Abstract

This study aimed to develop a functional measurement that combines quantitative motor evaluation index of various body regions in patients with spinal and bulbar muscular atrophy (SBMA). We assessed subjects with SBMA and healthy controls with quantitative muscle strength measurements and functional scales. We selected tongue pressure, grip power, % peak expiratory flow (%PEF), timed walking test, and % forced vital capacity (%FVC) as components. By combining these values with Z-score, we created a functional composite (SBMA functional composite: SBMAFC). We also calculated the standardized response mean to compare the sensitivity of SBMAFC with that of existing measurements. A total of 97 genetically confirmed patients with SBMA and 36 age- and sex-matched healthy controls were enrolled. In the longitudinal analysis, the standardized response mean of SBMAFC was larger than that of existing rating scales. Receiver operating characteristic (ROC) analysis demonstrated that the SBMAFC is capable of distinguishing between subjects with early-stage SBMA and healthy controls. SBMAFC is more sensitive to disease progression than existing functional rating scales and is a potential outcome measure in clinical trials of SBMA.

Details

Title
Development of a functional composite for the evaluation of spinal and bulbar muscular atrophy
Author
Inagaki, Tomonori 1 ; Hashizume, Atsushi 2 ; Hijikata, Yasuhiro 1 ; Yamada, Shinichiro 1 ; Ito, Daisuke 1 ; Kishimoto, Yoshiyuki 1 ; Torii, Ryota 1 ; Sato, Hiroyuki 3 ; Hirakawa, Akihiro 3 ; Katsuno, Masahisa 2 

 Nagoya University Graduate School of Medicine, Department of Neurology, Nagoya, Japan (GRID:grid.27476.30) (ISNI:0000 0001 0943 978X) 
 Nagoya University Graduate School of Medicine, Department of Neurology, Nagoya, Japan (GRID:grid.27476.30) (ISNI:0000 0001 0943 978X); Nagoya University Graduate School of Medicine, Department of Clinical Research Education, Nagoya, Japan (GRID:grid.27476.30) (ISNI:0000 0001 0943 978X) 
 Tokyo Medical and Dental University, Department of Clinical Biostatistics, Graduate School of Medical and Dental Sciences, Tokyo, Japan (GRID:grid.265073.5) (ISNI:0000 0001 1014 9130) 
Publication year
2022
Publication date
2022
Publisher
Nature Publishing Group
e-ISSN
20452322
Source type
Scholarly Journal
Language of publication
English
ProQuest document ID
2726152768
Copyright
© The Author(s) 2022. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.