Muscular Dystrophy Association U.S. Neuromuscular Disease Registry - Preliminary Findings (P7.008)

OBJECTIVE: To track, compare and improve patient care while gaining insight into natural history of disease states through a comprehensive neuromuscular disease registry across MDA clinics in the U.S. BACKGROUND: MDA began piloting its web-based clinical registry in January 2013 with 18 MDA-sponsored clinics entering demographic, genetic and clinical data for patients with ALS, Duchenne and Becker muscular dystrophy (DBMD), and SMA. The MDA Registry Advisory Board (RAB), comprising nine clinical and scientific professionals, maintains long-term oversight of the registry. The registry will be expanded to include all clinics and all diseases supported by MDA. DESIGN/METHODS: All patients with ALS, DBMD and SMA who are seen in the 18 pilot clinics are invited to participate. Demographic, diagnostic and longitudinal clinical information is collected on a prospective basis. Aggregate, de-identified data will be analyzed: descriptive statistics will be used to analyze and display demographic data and inferential and quantitative statistics will be used to analyze trends in patient care and compliance with care standards. RESULTS: Following informed consent, 442 patients have been entered into the registry. Of those with ALS (n=121), 56% of patients are male, 44% are female; average age of symptom onset is 56.9; average age at diagnosis is 57.6 years. 78.3% are Caucasian, 94.8% of patients have no known family history of ALS. Riluzole is taken by 43%, 74% do not use any nutritional therapies, and average BMI is 26.5. Mental status exam was performed for 27.4% of patients; ALS FRS was performed for 85.7%. Data for individuals with DBMD and SMA will also be presented. CONCLUSIONS: Demographics of patients in the registry are currently skewed to those newly diagnosed; data illustrate opportunities to obtain longitudinal clinical information and inform standards of care for neuromuscular diseases. All data will be updated prior to presentation and compared to other surveillance efforts. Study Supported by: Muscular Dystrophy Association Disclosure: Dr. Wolff has nothing to disclose. Dr. Dalton has nothing to disclose. Dr. Cwik has nothing to disclose. Dr. Kennedy has nothing to disclose. Dr. Morgan has nothing to disclose. Dr. Larkindale has nothing to disclose. Dr. Beggs has nothing to disclose. Dr. Benditt has nothing to disclose. Dr. Berry has received personal compensation for activities with Oakstone Publishing, the Muscular Dystrophy Association, and ALS Therapy Alliance. Dr. Crawford has nothing to disclose. Dr. Munson has nothing to disclose. Dr. Richesson has nothing to disclose. Dr. Rosenfeld has received personal compensation for activities with Avanir Pharmaceuticals, Cytokinetics, Hill-Rom, Inc., Gerson Lehrman Group and Guidepoint Global Group. Dr. Rosenfeld has received research support from Hill-Rom, Inc. Dr. Siskind has nothing to disclose. Dr. Flanigan has received personal compensation for activities with Pfizer Inc. Dr. Flanigan has received research support from PTC Therapeutics, Halo Therapeutics, and GlaxoSmithKline, Inc.