Correspondence: Christopher Damlos (christopher.damlos@va.gov)
aVeteran Health Indiana, Indianapolis
bIndiana University School of Medicine, Indianapolis
Author disclosures
The authors report no actual or potential conflicts of interest or outside sources of funding with regard to this article.
Disclaimer
The opinions expressed herein are those of the authors and do not necessarily reflect those of Federal Practitioner, Frontline Medical Communications Inc., the US Government, or any of its agencies. This article may discuss unlabeled or investigational use of certain drugs. Please review the complete prescribing information for specific drugs or drug combinations—including indications, contraindications, warnings, and adverse effects—before administering pharmacologic therapy to patients.
Ethics and consent
This study was reviewed by VA Research and Indiana University/Purdue University-Indianapolis Institutional Review Board (IRB) and determined to be IRB exempt.
Comparisons between the edaravone and control groups for differences in patient characteristics were made using χ2 and 2-sample t tests for categorical and continuous variables, respectively. Comparisons between the 2 groups for differences in study outcomes (ALSFRS-R scores, %FVC, SIS) at each time point were evaluated using 2-sample t tests. Adverse events and adverse drug reactions were compared between groups using χ2 tests. Statistical significance was set at 0.05.
We estimated that a sample size of 21 subjects in the edaravone (case) group and 42 in the standard-of-care (control) group would be needed to achieve 80% power to detect a difference of 6.5 between the 2 groups for the change in ALSFRS-R scores. This 80% power was calculated based on a 2-sample t test, and assuming a 2-sided 5% significance level and a within-group SD of 8.5.9 Statistical analysis was conducted using Microsoft Excel.
Results
A total of 96 unique patients were seen in our multidisciplinary ALS clinic between September 1, 2014, and August 31, 2017 (Figure).
Of the 96 patients, 10 met exclusion criteria. From the remaining 86, 42 were randomly selected for the standard-of-care group. A total of 27 patients seen in multidisciplinary ALS clinic between September 1, 2017, and August 31, 2020, received at least 1 dose of IV edaravone. Of the 27 edaravone patients, 6 were excluded for not completing a total of 6 months of edaravone. Two of the 6 excluded developed a rash, which resolved within 1 week after discontinuing edaravone. The other 4 discontinued edaravone before 6 months because of disease progression.
Baseline Characteristics
Baseline demographics were similar between the groups (Table 1). Most patients were White men with a mean age of 60 years. Baseline %FVC was about 68%. Fewer patients in the standard-of-care group were taking riluzole than in the edaravone group (67% vs 95%, respectively; P = .002). Mean (SD) baseline SIS scores were slightly higher in the standard-of-care group vs the edaravone group (2.0 [1.0] vs 1.4 [0.6], respectively; P = .01).
Efficacy
No difference was found in the ALSFRS-R scores at 6 months between the IV edaravone and standard-of-care groups (P = .84) (Table 2). Our study did not meet power to calculate statistical analysis at 12, 18, and 24 months due to its size. No difference was found in change from baseline %FVC at 6 months between the 2 groups (P = .30) (Table 3). Change between the 2 groups in baseline SIS at 6 months also was not different (P = .69) (Table 4). Sample size was insufficient to calculate %FVC and SIS at the 12, 18, and 24 month intervals.
No difference was noted in time to death between groups (P = .93) (Table 5), and no adverse events were reported in either group.
