Clinical staging in amyotrophic lateral sclerosis: analysis of Edaravone Study 19.
Clinical staging in amyotrophic lateral sclerosis: analysis of Edaravone Study 19.
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肌萎缩性侧索硬化症的临床分期：依达拉奉研究分析
DOI：
10.1136/jnnp-2020-323271
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发表时间：
2021-03
期刊：
影响因子：
--
通讯作者：
Apple S
中科院分区：
文献类型：
--
作者：
Al-Chalabi A;Chiò A;Merrill C;Oster G;Bornheimer R;Agnese W;Apple S
这是对依达拉奉III期研究MCI 186 -19（“研究19”）的事后分析，旨在检查临床分期系统作为肌萎缩侧索硬化症（ALS）临床试验终点的效用。肌萎缩侧索硬化症功能评定量表-研究19的修订项目评分与King分期和Milano-Torino分期（MiToS）进行回顾性映射。我们评估了在研究19期间经历King's和MiToS阶段进展的患者的百分比。我们还根据基线King's分期评估了患者亚组的疾病进展。在双盲治疗期间，依达拉奉组（42. 0%，95% CI 30. 4%-53. 6%）发生King's分期进展的患者百分比低于安慰剂组（55. 9%，95% CI 44. 1%-67. 6%）。在第1阶段的患者中观察到最明显的效果，并在整个开放标签治疗期间保持。MiToS分期≥2期进展的分析显示，在双盲治疗期间，治疗组之间无差异，但在开放标签期间，安慰剂-依达拉奉组患者的进展速度比依达拉奉-依达拉奉组患者更快（对数秩检验，p<0.001）。King's和MiToS分期系统在依达拉奉研究19中评估临床进展方面具有实用性。这些发现可能支持使用分期系统作为ALS临床试验的终点，并了解这些量表测量的获益时间。
This was a post hoc analysis of the Edaravone Phase III Study MCI186-19 (‘Study 19’) to examine the utility of clinical staging systems as end points in clinical trials in amyotrophic lateral sclerosis (ALS). Amyotrophic Lateral Sclerosis Functional Rating Scale—Revised item scores from Study 19 were retrospectively mapped to King’s stage and Milano-Torino staging (MiToS) stage. We assessed the percentage of patients who experienced progression in King’s and MiToS stages during Study 19. We also assessed disease progression in subgroups of patients according to baseline King’s stage. During double-blind treatment, the percentage of patients who experienced a progression in King’s stage was lower for edaravone (42.0%, 95% CI 30.4% to 53.6%) than placebo (55.9%, 95% CI 44.1% to 67.6%). The most pronounced effect was noted among patients who were in stage 1 and was maintained throughout open-label treatment. An analysis of a ≥2-stage progression in MiToS stage showed no difference between treatment arms during double-blind treatment, but during the open-label period, more rapid progression was noted among patients in the placebo–edaravone arm than among those in the edaravone–edaravone arm (log-rank test, p<0.001). The King’s and MiToS staging systems provided utility in assessing clinical progression in Edaravone Study 19. These findings may support the use of staging systems as end points in ALS clinical trials and to understand the timing of benefit as measured by these scales.
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DOI：
10.1093/brain/awr351
发表时间：
2012-03
期刊：
Brain : a journal of neurology
影响因子：
--
作者：
Roche JC;Rojas-Garcia R;Scott KM;Scotton W;Ellis CE;Burman R;Wijesekera L;Turner MR;Leigh PN;Shaw CE;Al-Chalabi A
通讯作者：
Al-Chalabi A
影响因子：
11
作者：
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通讯作者：
Filippini, Graziella
DOI：
10.1007/978-3-030-05542-4_2
发表时间：
2019-01-01
期刊：
REVIEWS ON BIOMARKER STUDIES IN PSYCHIATRIC AND NEURODEGENERATIVE DISORDERS
影响因子：
--
作者：
Cummings, Jeffrey
通讯作者：
Cummings, Jeffrey
影响因子：
2.9
作者：
Sullivan, Paul;Bell, Derek
通讯作者：
Bell, Derek
影响因子：
8.6
作者：
Goetz, CG;Poewe, W;Seidl, L
通讯作者：
Seidl, L