Real-world evidence of riluzole effectiveness in treating amyotrophic lateral sclerosis

Jinsy A. Andrews*, Carlayne E. Jackson, Terry D. Heiman-Patterson, Paolo Bettica, Benjamin Rix Brooks, Erik P. Pioro

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

53 Scopus citations


Objective: To compare the effect of riluzole on median survival in population studies of patients with amyotrophic lateral sclerosis (ALS) with that observed in clinical trials. Methods: Two independent PubMed searches were conducted, to identify population studies that reported median survival for ALS patients who were either treated with riluzole or remained riluzole-free. Results: We identified 14 studies that met the inclusion criteria of reporting median survival and an additional study that reported mean survival of both riluzole and riluzole-free patients. Analysis of the 15 studies found that a majority reported increased survival of riluzole vs. riluzole-free patients. In 8 studies, the median survival for patients treated with riluzole was 6–19 months longer compared with patients not treated with riluzole (p < 0.05). Three additional studies reported a clinically meaningful treatment effect (range 3–5.9 months) but did not meet statistical significance. The remaining 4 studies did not show a meaningful treatment effect between riluzole and riluzole-free groups (<3 months), and differences among the groups were not significant. Also, 5 of the studies used multivariate regression analysis to investigate the level of association between treatment with riluzole and survival; these analyses supported the positive effect of riluzole on survival. Conclusions: A majority of population studies that compared riluzole vs. riluzole-free ALS patients found significant differences in median survival between the two groups, ranging from 6 to 19 months. This is substantially longer than the 2- to 3-month survival benefit observed in the pivotal clinical trials of riluzole.

Original languageEnglish (US)
Pages (from-to)509-518
Number of pages10
JournalAmyotrophic Lateral Sclerosis and Frontotemporal Degeneration
Issue number7-8
StatePublished - 2020


  • Riluzole
  • amyotrophic lateral sclerosis
  • neurodegenerative
  • real-world
  • survival

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology


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