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The Spinal Muscular Atrophy Health Index: A novel outcome for measuring how a patient feels and functions
- 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
- Subjects :
- 0301 basic medicine
Adult
Male
medicine.medical_specialty
Adolescent
Physiology
Intraclass correlation
Disease
030105 genetics & heredity
Severity of Illness Index
Muscular Atrophy, Spinal
03 medical and health sciences
Cellular and Molecular Neuroscience
Young Adult
0302 clinical medicine
Cronbach's alpha
Quality of life
Physiology (medical)
medicine
Humans
Patient Reported Outcome Measures
Disease burden
Reliability (statistics)
Aged
business.industry
Spinal muscular atrophy
Middle Aged
SMA
medicine.disease
Cross-Sectional Studies
Physical therapy
Disease Progression
Female
Neurology (clinical)
business
030217 neurology & neurosurgery
Subjects
Details
- ISSN :
- 10974598
- Volume :
- 63
- Issue :
- 6
- Database :
- OpenAIRE
- Journal :
- MusclenerveREFERENCES
- Accession number :
- edsair.doi.dedup.....b4ce4d21813b3a9799551b1f25d815d3