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Abstract Details

LEOPARD-DMD: Six-Month Results of Longitudinal Study of Disease Progression of Duchenne Muscular Dystrophy through the Patient and Caregiver Perspective
Child Neurology and Developmental Neurology
P7 - Poster Session 7 (5:00 PM-6:00 PM)
6-007

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. 

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.
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.     
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. 
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.
Authors/Disclosures
Christina Shupe (Center for Health and Technology, University of Rochester)
PRESENTER 		Ms. Shupe has nothing to disclose.
Charlotte Engebrecht (University of Rochester Center for Health + Technology) Ms. Engebrecht has nothing to disclose.
Jennifer Weinstein Jennifer Weinstein has nothing to disclose.
Anika Varma (Center for Health + Technology, University of Rochester) Anika Varma has nothing to disclose.
Spencer Rosero (University of Rochester, Center for Health and Technology) Spencer Rosero has nothing to disclose.
Charlotte Irwin Ms. Irwin has nothing to disclose.
Preshetha Kanagaiah (University of Rochester Center for Health and Technology) Miss Kanagaiah has nothing to disclose.
Judith Monickaraj, MS Ms. Monickaraj has nothing to disclose.
Alicia Brocht (University of Rochester, Center for Health and Technology (CHeT)) Alicia Brocht has nothing to disclose.
Peggy Auinger (University of Rochester) Ms. Auinger has nothing to disclose.
Debra Guntrum, MS, FNP (University of Rochester Medical Center) Ms. Guntrum has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avexis. Ms. Guntrum has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Ms. Guntrum has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC.
Emma Ciafaloni, MD, FAAN (University of Rochester Medical Center) Dr. Ciafaloni has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx, Alexion, Sarepta, UCB, Hoffman-LaRoche, Biogen. Dr. Ciafaloni has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis, AnnJi Pharmaceutical, ML-BIO, Avidity. The institution of Dr. Ciafaloni has received research support from CDC, CureSMA, FDA, Orphazyme, Sarepta, PCORI, Neurogene. Dr. Ciafaloni has received publishing royalties from a publication relating to health care.
Chad R. Heatwole, MD, FAAN (University of Rochester Medical Center) Dr. Heatwole has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Virginia Commonwealth University. Dr. Heatwole has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Legal Med. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurocrine. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Swan Bio. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Harmony. Dr. Heatwole has received personal compensation in the range of $0-$499 for serving as a Consultant for Iris. Dr. Heatwole has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Recursion. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity Biosciences. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lupin. Dr. Heatwole has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Neurocrine. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for New York Central Mutual. Dr. Heatwole has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Penn Prop and Gas. The institution of Dr. Heatwole has received research support from Department of Defense. The institution of Dr. Heatwole has received research support from Novartis. The institution of Dr. Heatwole has received research support from MJFF. The institution of Dr. Heatwole has received research support from FARA. The institution of Dr. Heatwole has received research support from NIH. The institution of Dr. Heatwole has received research support from University of Miami. The institution of Dr. Heatwole has received research support from MDA. Dr. Heatwole has received intellectual property interests from a discovery or technology relating to health care.