The objective of this study was to analyze the Health Utilities Index Mark 2 (HUI2) results and the EQ-5D results from the 2020 Cure SMA Community Update Survey.

In April 2020, Cure SMA launched their fourth annual community update survey. The purpose of the survey was to collect longitudinal data on the health states of individuals living with SMA using the HUI2 and EQ-5D. The scores of both the HUI and the EQ-5D range from 0.00 (death) to 1.00 (perfect health).

Data was collected through Survey Gizmo, an online survey platform. Members of the Cure SMA database were invited to complete the survey through email and social media invites.

The HUI2 was completed for 296 affected adults. The mean (sd) scores were 0.35 (0.12), 0.46 (0.09), and 0.61 (0.19) for type I, II, and III, respectively; 0.49 (0.17), 0.50 (0.16), and 0.62 (0.20) for 2, 3, and 4 SMN2 copies, respectively; and 0.45 (0.09), 0.47 (0.12), 0.47 (0.18), and 0.70 (0.20) for non-sitters, sitters, standers, and walkers, respectively. The EQ-5D was completed for 339 affected adults. The mean (sd) EQ-5D scores were 0.17 (0.08), 0.31 (0.19), 0.59 (0.26) for type I, II, and III, respectively; 0.36 (0.22), 0.37 (0.25), and 0.57 (0.30) for 2, 3, and 4 SMN2 copies, respectively; and 0.30 (0.19), 0.37 (0.22), 0.47 (0.30), and 0.72 (0.16) for non-sitters, sitters, standers, and walkers, respectively.

Findings from this study highlight the severe burden experienced among the SMA community. The scores of both instruments are inversely correlated with disease severity, with the most affected scoring the lowest in health states. Additionally, the results from this study show these instruments are more sensitive to differences in SMA types but not SMN2 copy number nor current motor function.