Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Paganoni Sabrina, Hendrix Suzanne, Dickson Samuel P, Knowlton Newman, Berry James D, Elliott Michael A, Maiser Samuel, Karam Chafic, Caress James B, Owegi Margaret Ayo, Quick Adam, Wymer James, Goutman Stephen A, Heitzman Daragh, Heiman-Patterson Terry D, Jackson Carlayne, Quinn Colin, Rothstein Jeffrey D, Kasarskis Edward J, Katz Jonathan, Jenkins Liberty, Ladha Shafeeq S, Miller Timothy M, Scelsa Stephen N, Vu Tuan H, Fournier Christina, Johnson Kristin M, Swenson Andrea, Goyal Namita, Pattee Gary L, Babu Suma, Chase Marianne, Dagostino Derek, Hall Meghan, Kittle Gale, Eydinov Mathew, Ostrow Joseph, Pothier Lindsay, Randall Rebecca, Shefner Jeremy M, Sherman Alexander V, Tustison Eric, Vigneswaran Prasha, Yu Hong, Cohen Joshua, Klee Justin, Tanzi Rudolph, Gilbert Walter, Yeramian Patrick, Cudkowicz Merit
Аннотация
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.