Evidence for a multimodal effect of riluzole in patients with ALS?
2019; BMJ; Volume: 90; Issue: 10 Linguagem: Inglês
10.1136/jnnp-2018-320211
ISSN1468-330X
AutoresAdriaan D. de Jongh, Ruben P. A. van Eijk, Leonard H. van den Berg,
Tópico(s)biodegradable polymer synthesis and properties
ResumoAmyotrophic 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. ### 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. …
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