A functional scale for spinal and bulbar muscular atrophy: Cross-sectional and longitudinal study.
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.
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan.
Atsushi
A
Hashizume
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan.
Masahisa
M
Katsuno
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan.
Keisuke
K
Suzuki
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan; Institute for Advanced Research, Nagoya University, Nagoya 464-8601, Japan.
Haruhiko
H
Banno
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan.
Noriaki
N
Suga
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan.
Tomoo
T
Mano
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan.
Amane
A
Araki
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan.
Yasuhiro
Y
Hijikata
Neurogenetics Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA.
Christopher
C
Grunseich
Neurogenetics Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA.
Angela
A
Kokkinis
Biostatistics Section, Center for Advanced Medicine and Clinical Research, Nagoya University Graduate School of Medicine, Nagoya, Japan.
Akihiro
A
Hirakawa
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan.
Hirohisa
H
Watanabe
Department of Speech Pathology and Audiology, Aichi-Gakuin University School of Health Science, 12 Araike, Iwasaki-cho, Nisshin 470-0195, Japan.
Masahiko
M
Yamamoto
Neurogenetics Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA.
Kenneth H
KH
Fischbeck
Department of Neurology, Nagoya University Graduate School of Medicine, Nagoya 466-8550, Japan. Electronic address: sobueg@med.nagoya-u.ac.jp.
Gen
G
Sobue
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D003430:Cross-Sectional Studies; D018450:Disease Progression; D005163:Factor Analysis, Statistical; D006801:Humans; D007564:Japan; D007802:Language; D008137:Longitudinal Studies; D008297:Male; D008875:Middle Aged; D020966:Muscular Disorders, Atrophic / Q000175:diagnosis*; D015203:Reproducibility of Results; D012720:Severity of Illness Index
Functional rating scale; Motor neuron disease; Spinal and bulbar muscular atrophy; Validation