Loading...
Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial
Paganonin, Sabrina Hendrix, Suzanne Dickson, Samuel P. Knowlton, Newman Berry, James D. Elliott, Michael A. Maiser, Samuel Karam, Chafic Caress, James B. Owegi, Margaret Ayo
Paganonin, Sabrina
Hendrix, Suzanne
Dickson, Samuel P.
Knowlton, Newman
Berry, James D.
Elliott, Michael A.
Maiser, Samuel
Karam, Chafic
Caress, James B.
Owegi, Margaret Ayo
Citations
Altmetric:
Genre
Journal article
Date
2022-05-16
Advisor
Committee member
Group
Department
Neurology
Subject
Permanent link to this record
Collections
Research Projects
Organizational Units
Journal Issue
DOI
http://dx.doi.org/10.1136/jnnp-2022-329024
Abstract
Background: Coformulated sodium phenylbutyrate/taurursodiol (PB/TURSO) was shown to prolong survival and slow functional decline in amyotrophic lateral sclerosis (ALS). Objective Determine whether PB/TURSO prolonged tracheostomy/ventilation-free survival and/or reduced first hospitalisation in participants with ALS in the CENTAUR trial. Methods: Adults with El Escorial Definite ALS ≤18 months from symptom onset were randomised to PB/ TURSO or placebo for 6 months. Those completing randomised treatment could enrol in an open-label extension (OLE) phase and receive PB/TURSO for ≤30 months. Times to the following individual or combined key events were compared in the originally randomised treatment groups over a period spanning trial start through July 2020 (longest postrandomisation follow-up, 35 months): death, tracheostomy, permanent assisted ventilation (PAV) and first hospitalisation. Results: Risk of any key event was 47% lower in those originally randomised to PB/TURSO (n=87) versus placebo (n=48, 71% of whom received delayed-start PB/TURSO in the OLE phase) (HR=0.53; 95% CI 0.35 to 0.81; p=0.003). Risks of death or tracheostomy/PAV (HR=0.51; 95% CI 0.32 to 0.84; p=0.007) and first hospitalisation (HR=0.56; 95% CI 0.34 to 0.95; p=0.03) were also decreased in those originally randomised to PB/TURSO. Conclusions: Early PB/TURSO prolonged tracheostomy/PAV-free survival and delayed first hospitalisation in ALS. Trial registration number: NCT03127514; NCT03488524.
Description
Citation
Paganoni S, Hendrix S, Dickson SP, et alEffect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trialJournal of Neurology, Neurosurgery & Psychiatry 2022;93:871-875.
Citation to related work
BMJ Publishing Group
Has part
Journal of Neurology, Neurosurgery and Psychiatry (JNNP), Vol. 93, Iss. 8
ADA compliance
For Americans with Disabilities Act (ADA) accommodation, including help with reading this content, please contact scholarshare@temple.edu