PT  - JOURNAL ARTICLE
AU  - Sabrina Paganoni
AU  - Suzanne Hendrix
AU  - Samuel P Dickson
AU  - Newman Knowlton
AU  - James D Berry
AU  - Michael A Elliott
AU  - Samuel Maiser
AU  - Chafic Karam
AU  - James B Caress
AU  - Margaret Ayo Owegi
AU  - Adam Quick
AU  - James Wymer
AU  - Stephen A Goutman
AU  - Daragh Heitzman
AU  - Terry D Heiman-Patterson
AU  - Carlayne Jackson
AU  - Colin Quinn
AU  - Jeffrey D Rothstein
AU  - Edward J Kasarskis
AU  - Jonathan Katz
AU  - Liberty Jenkins
AU  - Shafeeq S Ladha
AU  - Timothy M Miller
AU  - Stephen N Scelsa
AU  - Tuan H Vu
AU  - Christina Fournier
AU  - Kristin M Johnson
AU  - Andrea Swenson
AU  - Namita Goyal
AU  - Gary L Pattee
AU  - Suma Babu
AU  - Marianne Chase
AU  - Derek Dagostino
AU  - Meghan Hall
AU  - Gale Kittle
AU  - Mathew Eydinov
AU  - Joseph Ostrow
AU  - Lindsay Pothier
AU  - Rebecca Randall
AU  - Jeremy M Shefner
AU  - Alexander V Sherman
AU  - Eric Tustison
AU  - Prasha Vigneswaran
AU  - Hong Yu
AU  - Joshua Cohen
AU  - Justin Klee
AU  - Rudolph Tanzi
AU  - Walter Gilbert
AU  - Patrick Yeramian
AU  - Merit Cudkowicz
TI  - Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial
AID  - 10.1136/jnnp-2022-329024
DP  - 2022 Aug 01
TA  - Journal of Neurology, Neurosurgery & Psychiatry
PG  - 871--875
VI  - 93
IP  - 8
4099  - http://jnnp.bmj.com/content/93/8/871.short
4100  - http://jnnp.bmj.com/content/93/8/871.full
SO  - J Neurol Neurosurg Psychiatry2022 Aug 01; 93
AB  - 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.Deidentified participant data will be made available upon reasonable request. Requests for data sharing can be sent to info@amylyx.com.