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:
No author received payment from a third party for any aspect of the submitted work. Dr. Andrews has served as a consultant for Avexis, Inc., AL-S Pharma, and Cytokinetics, Inc.; and has received research grants from Neuraltus, Roche, Orion, Biogen, and Novartis. Dr. Jackson reports receiving personal fees from BrainStorm Cell Therapeutics, Inc., Mallinckrodt Pharmaceuticals, Anelixis Therapeutics, and Alexion Pharmaceuticals; consulting and Speakers Bureau fees from ITF Pharma (outside the submitted work), Avanir Pharmaceuticals, Inc., Strongbridge Biopharma, CSL Behring, Mitsubishi Tanabe Pharma America, Cytokinetics, Inc., Orion Corporation, and Alexion Pharmaceuticals; and research grants from Mitsubishi Tanabe Pharma America, the Muscular Dystrophy Association, Cytokinetics, Inc., and the Amyotrophic Lateral Sclerosis Association. Dr. Heiman-Patterson reports receiving personal fees as an advisory board/consultant from Mitsubishi Tanabe Pharma America, Cytokinetics, Inc., ITF Pharma (outside the submitted work), and Biohaven Pharmaceuticals and as advisory board for Alnylam Pharmaceuticals Inc. Paolo Bettica, MD, PhD is a full-time employee of Italfarmaco S.p.A., and as such, reports personal fees from Italfarmaco S.p.A. during the conduct of the study. Dr. Brooks reports receiving research support from the Muscular Dystrophy Association, RTI International, MediciNova, Mitsubishi Tanabe Pharma America, Biohaven Pharmaceuticals, Orion, Neuraltus Pharmaceuticals, Cytokinetics, Inc., Santhera Pharmaceuticals, Biogen, ITF Pharma (outside the submitted work), Acceleron Pharma, Philips Respironics, and the Centers for Disease Control and Prevention (CDC). Dr. Pioro reports receiving personal fees from Avanir Pharmaceuticals, Inc., Biohaven Pharmaceuticals. Inc., Cytokinetics, Inc., ITF Pharma (outside the submitted work), Mitsubishi Tanabe Pharma America, and Otsuka America, Inc., and research grants from Centers for Disease Control and Prevention/National Institutes of Health (CDC/NIH) and the Amyotrophic Lateral Sclerosis Association. The authors have indicated that they have no other conflicts of interest regarding the content of this article.
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
VL - 21
SP - 509
EP - 518
JO - Amyotrophic Lateral Sclerosis and Other Motor Neuron Disorders
JF - Amyotrophic Lateral Sclerosis and Other Motor Neuron Disorders
SN - 2167-8421
IS - 7-8
ER -