日本版ALSFRS-Rを用いた電話調査によるALS患者の評価―広域的前向き縦断調査への応用

Development of a Telephone Survey System for Patients with Amyotrophic Lateral Sclerosis Using the ALSFRS-R (Japanese version) and Application of This System in a Longitudinal Multicenter Study

Ⅰ．背景・目的

　筋萎縮性側索硬化症（amyotrophic lateral sclerosis：ALS）は成人発症の神経変性疾患であり，進行性の上位および下位運動ニューロン変性をきたす。その結果，全身の筋萎縮，筋力低下をきたし，呼吸筋麻痺による呼吸不全などによって発症後平均3～4年程度で死亡もしくは人工呼吸器装着が必要となる神経難病である。

　ALSに対する根治的な治療法はいまだ存在しないが，唯一リルゾールは有効性が示されている。また，嚥下障害に対して経管栄養による栄養確保，呼吸障害に対して非侵襲的陽圧換気（noninvasive positive pressure：NPPV）の活用など適切な栄養，呼吸補助を行うことでquality of life（QOL）の確保，生存期間の延長が可能な場合がある。またALSの診療においては，胃瘻造設，気管切開や人工呼吸器装着など侵襲的処置を行うか否か，行う場合にどのタイミングで実施するかが経過に大きな影響を及ぼす。

Abstract

　To investigate the various clinical courses of patients with amyotrophic lateral sclerosis (ALS), we developed a telephone survey system for determining the activities of daily living (ADL) status of patients with ALS.　In this system, every 3 months, clinical research coordinators (CRCs) conducted a telephone survey using the flow charts of the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R; Japanese version).　To confirm the reliability of the results of the telephone survey, we compared the scores of 37 ALS patients obtained in the telephone surveys (telephone scores) to the scores obtained during clinical examinations by neurologists (neurologists' scores).　The mean (SD) age of the included patients was 62.6 (11.8) years.　Eighteen patients answered the surveys themselves, whereas the primary caregivers of 19 patients answered the surveys for the patients.　Before the study, we informed the CRCs of the study plan, general knowledge of ALS, procedures of the telephone survey, ethical issues relevant to the study, and requisite considerations for ALS patients and caregivers.

　The intraclass correlation coefficient (ICC) between the telephone scores and the neurologists' scores in the 37 ALS patients was 0.97 (95% confidence interval ［CI］, 0.94-0.98).　The kappa statistics of 12 questions of the ALSFRS-R between the telephone scores and the neurologists' scores ranged from 0.58 to 0.85.　The ICC of the scores of the 18 cases in which the patients answered the telephone questions themselves was 0.96 (95% CI, 0.89-0.98).　The ICC of the scores of the 19 cases in which the caregivers answered the telephone questions was 0.97 (95% CI, 0.92-0.99).　These results showed a good reliability of the telephone survey, regardless of whether the patients themselves or the caregivers answered telephone questions.

　In 2006, a longitudinal multicenter study of Japanese ALS patients was initiated called the Japanese Consortium for Amyotrophic Lateral Sclerosis research (JaCALS).　In the JaCALS, prospective clinical information was obtained using this telephone survey system.　Of the 284 ALS patients who were registered at JaCALS over a year ago, 93% were followed up and provided ALSFRS-R scores and information relevant to prognosis.

　We have established an efficient and reliable telephone survey system for studying the longitudinal clinical courses of patients with ALS.