SOCIAL AND DEMOGRAPHIC DETERMINANTS OF DISEASE BURDEN AMONG ADULTS WITH SPINAL MUSCULAR ATROPHY
Author(s)
Erin F Welsh, MPH, Sarah M. Whitmire, MS, Mary Curry, ND, Lisa Belter, MPH.
Cure SMA, Schaumburg, IL, USA.
Cure SMA, Schaumburg, IL, USA.
OBJECTIVES: Approved disease modifying therapies have improved survival, motor function, and quality of life in individuals with spinal muscular atrophy (SMA), but social determinates of health (SDOH) may also influence overall well-being and health outcomes. Understanding how SDOH contribute to differences in disease burden is essential for developing comprehensive care strategies for this population. This analysis examined the relationship between SDOH and patient-reported disease burden in adults with SMA Types 2 and 3.
METHODS: This cross-sectional analysis utilized self-reported data collected through the Cure SMA 2025 Annual Community Update Survey. Disease burden was measured by the Spinal Muscular Atrophy Health Index (SMA-HI), where higher scores indicate higher disease burden. A linear regression model was used to measure the impact of SDOH on SMA-HI scores.
RESULTS: The final sample included 127 adults with SMA. After adjusting for SMA type, age, and treatment status, several SDOH were associated with differences in disease burden. Individuals with income of $41,000 - $70,000 had significantly lower SMA-HI scores compared to those earning less than $41,000 (β = -16.8, p=0.009). Part-time employment was associated with higher SMA-HI scores relative to full-time employment (β = 12.1, p=0.047). Non-white individuals had lower SMA-HI scores compared to White individuals (β = -9.7, p=0.019). No significant associations were observed for higher income brackets, unemployment, urban or rural residence, SMA type, age, or treatment status.
CONCLUSIONS: A few of the SDOH evaluated were modestly associated with disease burden in adults with SMA, independent of clinical factors. These findings suggest that socioeconomic and demographic factors may contribute to disparities in SMA-related outcomes and should be considered when assessing and supporting patients. Further research with larger and more clinically diverse SMA populations is warranted to better understand the impact of SDOH across the full spectrum of disease severity.
METHODS: This cross-sectional analysis utilized self-reported data collected through the Cure SMA 2025 Annual Community Update Survey. Disease burden was measured by the Spinal Muscular Atrophy Health Index (SMA-HI), where higher scores indicate higher disease burden. A linear regression model was used to measure the impact of SDOH on SMA-HI scores.
RESULTS: The final sample included 127 adults with SMA. After adjusting for SMA type, age, and treatment status, several SDOH were associated with differences in disease burden. Individuals with income of $41,000 - $70,000 had significantly lower SMA-HI scores compared to those earning less than $41,000 (β = -16.8, p=0.009). Part-time employment was associated with higher SMA-HI scores relative to full-time employment (β = 12.1, p=0.047). Non-white individuals had lower SMA-HI scores compared to White individuals (β = -9.7, p=0.019). No significant associations were observed for higher income brackets, unemployment, urban or rural residence, SMA type, age, or treatment status.
CONCLUSIONS: A few of the SDOH evaluated were modestly associated with disease burden in adults with SMA, independent of clinical factors. These findings suggest that socioeconomic and demographic factors may contribute to disparities in SMA-related outcomes and should be considered when assessing and supporting patients. Further research with larger and more clinically diverse SMA populations is warranted to better understand the impact of SDOH across the full spectrum of disease severity.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR189
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Rare & Orphan Diseases