Date Presented 03/28/20
Our longitudinal study is exploring the feasibility and reliability of using actigraphy as a meaningful outcome measure in children ages four through seven involved in a clinical trial. Our data examine activity levels and sleep patterns in boys with DMD in their natural environment throughout the drug trial. We hypothesize that using activity monitoring as an outcome measure will provide a meaningful view of daytime and nighttime activity for these young boys to show treatment effect.
Primary Author and Speaker: Natalie Silverman
Contributing Authors: Michelle Beck, Danielle Hall, Amy Hartman, Roxanna Bendixen
PURPOSE: Multiple motor function and strength assessment tools exist for the evaluation of boys with Duchenne muscular dystrophy (DMD) and are typically used in a clinic setting to determine disease progression. These clinical assessments include time to stand, time to climb, 10-meter walk/run, North Star Ambulatory Assessment, and the Six-minute Walk Test. These assessments are psychometrically sound, but do not assess functional ability throughout the day/night within the natural community environment. The objective of this research project is to establish the potential of the ActiGraph GT9x, a research grade activity monitor, as an outcome measure to quantify and detect change in daily activity and sleep patterns during a clinical drug trial in young boys with DMD.2 This research will provide information on daytime/nighttime activity of boys with DMD in their everyday lives and while enrolled in a 12-month clinical trial. The ability to collect data on activity in the natural environment is essential for monitoring meaningful change and response to treatment.1
DESIGN: Longitudinal exploratory study to determine the feasibility, acceptability, and reliability of using an activity monitor in very young children involved in a 12-month clinical drug trial (Vamorolone NCT03439670). Data are being gathered in the natural community environment for boys aged 4-7 years with DMD who are enrolled in the clinical trial.
METHODS: Participants enrolled in the Vamorolone trial may participate in this study, and recruitment occurs during consenting for the Vamorolone trial. Once consented, participants are instructed to wear the ActiGraph day and night throughout the clinical trial for at least 10 hours per day, while sleeping, and during all clinical assessments. Proxy-reports include 1) the PROMIS Parent Proxy – Fatigue survey; 2) the Children’s Sleep Habits Questionnaire (CSHQ); and 3) proxy-report of daily wear time compliance and user satisfaction with the Actigraph. Data collected from the ActiGraph are uploaded daily to a secure cloud-based website. All clinical assessments occur in the corresponding clinic where the child is participating in the Vamorolone trial. Data from the clinical assessment measures of function and mobility will be correlated with the ActiGraph (reliability) at the 12-month time-point. The ActiGraph data will report on daily activity levels (sedentary, moderate, vigorous) and nighttime percent sleep efficiency.
RESULTS: Currently, 5 participants (mean age 5.2 years (SD=1.1)) are enrolled. Participants are geographically from the East and West coasts of the United States. Of the 5 participants, 2 have completed the first 24-week phase having worn the ActiGraph with an average of 54% compliance. A high percentage of our parents (39%) report that fatigue “sometimes” affects their child’s ability to play and engage in activities. Nighttime awakenings and daytime tiredness were also frequently reported.
CONCLUSION: Activity monitors are one method to systematically measure contextually relevant meaningful performance in daily activities, as well as sleep efficiency.2 While actigraphy has been shown to be a reliable measurement tool for activity monitoring, further examination is still required as it pertains specifically to the DMD population and its use in a clinical drug trial.
IMPACT: By using Actigraphy over the course of this longitudinal study, daytime activity performance data, along with nighttime sleep patterns, can be collected and analyzed to better understand the community-based changes in daytime/nighttime patterns that may be related to treatment in boys with DMD.
References
1. Jimenez-Moreno, A. C., Newman, J., Charman, S. J., Catt, M., Trenell, M. I., Gorman, G. S., . . . Lochmüller, H. (2017). Measuring Habitual Physical Activity in Neuromuscular Disorders: A Systematic Review. Journal of Neuromuscular Diseases, 4(1), 25-52. doi:10.3233/jnd-160195
2. Stephens, S., Pullenayegum, E., Schneiderman, J., Mccrindle, B., Abad, A., Ignas, D., . . . Feldman, B. (2016). Validation of Accelerometer Prediction Equations in Children with Chronic Disease. Pediatric Exercise Science, 28(1), 117-132. doi:10.1123/pes.2014-0185
