Real-world safety and tolerability of intravenous edaravone in patients with amyotrophic lateral sclerosis

This retrospective cohort study describes real-world safety and tolerability outcomes in United States-based edaravone-treated patients with ALS. Amerita Specialty Infusion Services provided IV edaravone to patients with ALS treated with their first dose between 25 September 2017-30 September 2022. Mean ALS Functional Rating Scale-Revised (ALSFRS-R) scores and forced vital capacity (FVC) %–predicted measures were recorded within ± 100 days from care initiation to approximate baseline values. Included patients (<i>n</i> = 243) received/were still receiving IV edaravone/edaravone oral suspension as of 30 September 2022. At initiation, 66.7% were male, mean age ± SD was 61.2 ± 11.2 years, and 61.3% were covered by government insurance. In patients with provider–recorded ALSFRS-R (<i>n</i> = 115) and FVC (<i>n</i> = 84) %–predicted measures within ± 100 days from care initiation, mean ± SD values were 35.1 ± 8.9 and 72.3% ± 21.7%, respectively. Mean ± SD therapy duration was 13.5 ± 11.4 months. Discontinuation reasons included death/hospice (<i>n</i> = 82), patient’s choice (<i>n</i> = 38), doctor’s choice (<i>n</i> = 31), insurance (<i>n</i> = 18), and other (<i>n</i> = 12). Reasons for IV edaravone discontinuation and IV edaravone administration access device were not associated. Treatment discontinuation was primarily related to ALS disease progression/death, rather than safety or tolerability. This study representative of real-world patients with ALS suggests that edaravone showed consistent safety and tolerability profiles with previous studies.

本项回顾性队列研究旨在阐明美国接受依达拉奉 (edaravone) 治疗的肌萎缩侧索硬化 (ALS, Amyotrophic Lateral Sclerosis) 患者的真实世界安全性与耐受性结局。Amerita专科输液服务公司 (Amerita Specialty Infusion Services) 为2017年9月25日至2022年9月30日期间首次接受静脉 (IV, Intravenous) 依达拉奉治疗的ALS患者提供了静脉给药服务。研究人员在患者治疗启动前后±100天内记录其平均肌萎缩侧索硬化功能评定量表修订版 (ALSFRS-R, Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) 评分及用力肺活量 (FVC, Forced Vital Capacity) 预计值百分比，以近似获取基线临床数据。截至2022年9月30日，纳入分析的243例患者均已接受或仍在接受静脉依达拉奉/依达拉奉口服混悬液治疗。治疗启动时，入组患者中66.7%为男性，平均年龄±标准差 (SD, Standard Deviation) 为61.2±11.2岁，61.3%的患者享有政府医保覆盖。在治疗启动前后±100天内拥有医护人员记录的ALSFRS-R评分 (n=115) 及FVC预计值百分比 (n=84) 数据的患者亚组中，其平均±标准差评分与指标分别为35.1±8.9和72.3%±21.7%。患者的平均治疗持续时间±标准差为13.5±11.4个月。停药原因包括死亡/临终关怀 (hospice, n=82)、患者自主选择 (n=38)、医师决策 (n=31)、医保报销问题 (n=18) 及其他原因 (n=12)。静脉依达拉奉的停药原因与给药通路装置并无关联。研究结果显示，治疗停药主要与ALS疾病进展/死亡相关，而非安全性或耐受性问题。本研究作为真实世界ALS患者队列的代表性研究，提示依达拉奉的安全性与耐受性特征与既往同类研究结果一致。