Abe Koji, Itoyama Yasuto, Sobue Gen, Tsuji Shoji, Aoki Masashi, Doyu Manabu, Hamada Chikuma, Kondo Kazuoki, Yoneoka Takatomo, Akimoto Makoto, Yoshino Hiide
Department of Neurology, Okayama University Hospital , Okayama.
Amyotroph Lateral Scler Frontotemporal Degener. 2014 Dec;15(7-8):610-7. doi: 10.3109/21678421.2014.959024. Epub 2014 Oct 6.
Our objective was to confirm the efficacy and safety of edaravone in amyotrophic lateral sclerosis (ALS) patients. We conducted a 36-week confirmatory study, consisting of 12-week pre-observation period followed by 24-week treatment period. Patients received placebo or edaravone i.v. infusion over 60 min for the first 14 days in cycle 1, and for 10 of the first 14 days during cycles 2 to 6. The efficacy primary endpoint was changed in the revised ALS functional rating scale (ALSFRS-R) scores during the 24-week treatment. Patients were treated with placebo (n = 104) and edaravone (n = 102). Changes in ALSFRS-R during the 24-week treatment were -6.35 ± 0.84 in the placebo group (n = 99) and -5.70 ± 0.85 in the edaravone group (n = 100), with a difference of 0.65 ± 0.78 (p = 0.411). Adverse events amounted to 88.5% (92/104) in the placebo group and 89.2% (91/102) in the edaravone group. In conclusion, the reduction of ALSFRS-R was smaller in the edaravone group than in the placebo group, but efficacy of edaravone for treatment of ALS was not demonstrated. Levels and frequencies of reported adverse events were similar in the two groups.
我们的目标是确认依达拉奉对肌萎缩侧索硬化症（ALS）患者的疗效和安全性。我们进行了一项为期36周的验证性研究，包括12周的观察前期，随后是24周的治疗期。在第1周期的前14天，患者接受安慰剂或依达拉奉静脉输注60分钟，在第2至6周期的前14天中的10天接受该治疗。疗效的主要终点是在24周治疗期间修订的ALS功能评定量表（ALSFRS-R）评分的变化。患者分别接受安慰剂治疗（n = 104）和依达拉奉治疗（n = 102）。安慰剂组（n = 99）在24周治疗期间ALSFRS-R的变化为-6.35±0.84，依达拉奉组（n = 100）为-5.70±0.85，差异为0.65±0.78（p = 0.411）。安慰剂组的不良事件发生率为88.5%（92/104），依达拉奉组为89.2%（91/102）。总之，依达拉奉组ALSFRS-R的降低幅度小于安慰剂组，但依达拉奉治疗ALS的疗效未得到证实。两组报告的不良事件水平和发生率相似。
