title: Kennedy's disease 1234 scale: Preliminary design and test.
abstract: Kennedy's disease (KD), also known as spinal and bulbar muscular atrophy (SBMA), is a rare x-linked genetic disorder which is characterized by muscle weakness and atrophy. In previous clinical trials, KD patients had been assessed using the ALSFRS scale, which was specifically designed for ALS patients. However, the progression of KD is very slow, and thus, the ALSFRS does not accurately reflect changes in the clinical condition of KD patient. Here, we developed the KD 1234 scale which designed specially for KD. We evaluated KD 1234 and ALSFRS in 81 KD patients and 100 healthy age-matched male participants. Of 81 cases, 52 were followed up after 32months. Scale reliability was assessed using inter-rater reliability, split-half reliability and internal consistency reliability (Cronbach's α coefficient). Scale validity was evaluated using content and structure validity. Longitudinal analysis was performed. The results showed the KD 1234 scale was simple and easy to operate, with good reliability, validity and reactivity. We think this new scale can quantitatively evaluate the clinical condition of KD patients and any changes in this condition, and is thus suitable for clinical studies.
authors.affiliation: Department of Neurology, Peking University Third Hospital, Beijing, China. Electronic address: brian0317@sina.com.
authors.forename: Ming
authors.initials: M
authors.lastname: Lu
authors.affiliation: Department of Neurology, Civil Aviation General Hospital, Beijing, China.
authors.forename: Haixiao
authors.initials: H
authors.lastname: Guo
authors.affiliation: Department of Neurology, Peking University Third Hospital, Beijing, China.
authors.forename: Dongsheng
authors.initials: D
authors.lastname: Fan
mesh_terms: D000328:Adult; D000368:Aged; D055534:Bulbo-Spinal Atrophy, X-Linked / Q000473:pathology*; D018450:Disease Progression; D006801:Humans; D008297:Male; D008875:Middle Aged; D018908:Muscle Weakness; D009460:Neurologic Examination / Q000592:standards*; D015203:Reproducibility of Results
keywords: Amyotrophic Lateral Sclerosis Functional Rating Scale; Biomarker; Kennedy’s disease; Spinal and bulbar muscular atrophy