TY - JOUR
T1 - Real-world evidence of riluzole effectiveness in treating amyotrophic lateral sclerosis
AU - Andrews, Jinsy A.
AU - Jackson, Carlayne E.
AU - Heiman-Patterson, Terry D.
AU - Bettica, Paolo
AU - Brooks, Benjamin Rix
AU - Pioro, Erik P.
N1 - Funding Information:
Medical writing assistance (funded by ITF Pharma, a subsidiary of Italfarmaco S.p.A.) was provided by ECIR Medical Communications (Benjamin J Epstein, PharmD; President). ECIR developed the first draft based on an author-approved outline and assisted in implementing author revisions. Editorial assistance in formatting, proofreading, copyediting, fact-checking, and publication submission was also provided by ECIR. All authors contributed equally to the article and approved the final version for submission.
Publisher Copyright:
© 2020 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.
PY - 2020
Y1 - 2020
N2 - 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.
AB - 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.
KW - Riluzole
KW - amyotrophic lateral sclerosis
KW - neurodegenerative
KW - real-world
KW - survival
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U2 - 10.1080/21678421.2020.1771734
DO - 10.1080/21678421.2020.1771734
M3 - Review article
C2 - 32573277
AN - SCOPUS:85087408790
SN - 2167-8421
VL - 21
SP - 509
EP - 518
JO - Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
JF - Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
IS - 7-8
ER -