LEOPARD-DMD: Six-Month Results of Longitudinal Study of Disease Progression of Duchenne Muscular Dystrophy through the Patient and Caregiver Perspective
Christina Shupe1, Charlotte Engebrecht1, Jennifer Weinstein1, Anika Varma1, Spencer Rosero2, Charlotte Irwin1, Preshetha Kanagaiah1, Judith Monickaraj1, Alicia Brocht1, Peggy Auinger1, Debra Guntrum3, Emma Ciafaloni3, Chad Heatwole3
1Center for Health and Technology, University of Rochester, 2University of Utah Spencer Fox Eccles School of Medicine, 3University of Rochester Medical Center
Objective:
To conduct a remote, 2-year longitudinal study with individuals with DMD and caregivers of individuals with DMD to determine track disease burden over time and identify factors associated with a faster or slower progression of disease.
Background:
There is a need for fully-validated, disease-specific patient and observer-reported outcome measures to bolster therapeutic development for Duchenne muscular dystrophy (DMD). The DMD-Health Index (DMD-HI) and DMD Caregiver-Reported- Health Index (DMDCR-HI) are patient and observer-reported outcome measures developed according to FDA guidance for use in clinical trials and drug labelling claims.
Design/Methods:
Individuals with DMD ages 11 years and older and caregivers of individuals with DMD ages 0-21 years are asked to complete DMDCR-HI and DMD-HI semiannually to track multifactorial disease burden. Participants are asked to additionally complete the Peds-QL, a survey preference form, and a global impression of change survey following baseline. At study completion, we will determine the minimal clinically important difference (MCID) for each instrument and identify which demographic features are associated with a faster or slower disease progression.
Results:
Thirty-eight individuals with DMD and 93 caregivers have enrolled in the study. The average DMD participant age is 21 years (range 11-49 years) and average caregiver participant age is 44 years (range 29-75 years). The average age of diagnosis is 5 years old. At baseline, 61% of individuals with DMD and 38% of caregivers report the individual experiences a moderately severe disability. Six-month assessments are currently being administered.
Conclusions:
The DMD-HI and DMDCR-HI are regulatory-grade patient and observer-reported outcome measures designed to quantify multi-systemic disease burden in DMD. Continued data collection and analysis will further define the performance metrics and responsiveness of these outcome measures for optimal use in clinical trials.
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