TY - JOUR
T1 - A functional scale for spinal and bulbar muscular atrophy
T2 - Cross-sectional and longitudinal study
AU - Hashizume, Atsushi
AU - Katsuno, Masahisa
AU - Suzuki, Keisuke
AU - Banno, Haruhiko
AU - Suga, Noriaki
AU - Mano, Tomoo
AU - Araki, Amane
AU - Hijikata, Yasuhiro
AU - Grunseich, Christopher
AU - Kokkinis, Angela
AU - Hirakawa, Akihiro
AU - Watanabe, Hirohisa
AU - Yamamoto, Masahiko
AU - Fischbeck, Kenneth H.
AU - Sobue, Gen
N1 - Publisher Copyright:
© 2015 Elsevier B.V.
PY - 2015/7/1
Y1 - 2015/7/1
N2 - 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.
AB - 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.
UR - https://www.scopus.com/pages/publications/84930541640
UR - https://www.scopus.com/pages/publications/84930541640#tab=citedBy
U2 - 10.1016/j.nmd.2015.03.008
DO - 10.1016/j.nmd.2015.03.008
M3 - Article
C2 - 25913211
AN - SCOPUS:84930541640
SN - 0960-8966
VL - 25
SP - 554
EP - 562
JO - Neuromuscular Disorders
JF - Neuromuscular Disorders
IS - 7
ER -