The purpose of this study is to present the overall Health Utility Index (HUI) scores by treatment status for those affected with spinal muscular atrophy (SMA) using the data collected in the Cure SMA community update survey.  

Cure SMA is a non-profit organization that works towards funding and directing research in treatment and care of SMA. In March 2019, Cure SMA launched their third annual community update survey. The survey included questions on clinical trial participation, treatment use, and the HUI questionnaire. The HUI is a generic health-related quality of life (HRQoL) measure that assesses the respondent's functional status in eight dimensions (vision, hearing, speech, mobility, dexterity, emotion, cognition, and pain) for those ages 5 and older.¹ HUI scores range from 0.00 (death) to 1.00 (perfect health).

HUI scores will be provided across all participants and within participants’ type (self-reported or proxy scores). HUI scores will also be described and compared within SMA type, and by treatment. Treatment is defined as those who participated in a clinical trial (regardless of trial arm) and/or used a commercially available treatment.

281 surveys with completed HUI questionnaires were analyzed. The majority of surveys were completed by someone with SMA type II, followed by type III then type I (50.5%, 35.6%, and 13.9%, respectively). The overall HUI2 scores was 0.37, 0.49, and 0.58 for untreated SMA and 0.43, 0.51, and 0.66 for treated SMA types I, II and III, respectively. The overall HUI3 scores was -0.05, 0.18, and 0.35 for untreated SMA and 0.01, 0.25, and 0.46 for treated SMA types I, II and III, respectively.

Findings from this study will assist future cost benefit analyses. However, due to the differences across SMA phenotypes, a study evaluating what HRQoL tool(s) is best suited for the SMA sub-populations is needed.