Responsiveness of performance-based outcome measures for mobility, balance, muscle strength and manual dexterity in adults with myotonic dystrophy type 1

Kierkegaard, Marie; Petitclerc, Émilie; Hébert, Luc J.; Mathieu, Jean; Gagnon, Cynthia

doi:10.2340/16501977-2304

Cited by 15 publications

(21 citation statements)

References 34 publications

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“…A significant increase in hip flexor strength assessed by stationary dynamometry was found in this study as well as in a previous DM1 study. 8 However, this is considered a spurious type II error finding because of no change in physical activity level in this study and the loss of hip flexor strength recorded by HHD in the present-and a previous DM1 study. 14 This study demonstrated loss of strength in knee and hip flexors and ankle plantar flexors assessed by HHD.…”

Section: Muscle Strength Measurementsmentioning

confidence: 51%

“…The objective measurements were generally poorly reflected by the subjective scoring of changes in muscle strength, balance, and functional mobility using the GRS after 1 year. This relationship was better matched when the observation period was 9 years, 8 where larger changes occur that can be more easily perceived. However, overall the true agreement between the objective measurements and subjective assessments in this study is somewhere Step test: missing values (n = 1) because patient did not finish the test due to knee pain.…”

Section: Outcome Measures Against Grsmentioning

confidence: 89%

“…Our study did not show change in knee extensors and ankle dorsal flexors with HHD, but studies of longer duration in DM1 have shown this. 8,14 It has previously been shown that 1 year is too short to register progression in these muscles in DM1. 15 Compared to previous findings of significant decline in the knee extensors, but not in the knee flexors using stationary dynamometry in DM, 16 this study found a reduction of strength in both knee extensors and flexors.…”

Section: Muscle Strength Measurementsmentioning

confidence: 99%

“…The failure of capturing a change in the one-leg-stance eyes closed test was probably caused by a floor effect, missing values, and heterogeneity of the patients' performances. After 5-9 years of observation, deterioration in DM1 has been shown not only in the TUG 8,14 but also in the step test. 14 The ceiling effects in the feet-together stance, tandem stance, and one-leg-stance eyes open and the floor effect in the one-leg-stance eyes closed test suggests that these tests are unresponsive outcomes in a heterogeneous DM1 cohort.…”

Section: Balance Measurementsmentioning

confidence: 99%

“…Responsiveness is a tool's ability to detect change in a condition over time. 7 Responsiveness of timed-up-and-go test (TUG) and handheld dynamometry (HHD) in the lower limbs has previously been investigated in DM1, 8 but the follow-up period was 9 years. Because clinical trials often have a maximum duration of 1 year, the challenge is to identify responsive endpoints within 1 year despite the slowly progressive nature of DM1.…”

Section: Introductionmentioning

confidence: 99%

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Responsiveness of outcome measures in myotonic dystrophy type 1

Knak

Sheikh

Witting

et al. 2020

Ann Clin Transl Neurol

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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.

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Section: Muscle Strength Measurementsmentioning

confidence: 51%

Section: Outcome Measures Against Grsmentioning

confidence: 89%

Section: Muscle Strength Measurementsmentioning

confidence: 99%

Section: Balance Measurementsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Responsiveness of outcome measures in myotonic dystrophy type 1

Knak

Sheikh

Witting

et al. 2020

Ann Clin Transl Neurol

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Remote assessment of myotonic dystrophy type 1: A feasibility study

et al. 2022

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Introduction/Aims Remote study visits (RSVs) are emerging as important tools for clinical research. We tested the feasibility of using RSVs to evaluate patients with myotonic dystrophy type 1 (DM1), including remote quantitative assessment of muscle function, and we assessed correlations of remote assessments with patient‐reported function. Methods Twenty three subjects with DM1 were consented remotely. Toolkits containing a tablet computer, grip dynamometer, and spirometer were shipped to participants. The tablets were loaded with software for video‐conferencing and questionnaires about functional impairment, patient experience with technology, and willingness to participate in future remote studies. Grip strength, forced vital capacity, peak cough flow, timed‐up‐and‐go (TUG), and grip myotonia (hand opening time) were determined during RSVs. We assessed correlations of remote assessments with patient‐reported outcomes of muscle function and with CTG repeat size. Results All 23 subjects completed RSVs. 95% of participants were able to complete all components of the remote study. All toolkit components were returned upon completion. Grip strength and TUG demonstrated moderate to strong correlations with self‐reported inventories of upper and lower extremity impairment, respectively (ρ = 0.7 and ρ = −0.52). A total of 91% of subjects expressed interest in participating in future RSVs. Discussion Results of this study support the feasibility of using portable devices and video‐conferencing for remote collection of patient‐reported outcomes and quantitative assessment of muscle function in DM1.

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