Berry James D, Hagan Melissa, Zhang Jeffrey, Liu Ying, Ciepielewska Malgorzata
Healey Center for ALS, Massachusetts General Hospital, Neurology, Boston, MA, 02114 USA.
Mitsubishi Tanabe Pharma America, Inc., Health Economics and Outcomes Research (HEOR), Medical Affairs, Jersey City, NJ, 07310 USA.
J Comp Eff Res. 2025 Feb;14(2):e240007. doi: 10.57264/cer-2024-0007. Epub 2025 Jan 21.
To estimate time-to-progression milestones in people with amyotrophic lateral sclerosis (PALS) treated versus not treated with intravenous (IV) edaravone (Radicava IV, Mitsubishi Tanabe Pharma America [MTPA], hereafter "IV edaravone") in a real-world setting. IV edaravone is US FDA approved for the treatment of ALS and was shown in clinical trials to slow the rate of physical functional decline. This retrospective observational analysis included PALS continuously enrolled in Optum's Clinformatics Data Mart between 8 August 2017 and 31 December 2021. Cases treated with IV edaravone and controls not treated with IV edaravone were propensity score matched for: age, sex, race, US region of residence, pre-index disease duration, insurance, riluzole prescription; and pre-index claims for cardiovascular disease, artificial nutrition/gastrostomy tube, noninvasive ventilation and all-cause hospitalization. The index date was the first IV edaravone claim for cases; for controls, the index date was randomly assigned after IV edaravone market availability. Restricted mean time lost was calculated for the following disease progression milestones: new use of canes/walkers/wheelchairs, artificial nutrition, noninvasive ventilation, invasive ventilation, speech-generating devices and hospice. Cases (n = 395) were matched to controls (n = 395). Cases had less restricted mean time lost, indicating longer disease progression milestone-free time, for all disease progression milestones. From 0 to 24 months post index, more cases (n = 129) than controls (n = 103) reported no milestones and more controls (n = 232) than cases (n = 131) reported deaths. In a US-based real-world setting, IV edaravone-treated PALS had a longer time to disease progression milestone events and fewer deaths in 2 years compared with PALS not treated with IV edaravone.
在真实世界环境中，评估肌萎缩侧索硬化症（PALS）患者接受与未接受静脉注射依达拉奉（Radicava IV，三菱田边制药美国公司[MTPA]，以下简称“静脉注射依达拉奉”）治疗后的疾病进展至特定节点的时间。静脉注射依达拉奉已获美国食品药品监督管理局（FDA）批准用于治疗肌萎缩侧索硬化症，并且在临床试验中显示可减缓身体功能衰退的速度。这项回顾性观察性分析纳入了2017年8月8日至2021年12月31日期间持续登记在Optum临床信息数据集市中的PALS患者。接受静脉注射依达拉奉治疗的病例与未接受静脉注射依达拉奉治疗的对照在以下方面进行倾向得分匹配：年龄、性别、种族、美国居住地区、索引前疾病持续时间、保险、利鲁唑处方；以及索引前心血管疾病、人工营养/胃造瘘管、无创通气和全因住院的索赔情况。索引日期对于病例是首次静脉注射依达拉奉索赔日期；对于对照，索引日期在静脉注射依达拉奉上市后随机分配。计算了以下疾病进展节点的受限平均失能时间：新使用手杖/助行器/轮椅、人工营养、无创通气、有创通气、语音生成设备和临终关怀。病例（n = 395）与对照（n = 395）进行了匹配。对于所有疾病进展节点，病例的受限平均失能时间更少，表明无疾病进展节点的时间更长。在索引后0至24个月，报告无节点的病例（n = 129）多于对照（n = 103），报告死亡的对照（n = 232）多于病例（n = 131）。在基于美国的真实世界环境中，与未接受静脉注射依达拉奉治疗的PALS患者相比，接受静脉注射依达拉奉治疗的PALS患者疾病进展至节点事件的时间更长，且在2年内死亡人数更少。
