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P.046 Real-world survival effectiveness of edaravone in amyotrophic lateral sclerosis: a propensity score weighted, registry-based, Canada-wide cohort study

Published online by Cambridge University Press:  05 June 2023

A Abrahao
Affiliation:
(Toronto)
MV Vyas
Affiliation:
(Toronto)
A Parks
Affiliation:
(Toronto)
V Hodgkinson
Affiliation:
(Calgary)*
A Dyck
Affiliation:
(Calgary)
T Benstead
Affiliation:
(Halifax)
H Briemberg
Affiliation:
(Vancouver)
A Genge
Affiliation:
(Montreal)
I Grant
Affiliation:
(Halifax)
G Jewett
Affiliation:
(Calgary)
W Johnston
Affiliation:
(Edmonton)
S Kalra
Affiliation:
(Edmonton)
A Marrero
Affiliation:
(Moncton)
R Massie
Affiliation:
(Montreal)
M Melanson
Affiliation:
(Kingston)
C O’Connell
Affiliation:
(Fredericton)
G Pfeffer
Affiliation:
(Calgary)
KL Schellenberg
Affiliation:
(Saskatoon)
S Taylor
Affiliation:
(Halifax)
C Shoesmith
Affiliation:
(London)
G Matte
Affiliation:
(Montreal)
L Zinman
Affiliation:
(Toronto)
L Korngut
Affiliation:
(Calgary)
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Abstract

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Background: ALS is a progressive neurodegenerative disease without a cure and limited treatment options. Edaravone, a free radical scavenger, was shown to slow disease progression in a select group of patients with ALS over 6 months; however, the effect on survival was not investigated in randomized trials. The objective of this study is to describe real-world survival effectiveness over a longer timeframe. Methods: This retrospective cohort study included patients with ALS across Canada with symptom onset up to three years. Those with a minimum 6-month edaravone exposure between 2017 and 2022 were enrolled in the interventional arm, and those without formed the control arm. The primary outcome of tracheostomy-free survival was compared between the two groups, accounting for age, sex, ALS-disease progression rate, disease duration, pulmonary vital capacity, bulbar ALS-onset, and presence of frontotemporal dementia or C9ORF72 mutation using inverse propensity treatment weights. Results: 182 patients with mean ± SD age 60±11 years were enrolled in the edaravone arm and 860 in the control arm (mean ± SD age 63±12 years). Mean ± SD time from onset to edaravone initiation was 18±10 months. Tracheostomy-free survival will be calculated. Conclusions: This study will provide evidence for edaravone effectiveness on tracheostomy-free survival in patients with ALS.

Type
Abstracts
Copyright
© The Author(s), 2023. Published by Cambridge University Press on behalf of Canadian Neurological Sciences Federation