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Introduction
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease with a median survival of 3 years. Currently, riluzole is the only drug that can prolong the life expectancy of patients with ALS.1 In the original trial, riluzole prolonged median survival by approximately 3 months, although no effect on function was observed.1 Therefore, it is unknown whether the survival benefit of riluzole occurs at an early stage, late stage or is spread throughout the course of the disease.
Interestingly, in a recent reanalysis of the riluzole trial only a late-stage effect of riluzole was found.2 The investigators noted, however, that the earliest stage could not be analysed because of inclusion criteria and that further studies are needed.2
The question, therefore, remains whether riluzole also prolongs the earliest disease stage. This information is vital as, from a patient’s perspective, prolonging the earliest disease stages, when disability is lowest and quality of life is highest, is most desirable. We aim, therefore, to determine the timing of benefit of riluzole in a large cohort of patients with ALS.
Methods
Data
We used the Pooled Resource Open-Access ALS Clinical Trial database which contains data from 23 phase II/III clinical trials, including the riluzole trial.1
Staging systems
Two staging algorithms in ALS, Milano-Torino (MITOS) and King’s staging, were used to estimate clinical stage from clinical trial data (ALS Functional Rating Scale-Revised [ALSFRS-R] scores, weight and vital capacity measurements).2 3 There are five MITOS stages, based on autonomy loss in four key domains of the ALSFRS-R: walking/self-care, swallowing, communicating and breathing. …
Footnotes
Contributors AdJ, RPAvE and LHvdB designed the study. AdJ analysed the data and drafted the manuscript. RPAvE and LHvdB critically reviewed the manuscript.
Funding This study was supported by the Netherlands ALS Foundation.
Competing interests AdJ and RPAvE report no disclosures. LHvdB reports grants from Netherlands ALS Foundation, the Netherlands Organization for Health Research and Development (Vici scheme), the Netherlands Organization for Health Research and Development (SOPHIA, STRENGTH, ALS-CarE project), funded through the EU Joint Programme – Neurodegenerative Disease Research, JPND), served on the Scientific Advisory Board of Biogen, Cytokinetics, Prinses Beatrix SpierFonds, and the Latran Foundation.
Patient consent for publication Not required.
Ethics approval The Pooled Resource Open-Access ALS Clinical Trial database is institutional review board approved and only uses anonymised data. Patients consented to participate during the individual trials.
Provenance and peer review Not commissioned; externally peer reviewed.