials often have a short duration, responsive outcomes in DM1 are needed. The objective of this study was to determine the responsiveness of muscle strength, balance, and functional mobility measurements after a 1‐year follow‐up period in individuals with DM1. Methods Sixty‐three adults with noncongenital DM1 completed the following assessments at baseline and at 1‐year follow‐up: Handheld dynamometry (lower limbs), stationary dynamometry (lower limbs), step test, timed‐up‐and‐go test (TUG), modified clinical test of sensory integration and balance (mCTSIB), feet‐together stance, tandem stance, one‐leg stance, 10‐meter walk test, and sit‐to‐stand test. Results Change was captured by stationary dynamometry (proximal flexor and extensor muscles), handheld dynamometry (proximal flexor and distal extensor muscles), TUG, and mCTSIB ( P ≤ 0.04). Ceiling or floor effects were shown for most static balance tests. Interpretation Overall, adequate responsiveness was shown for both muscle strength dynamometers, TUG and mCTSIB. These outcomes are therefore likely candidate endpoints for clinical trials lasting 1 year. Most static balance tests are not responsive and not recommended in a heterogeneous DM1 population.