Abstract Details

Title Symptoms and Complications Over Three Years among Later Childhood, Adolescent and Adult Spinal Muscular Atrophy Patients: A Natural History Study within U.S. Hospitals

Topic Child Neurology and Developmental Neurology

Presentation(s) P1 - Poster Session 1 (5:30 PM-6:30 PM)

Poster/Presentation
Number 7-051

Objective Characterize the natural history of spinal muscular atrophy (SMA) for later childhood, adolescent and adult patients in U.S. hospital settings through the assessment of symptoms/complications over three years.

Background Limited information is available on disease progression among older SMA patients, particularly adults.

Design/Methods The study population included patients of all ages with inpatient and/or hospital outpatient discharge records and ≥2 primary or secondary SMA ICD-9 codes ≥30 days apart in the Premier Healthcare Database (2007–2014). Index date was the date of the first SMA ICD-9 code. Patients included here were those classified into three groups based on age at index: later childhood (4-12 years), adolescent (13-17 years), and adult (>18 years). The frequency of SMA-related symptoms/complications was assessed for each group 12 months pre- through 24 months post-index to characterize disease progression.

Results

659 patients, from 337 U.S. hospitals, met inclusion criteria for these analyses: 144 later childhood, 69 adolescent, and 446 adult patients. Overall, the most frequent symptoms/complications over the three-year period included respiratory symptoms, scoliosis, ventilation support, pneumonia, and gastro/reflux. Although the most common symptom 24 months post-index differed across age groups (later childhood: scoliosis, adolescents: ventilation support, adults: respiratory), the overall pattern was for increased symptoms/complications from the pre-index period. Ventilation support increased most substantially over three years (overall: 2.0% to 16.9%, later childhood: 0.7% to 20.0%, adolescents: 4.3% to 23.1%, adults: 2.0% to 14.9%) followed by scoliosis (overall: 5.2% to 18.3%, later childhood: 8.3% to 31.9%, adolescents: 7.2% to 20.0%, adults: 3.8% to 13.7%) and respiratory symptoms (overall: 5.6% to 18.5%, later childhood: 2.8% to 11.1%, adolescents: 5.8% to 20.0%, adults: 6.5% to 20.6%).

Conclusions Findings are consistent with increasing disease burden over time and suggest the potentially rapid progressive nature of SMA for later childhood, adolescent, and adult patients with hospital interactions.

Authors/Disclosures
Angela Paradis PRESENTER	Angela Paradis has received personal compensation for serving as an employee of Biogen. An immediate family member of Angela Paradis has received personal compensation for serving as an employee of Biogen. Angela Paradis has received stock or an ownership interest from Biogen. An immediate family member of Angela Paradis has received stock or an ownership interest from Biogen.
	No disclosure on file
	No disclosure on file
Sandra P. Reyna, MD	Dr. Reyna has received personal compensation for serving as an employee of Novartis Gene Therapies.
Nicole C. Johnson, MD (Harbor UCLA)	Dr. Johnson has nothing to disclose.
Mehul Jhaveri	Mehul Jhaveri has received personal compensation for serving as an employee of Biogen. Mehul Jhaveri has received stock or an ownership interest from Biogen.

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