Abstract Details

Title Predonisone Therapy for Duchenne Muscular Dystrophy: A Large-Cohort Study with Patient Registry

Topic Muscle Disease/Neuromuscular Junction

Presentation(s) (-)

Poster/Presentation
Number 002

Objective

Background It is nearly four decades since the potential benefit of glucocorticoids in DMD. Some studies supported short-term effect of predonisone therapy for functional benefits, but long-term effect not yet evaluated in large-size study. We investigated the long-term effect of predonisone therapy in a retrospective study with patient registry.

Design/Methods We used the retrospective data in registry of muscular dystrophy (Remudy) from July 2009 to June 2012 in Japan. The subjects were selected by the eligible criteria of DMD: (1) absent dystrophin on muscle biopsy, (2) Duchenne-type mutation in the dystophin gene. We excluded the subjects who had additional disease comorbidities, such as adrenal hypoplasty and nephrotic syndrome and less than 5 years old and over 40 years old patients. We compared the subjects with or without predonisone therapy for loss of ambulation. The time to event with loss of ambulation was demonstrated by the Kaplan-Meier method and calculated by the log-rank test. Also, we performed COX regression analysis for adjustment.

Results 560 DMD patients were included in this study. There were 245 patients with predonisone therapy and 315 patients without predonisone therapy. The loss of ambulation in the Predonisone group was median 132(range 126-138) months, whereas that of the non-Predonisone group was median 121(range 120-126) months (p=0.0002 with log-rank test) in Kaplan-Meire method. Adjusted Hazard ratio was 0.64 (95% Confidence intervals 0.50-0.82, p=0.0005).

Conclusions Our study with patient registry described predonisone therapy significantly prolonged walking in long-term in Duchenne Muscular Dystrophy patients.

Authors/Disclosures
Fumi Takeuchi PRESENTER	No disclosure on file
	No disclosure on file
	No disclosure on file
Harumasa Nakamura, MD (National Center of Neurology and Psychiatry)	Dr. Nakamura has nothing to disclose.
	No disclosure on file
Hirofumi Komaki	No disclosure on file
Madoka Mori-Yoshimura, MD, PhD (National Center Hospital, National Center of Neurology and Psychiatry)	Dr. Mori-Yoshimura has nothing to disclose.
	No disclosure on file
Ichizo Nishino, MD, PhD, FAAN (National Institute of Neuroscience, NCNP)	The institution of Dr. Nishino has received research support from AMED.
Roland G. Henry, PhD (University of California, San Francisco)	Dr. Henry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MEDDAY. Dr. Henry 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. Dr. Henry has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Henry has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sanofi/Genzyme.
Mitsuru Kawai, MD (NHO Higashisaitama National Hospital)	No disclosure on file
	No disclosure on file

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