Journal
AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION
Volume 18, Issue -, Pages 11-19Publisher
TAYLOR & FRANCIS LTD
DOI: 10.1080/21678421.2017.1363780
Keywords
Amyotrophie lateral sclerosis (ALS); edaravone; subgroup analysis; revised ALS functional rating scale (ALSFRS-R); MCI-186
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Funding
- Mitsubishi Tanabe Pharma Corporation
- Intramural Research Grant for Neurological Psychiatric Disorders from the National Center of Neurology and Psychiatry (NCNP)
- Mitsubishi Tanabe Pharma America, Inc.
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Our first phase III study failed to demonstrate efficacy of edaravone for amyotrophic lateral sclerosis (ALS) compared to placebo. Here, we performed post-hoc subgroup analysis to identify a subgroup in which edaravone might be expected to show efficacy. We focussed on two newly defined subgroups, EESP and dpEESP2y. The EESP was defined as the efficacy expected subpopulation with % forced vital capacity of >= 80%, and >= 2 points for all item scores in the revised ALS functional rating scale (ALSFRS-R) score before treatment. The dpEESP2y was defined as the greater-efficacy-expected subpopulation within EESP having a diagnosis of 'definite' or 'probable' ALS according to the El Escorial revised Airlie House diagnostic criteria and onset of disease within two years. The primary endpoint of the post-hoc analysis was the change in the ALSFRS-R score during the 24-week treatment period. The intergroup differences of the least-squares mean change in the ALSFRS-R score +/- standard error during treatment were 0.65+ 0.78 (p = 0.4108) in the full analysis set, 2.20 1.03 (p= 0.0360) in the EESP, and 3.01 +/- 1.33 (p 0.0270) in the dpEESP2y. Edaravone exhibited efficacy in the dpEESP2y subgroup. A further clinical study in patients meeting dpEESP2y criteria is warranted.
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