Retrospective Analysis of Healthcare Resource Utilization (HRU) in Patients with Spinal Muscular Atrophy (SMA) in MarketScan® (P3.186)

Objective: To characterize HRU among patients with SMA in claims data. Background: SMA is a debilitating rare, genetic neuromuscular disease. HRU in SMA is high due to increasing muscle weakness and atrophy, with eventual respiratory and nutritional failure. Design/Methods: We conducted a retrospective analysis of patients with SMA in the Truven Health MarketScan® Databases, which consists of commercial claims (January 2010 – September 2015) and multi-state Medicaid claims (January 2010 – December 2014). Cohort A included patients born after January 2010 with ≥2 diagnoses for SMA (ICD-9: 335.0, 335.1×) ≥31days apart. Cohort B patients had ≥2 diagnoses of SMA ≥31days apart, born prior to 2010, and ≤18years in 2015. Descriptive statistics were used. Results: 3,142 patients had ≥2 claims for SMA ≥31days apart, 314 met inclusion for Cohort A and 916 for Cohort B. COHORT A: Mean age 1.2years, 54% male, average follow-up time 22.7months. 69% had ≥1 inpatient (IP) admission (average 3.1 per patient), mean length of stay (LOS) 13 days, maximum 183 days. 60.2% had ER visits (average 3.7 per patient). Top IP claims were respiratory abnormality (39%), pulmonary collapse (30.3%), respiratory failure (30.3%), and feeding difficulties/management (29%). 54% of patients had claims for suction machines, 44% cough assist, 39% IPPB, and 32% BiPAP/CPAP. COHORT B: Mean age 12years, 50% male, average follow-up time 35.8 months. 47.4% had ≥1 IP admission, average 3.2, mean LOS 8days and maximum 122days. 60.3% had ER visits (average 3.5 per patient). Top IP claims were respiratory abnormality (24.3%), pneumonia (23.8%), pulmonary collapse (19.8%), scoliosis (19%), and respiratory failure (18.2%). 60% of patients had diagnostic codes for scoliosis, and 47% had procedure claims related to scoliosis. Conclusions: HRU in SMA focuses around respiratory support and care, with increasing orthopedic resource use driven by onset and management of scoliosis in older patients. Study Supported by: Funded by Biogen. Disclosure: Dr. Teynor has received personal compensation for activities with Biogen as an employee. Dr. Teynor holds stock/stock options in Biogen. Dr. Zhou has received personal compensation for activities with Biogen Idec. Dr. Zhou has received research support from Biogen Idec. Dr. Hou has received personal compensation for activities with Biogen as an employee. Dr. Wells has received personal compensation for activities with Biogen as an employee. Dr. Hall has received personal compensation for activities with Biogen Idec as an employee. Dr. Hall holds stock and/or stock options in Biogen Idec. Dr. Avendano has received personal compensation for activities with Biogen as an employee. Dr. Avendano holds stock and/or stock options in Biogen.