HUI, WPAI, and Neuromuscular PedsQL Scores in patients with Spinal Muscular Atrophy: Findings from the 2019 Cure SMA Community Update Survey


Real World Data - Disease registries, natural history, post marketing surveillance

Poster Number: 159


Lisa Belter, MPH, Rosangel Cruz, Jill Jarecki, PhD


1. Cure SMA, 2. Cure SMA, 3. Cure SMA

Spinal muscular atrophy (SMA) is a rare autosomal disease that affects the motor nerve cells in the spinal cord, which leads to wasting of muscles. New SMA therapies have become available, and so now there is a need to understand the caregiver and patient’s health related quality of life (HRQoL). The purpose of this analysis is to present a baseline analysis of caregiver and patient’s quality of life among the Cure SMA community.

In March 2019, Cure SMA launched their third annual community update survey. The purpose of the community update survey is to collect the most current information from those affected with SMA, such as caregiver burden and patient’s HRQoL. These outcomes were measured through the following validated instruments: Health Utilities Index (HUI), the Neuromuscular PedsQL, and the Work Productivity and Activity Impairment (WPAI) questionnaires.

HUI, PedsQL, and WPAI scores will be described by subtype of SMA and by patient or caregiver perspective. Furthermore, all three scores will be described by maximum motor function ever achieved as well as current motor function. For those with permanent ventilation, their three scores will be compared to those not on permanent ventilation. Lastly, all three scores will be described and compared among those who have had scoliosis surgery to those who have not.

The finding from this study will provide a baseline understanding of the current quality of life experienced by those affected with SMA which will be able to be compared to future quality of life measures as new therapies emerge.

Cure SMA acknowledges the Cure SMA Industry Collaboration. The members of the Cure SMA Industry Collaboration are AveXis, Inc. a Novartis company, Biogen, Genentech/Roche Pharmaceuticals, Scholar Rock and Cytokinetics Inc.