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评价依达拉奉治疗肌萎缩侧索硬化症的临床疗效和安全性:一项 72 周单中心经验。

Evaluation of clinical outcome and safety profile of edaravone in treatment of amyotrophic lateral sclerosis: a 72-week single-center experience.

机构信息

Department of Neurology, Ibn Sina Hospital, Gamal Abdel Nasser Street, Sabah Medical Area, Box 25427, 13115, Kuwait City, Kuwait.

Department of Neurology, Beni-Suef University, Bani Sweif, Egypt.

出版信息

Acta Neurol Belg. 2021 Dec;121(6):1591-1597. doi: 10.1007/s13760-020-01430-2. Epub 2020 Jul 10.

Abstract

Edaravone is a free radical scavenger that has been recently approved for treatment of Amyotrophic lateral sclerosis (ALS) to delay functional decline. We aim to evaluate edaravone efficacy and safety in ALS patients in the main neurology tertiary center in Kuwait over 72-week period. We conducted a prospective observational cohort study in the main tertiary hospital over 72-week period from July 2018 until January 2020. Patients were assessed at baseline, 24, 48 and 72 weeks of therapy using ALSFRS-R score, MRC sum score, FVC value, among other parameters. Seventeen consecutive patients were evaluated. All patients were assessed at baseline, 24 and 48 weeks, while 9 patients (52.9%) were further assessed at 72 weeks. There was a statistically significant decline of ALSFRS-R at 72 weeks, MRC sum score at 48 and 72 weeks, while the decline in FVC was not statistically significant. Glycosuria was found in only one patient. Our study showed significant functional decline after 1 year of edaravone therapy with preserved respiratory function. The drug had a high level of dissatisfaction among our cohort despite having a high safety profile.

摘要

依达拉奉是一种自由基清除剂,最近被批准用于治疗肌萎缩侧索硬化症(ALS)以延缓功能下降。我们旨在评估依达拉奉在科威特主要神经病学三级中心的 ALS 患者中的疗效和安全性,研究时间为 72 周。我们在 2018 年 7 月至 2020 年 1 月的 72 周期间在主要的三级医院进行了前瞻性观察队列研究。患者在基线、24、48 和 72 周治疗时使用 ALSFRS-R 评分、MRC 总和评分、FVC 值等参数进行评估。共评估了 17 名连续患者。所有患者均在基线、24 和 48 周进行了评估,而 9 名患者(52.9%)在 72 周时进一步进行了评估。72 周时 ALSFRS-R 评分、48 周和 72 周时 MRC 总和评分均有统计学意义下降,而 FVC 下降无统计学意义。仅 1 名患者出现尿糖。我们的研究表明,在依达拉奉治疗 1 年后,患者的功能显著下降,但呼吸功能得以保持。尽管安全性高,但该药物在我们的队列中引起了高度不满。

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