NEMO (NeuroMuscular Omnicentre) Clinical Center Milano, Fondazione Serena Onlus, Milan, Italy.
"Rita Levi Montalcini" Department of Neuroscience, ALS Centre, University of Turin, Via Cherasco 15, 10126, Turin, Italy.
J Neurol. 2020 Nov;267(11):3258-3267. doi: 10.1007/s00415-020-09993-z. Epub 2020 Jun 17.
The aim of the study is to analyze the ALS disease progression and respiratory function of Italian patients treated with edaravone (EVN), as well as the adherence to, and the effects of, the therapy.
We performed an observational study of patients treated with EVN from May 2017 to May 2019, in 39 Italian ALS Centers. Taking into account ALS patients with at least 12 months of EVN treatment, we compared the decline of ALSFRS-R and FVC with a group of matched historical controls from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database, using both descriptive and survival analysis approaches.
A total of 331 ALS Italian patients treated with EVN and 290 matched historical controls were recruited in this study. No significant differences on disease progression or respiratory function were found comparing the two cohorts in both descriptive and survival analyses. The EVN treatment was overall well tolerated.
The study showed that EVN treatment was well tolerated. No significant differences were reported in ALS patients treated and not treated with EVN, in terms of both disease progression and respiratory function. These findings prove that further studies are required to better clarify whether EVN could be considered an effective treatment for ALS disease.
本研究旨在分析意大利肌萎缩侧索硬化症（ALS）患者接受依达拉奉（EVN）治疗后的疾病进展和呼吸功能，以及治疗的依从性和效果。
我们对 2017 年 5 月至 2019 年 5 月期间在 39 家意大利 ALS 中心接受 EVN 治疗的患者进行了一项观察性研究。考虑到接受 EVN 治疗至少 12 个月的 ALS 患者，我们使用描述性和生存分析方法，将 EVN 组与来自 Pooled Resource Open-Access ALS Clinical Trials（PRO-ACT）数据库的一组匹配历史对照进行比较，比较 ALSFRS-R 和 FVC 的下降情况。
本研究共纳入 331 例接受 EVN 治疗的意大利 ALS 患者和 290 例匹配的历史对照。在描述性和生存分析中，两组在疾病进展或呼吸功能方面均无显著差异。EVN 治疗总体上耐受性良好。
该研究表明 EVN 治疗具有良好的耐受性。在接受和未接受 EVN 治疗的 ALS 患者中，疾病进展和呼吸功能方面均未报告明显差异。这些发现证明需要进一步研究，以更好地阐明 EVN 是否可被视为 ALS 疾病的有效治疗方法。
