Abstract Details

Title Age at First Symptoms/Signs and Loss of Ambulation in Duchenne-Becker Muscular Dystrophy: Data from the MD STARNet

Topic Muscle Disease/Neuromuscular Junction

Presentation(s) (-)

Poster/Presentation
Number 001

Objective

Background Corticosteroid use has changed the natural history of DBMD by prolonging ambulation: therefore, age at loss of ambulation may no longer be useful in differentiating Duchenne and Becker. The prognostic utility of age at onset of SS has not been well studied.

Design/Methods The study cohort included 825 males with DBMD who were ascertained through the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet), a population-based surveillance system that identified all individuals with DBMD born from 1982 through 2009 and residing within Arizona, Colorado, Iowa, Georgia, Hawaii and Western New York when ascertained. We used a Cox proportional hazard regression model to examine the association between age at onset of first SS and loss of ambulation adjusted for cumulative exposure to steroids, age at start of corticosteroids, birth year, state of residence, and family history of DBMD. Results for associations with outcome are reported as hazard ratios (HR) and their corresponding 95% confidence intervals (CI).

Results Among the 825 individuals in the study cohort, 330 (40%) were still walking at the time of their most recent clinical evaluation. A one year increase in the age at onset of first SS was significantly associated with an 11% reduction in the risk of loss of ambulation (HR 0.89, 95% CI 0.86-0.92, p<0.0001). Multiple regression analyses using the Cox proportional hazard model will be presented that examine this association after adjusting for possible confounders including corticosteroids.

Conclusions Age at onset of first SS was associated with age at loss of ambulation in this large DBMD cohort and may have clinical utility in differentiating Duchenne and Becker penotypes.

Authors/Disclosures
Emma Ciafaloni, MD, FAAN (University of Rochester Medical Center) PRESENTER	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.
Michael McDermott	No disclosure on file
	No disclosure on file
Timothy K. Vartanian, MD, PhD (Weill Cornell Medical College)	Dr. Vartanian has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Vartanian has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Genentech. Dr. Vartanian has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis. Dr. Vartanian has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Tisch MS Center. Dr. Vartanian has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen.
	No disclosure on file
Shree Pandya, DPT	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
Christopher Cunniff, MD (Weill Cornell Medicine)	No disclosure on file
Katherine D. Mathews, MD, FAAN (University of Iowa - Dept of Pediatrics)	Dr. Mathews has received personal compensation for serving as an employee of Avidity Bioscience. The institution of Dr. Mathews has received research support from NIH. The institution of Dr. Mathews has received research support from Centers for Disease Control and Prevention. The institution of Dr. Mathews has received research support from Muscular Dystrophy Association . The institution of Dr. Mathews has received research support from Friedreich's Ataxia Research Alliance . The institution of Dr. Mathews has received research support from Sarepta . The institution of Dr. Mathews has received research support from Pfizer. The institution of Dr. Mathews has received research support from Reata . The institution of Dr. Mathews has received research support from PTC Therapeutics, Inc. The institution of Dr. Mathews has received research support from Italfarmaco . The institution of Dr. Mathews has received research support from AMO. The institution of Dr. Mathews has received research support from FibroGen. The institution of Dr. Mathews has received research support from Capricor. The institution of Dr. Mathews has received research support from edgewise. The institution of Dr. Mathews has received research support from biogen.
	No disclosure on file
	No disclosure on file

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