Date Presented Accepted for AOTA INSPIRE 2021 but unable to be presented due to online event limitations.
This research presentation provides information on daytime and nighttime activity of boys with DMD in their everyday lives and while enrolled in a 12-month clinical trial. This research addresses the gap in knowledge regarding community-based outcomes in DMD, such as daily activity and sleep, and how therapeutic interventions may affect these outcomes.
Primary Author and Speaker: Roxanna Bendixen
Additional Authors and Speakers: Sarah McKendry, Natalie Silverman
Contributing Authors: Amy Gore Hartman
PURPOSE: Duchenne Muscular Dystrophy (DMD) is an X-linked neuromuscular disorder—the most prevalent and lethal of all muscular dystrophies. While clinical trials of promising therapeutics in DMD are increasing, travel to expert medical centers to participate in these clinical trials is difficult and disruptive to the child and family. Recent events have heightened the need for remote methods for clinical assessments, especially during clinical trials. We present data from a current clinical trial focused on replacing the standard of care in DMD—prednisone (a well-known corticosteroid). Our primary objective is the exploration of community-based daily activity and sleep. The ability to measure activity and sleep remotely in tandem with a drug trial has provided data necessary to capture real-life outcomes and explore their relationship with a new, safe alternative to current standard of care in DMD.
DESIGN: Longitudinal exploratory study to determine the effect of VBP (NCT03439670) on daily activity and sleep using continuous actigraphy monitoring over a 12-month period. Boys are randomized to 4-arms: low-dose VBP, high-dose VBP, prednisone, and placebo. The study is double-blinded with cross-over to VBP at 6 months.
METHODS: Participants in this study are active subjects in a national drug trial. They are wearing the FDA-approved ActiGraph GT9x for 10 hours per day and all night throughout the clinical trial. Actigraphy data provide activity levels (moderate-vigorous physical activity (MVPA)) and nighttime percent sleep efficiency. Additionally, caregivers complete a weekly activity log and monthly parent proxy-reports on their child's sleep using the Children’s Sleep Habits Questionnaire (CSHQ).
RESULTS: To date we have enrolled 21 participants. Eight subjects have completed the first 6-months (age 5.4 + 1.3); four subjects have crossed-over to VBP and completed 12-months. Baseline to 6-month data show daily activity (MVPA) at more than 70% spent in sedentary/light activities, 30% in moderate, with no activities in the vigorous range. We recorded low percent sleep efficiency (baseline 74.3% + 10.9; 6-months 67.8% + 10.2) and sleep quality (CSHQ) (baseline 56.3 + 6.5; 6-months 54.5 + 6.5) in all participants. At 12-months, MVPA remained constant, while two subjects demonstrated a single shift in longitudinal sleep efficiency data that corresponds to the cross-over point to VBP, whereby sleep efficiency improved, and variability in sleep patterns declined.
CONCLUSION: This is one of the first trials to implement actigraphy outcomes for community-based monitoring of daily activity and sleep in young boys with DMD. Although our long-term objective is the detection of drug effect on daily activity and sleep, our data clearly illustrate that young boys with DMD experience high levels of sedentary behavior, poor sleep efficiency, and low rates of parent-reported sleep quality. At 12-months on drug, we observed an overall improvement in sleep efficiency following crossover to VBP for two participants. We speculate that these two subjects were randomized to prednisone, a corticosteroid well-known to disrupt sleep patterns, and the cross-over to VBP mitigated these sleep disruptions. Clinical drug trials have not considered sleep as an outcome for DMD, as the focus is locomotor based (walking/running/climbing stairs). The low levels of activity and poor sleep in young boys with DMD deserve particular attention due the strong influence of physical activity and sleep on overall health. Boys with DMD often receive therapy at a very young age; occupational therapists are uniquely qualified and prepared to intervene early in safe ways to increase activity and improve sleep outcomes (in partnership with oth
References
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