{
  "abstract": "Objectives Edaravone is a free radical scavenger that mitigates oxidative stress, a process that is critically linked to disease progression in amyotrophic lateral sclerosis (ALS). Clinical assessment of progression typically relies on the ALS-Functional-Rating-Scale-Revised (ALSFRS-R), with a reduced rate of ALSFRS-R score decline associated with slower disease progression. The present meta-analysis was undertaken to investigate the benefit of edaravone across patient cohorts, with effectiveness determined by ALSFRS-R in the recommended Pharmaceutical Benefits Scheme (PBS) population.Methods This meta-analysis included data from two pivotal trials, Study16 ( NCT00330681) and Study19 (NCT01492686), where patients with ALS were randomised to receive either intravenous edaravone or placebo. In both studies, eligible patients were within 2 years of disease onset, independent in daily activities of living, and had 80% of forced vital capacity (FVC). The primary endpoint was reduction in decline of the ALSFRS-R score in patients on edaravone, compared to the placebo group over 6 treatment cycles. Safety outcomes were also analysed.Result In total, 209 patients were included in the meta-analysis (n=109 edaravone, n=100 placebo). On the 48-point scale, reduction in the rate of decline was 2.98 (95% CI 1.67, 4.29) in favour of edaravone, a clinically important 40% reduction versus placebo. Safety analyses for patients with one or more adverse events or serious adverse events showed no significant differences between the edaravone and placebo groups.Conclusions Meta-analysis results demonstrate the safety and efficacy of edaravone in slowing disease progression in ALS patients, using a clinical population consistent with the Australian PBS population.",
  "authors": [
    {
      "affiliations": [
        "KMC Healthcare, Melbourne, VIC, Australia"
      ],
      "name": "Carolyn Rutherford"
    },
    {
      "affiliations": [
        "Teva Pharmaceuticals, Macquarie Park, NSW, Australia"
      ],
      "name": "Bernd Merkel"
    },
    {
      "affiliations": [
        "KMC Healthcare, Melbourne, VIC, Australia"
      ],
      "name": "Kelly Makarounas-Kirchmann"
    },
    {
      "affiliations": [
        "KMC Healthcare, Melbourne, VIC, Australia"
      ],
      "name": "Matt Kirchmann"
    },
    {
      "affiliations": [
        "NEURA, Randwick, NSW, Australia"
      ],
      "name": "Matthew C Kiernan"
    }
  ],
  "title": "3417 Clinical outcomes of edaravone treatment: a meta-analysis",
  "uid": "252fafc9-3639-541b-b2a8-d64344a8ff85"
}