Back to Search Start Over

The Spinal Muscular Atrophy Health Index: A novel outcome for measuring how a patient feels and functions

Authors :
Michael Hunter
Elizabeth Luebbe
Christine Zizzi
Craig M. Zaidman
Emma Ciafaloni
Valeria A. Sansone
Nicholas E. Johnson
Phillip Mongiovi
Nuran Dilek
Connie Garland
Chad Heatwole
Michael P. McDermott
John T. Kissel
Source :
MusclenerveREFERENCES. 63(6)
Publication Year :
2021

Abstract

INTRODUCTION The Spinal Muscular Atrophy Health Index (SMA-HI) is a multifaceted, disease-specific, patient-reported outcome to measure an SMA patient's perception of their disease burden. In preparation for upcoming therapeutic trials, we examine the validity, reliability, and usability of the SMA-HI in adults, teenagers, and children with SMA. METHODS Using data from a cross-sectional study of 359 international adult patients with SMA, we identified the most relevant symptoms to include in the SMA-HI. We utilized factor analysis, patient interviews with adults and minors (age 8-15 years), known-group validity testing, and test-retest reliability assessments to evaluate and refine the SMA-HI. RESULTS The SMA-HI measures overall disease burden and 15 areas of SMA health. Fifteen adult patients and five patients, age 8 to 15 years, participated in semistructured qualitative interviews and found the SMA-HI to be comprehensive, easily completed, and to have clear meaning. The final SMA-HI and its subscales demonstrated good internal consistency (Cronbach α = 0.77-0.96), high test-retest reliability (intraclass correlation coefficient = 0.60-0.96), and an ability to differentiate between SMA groups with different disease severities affecting areas such as employment and ambulation (P

Details

ISSN :
10974598
Volume :
63
Issue :
6
Database :
OpenAIRE
Journal :
MusclenerveREFERENCES
Accession number :
edsair.doi.dedup.....b4ce4d21813b3a9799551b1f25d815d3