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Neuromuscul Disord. 2015 Jul;25(7):554-62. doi: 10.1016/j.nmd.2015.03.008. Epub 2015 Mar 20.

A functional scale for spinal and bulbar muscular atrophy: Cross-sectional and longitudinal study.

Author information

1
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan.
2
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan; Institute for Advanced Research, Nagoya University, Nagoya 464-8601, Japan.
3
Neurogenetics Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA.
4
Biostatistics Section, Center for Advanced Medicine and Clinical Research, Nagoya University Graduate School of Medicine, Nagoya, Japan.
5
Department of Speech Pathology and Audiology, Aichi-Gakuin University School of Health Science, 12 Araike, Iwasaki-cho, Nisshin 470-0195, Japan.
6
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan. Electronic address: sobueg@med.nagoya-u.ac.jp.

Abstract

We aimed to develop, validate, and evaluate a disease-specific outcome measure for SBMA: the Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS). We examined the Japanese version (SBMAFRS-J) in 80 Japanese SBMA subjects to evaluate its validity and reliability. We then assessed this scale longitudinally in 41 additional SBMA subjects. The English version (SBMAFRS-E) was also tested in 15 US subjects. The total score of the SBMAFRS-J was distributed normally without an extreme ceiling or floor effect. For SBMAFRS-J, the high intra- and inter-rater agreement was confirmed (intra-class correlation coefficients [ICCs] 0.910 and 0.797, respectively), and internal consistency was satisfactory (Cronbach's alpha 0.700-0.822). In addition, SBMAFRS-J demonstrated concurrent, convergent, and discriminant validity, except for the respiratory subscale. The inter-rater reliability and internal consistency of SBMAFRS-E were also satisfactory. Longitudinally, SBMAFRS-J showed a higher sensitivity to disease progression than the existing clinical measures. In conclusion, we developed and validated a disease-specific functional rating scale for SBMA in both Japanese and English versions, although it needs to be re-assessed in interventional studies with a larger sample size including English speaking subjects.

KEYWORDS:

Functional rating scale; Motor neuron disease; Spinal and bulbar muscular atrophy; Validation

PMID:
25913211
PMCID:
PMC5608513
DOI:
10.1016/j.nmd.2015.03.008
[Indexed for MEDLINE]
Free PMC Article

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