For a study, researchers sought to determine whether or not PB/TURSO lengthened tracheostomy/ventilation-free survival and lowered the number of patients with ALS. The latter required their first hospitalization during the CENTAUR trial. Adults with El Escorial Definite ALS who were less than or equal to 18 months removed from the onset of their symptoms were randomly assigned to receive either PB/TURSO or a placebo for 6 months. Those who finished the randomized therapy were eligible to enroll in the study’s open-label extension (OLE) phase and continue receiving PB/TURSO for less than or equal to 30 months. Throughout the trial, which began in January 2015 and will end in July 2020 (the longest postrandomization follow-up was 35 months), the following individual or combined key events’ times to occur were compared in the treatment groups that were originally randomized: death, tracheostomy, permanent assisted ventilation (PAV), and first hospitalization. Those who were initially assigned to receive PB/TURSO (n=87) had a risk of any critical event that was 47% lower than those who were initially randomized to receive a placebo (n=48, of whom 71% received delayed-start PB/TURSO in the OLE phase) (HR=0.53; 95% CI 0.35 to 0.81; P=0.003). Those who were initially assigned to PB/TURSO had lower risks of death, tracheostomy/PAV (HR=0.51; 95% CI 0.32 to 0.84; P=0.007), and first hospitalization (HR=0.56; 95% CI 0.34 to 0.95; P=0.03). Early PB/TURSO treatment prolonged tracheostomy/PAV-free survival and delayed the onset of their first hospitalization in patients with ALS.
Source – jnnp.bmj.com/content/93/8/871
