Open label and single cohort study of edaravon on Muro disease (amyotrophic lateral sclerosis and parkinsonism-dementia complex :Kii ALS/PDC) - Edaravon on Muro disease (Kii ALS/PDC)

Background: The purpose of this study was to reveal the clinical effect of edaravone, a free radical scavenger, in patients with amyotrophic lateral sclerosis/parkinsonism-dementia complex in the Kii Peninsula area of Japan (Kii ALS/PDC). Objective: Five patients with Kii ALS/PDC (four men and one woman; mean age, 68.4 years; mean duration of illness, 9.4 years) were included in the study. Method: Edaravone (30 mg per infusion) was administered twice a week for 24 weeks via intravenous drip to the patients with Kii ALS/PDC. Ascorbic acid (2000 mg/day) and tocopherol acetate (300 mg/day) were used concomitantly. Results: Two of the five patients with relatively mild symptoms showed improvement of spontaneity, mental, and physical activity and one of them with the mildest symptoms showed marked improvement on the Unified Parkinson Disease Rating Scale, apathy scale, and Clinical Assessment for Spontaneity. The other patient showed no apparent change. The remaining two bed-ridden patients showed irritability, violence, and hypersexuality; hence, edaravone was discontinued for three months. Discussion: Edaravone may be effective for patients in the mild-to moderate stage of Kii ALS/PDC, but a further large-scale trial will be needed to confirm this finding.