Long‐term survival of participants in the CENTAUR trial of sodium phenylbutyrate‐taurursodiol in amyotrophic lateral sclerosis
| Autor: | Meghan Hall, Janet P. Wittes, Gary L. Pattee, Eric A. Macklin, Eric Tustison, Tuan Vu, Zi-Fan Yu, Samuel P. Dickson, Rudolph E. Tanzi, Liberty Jenkins, Suma Babu, Michael A. Elliott, Jonathan S. Katz, Chafic Karam, Patricia L. Andres, Terry Heiman-Patterson, Stephen N. Scelsa, Christina Fournier, Joseph Ostrow, Timothy M. Miller, Joshua N Cohen, Daragh Heitzman, Edward J. Kasarskis, Derek Dagostino, Patrick D. Yeramian, Colin Quinn, Rebecca Randall, Lindsay Pothier, James Wymer, Hong Yu, Gale Kittle, Newman Knowlton, Michelle McGovern, Shafeeq Ladha, Jason Walker, Jeffrey D. Rothstein, Adam Quick, James Chan, Kent L. Leslie, Prasha Vigneswaran, Maria E. St. Pierre, Kristin M. Johnson, Walter Gilbert, Namita Goyal, James B. Caress, Marianne Chase, Sabrina Paganoni, Jonathan D. Glass, Justin Klee, Merit Cudkowicz, Jeremy M. Shefner, Suzanne Hendrix, Carlayne E. Jackson, Margaret Owegi, James D. Berry, David A. Schoenfeld, Alexander Sherman, Stephen A. Goutman, Matthew Eydinov, Andrea Swenson, Samuel Maiser |
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| Rok vydání: | 2020 |
| Předmět: |
0301 basic medicine
amyotrophic lateral sclerosis medicine.medical_specialty Physiology CENTAUR 030105 genetics & heredity Placebo survival analysis 03 medical and health sciences Cellular and Molecular Neuroscience 0302 clinical medicine Physiology (medical) Internal medicine Long term survival Medicine Amyotrophic lateral sclerosis Clinical Research Articles Survival analysis sodium phenylbutyrate‐taurursodiol Clinical Research Article business.industry Hazard ratio Sodium phenylbutyrate medicine.disease Confidence interval motor neuron disease Neurology (clinical) business 030217 neurology & neurosurgery Median survival medicine.drug |
| Zdroj: | Muscle & Nerve |
| ISSN: | 1097-4598 0148-639X |
| Popis: | An orally administered, fixed‐dose coformulation of sodium phenylbutyrate‐taurursodiol (PB‐TURSO) significantly slowed functional decline in a randomized, placebo‐controlled, phase 2 trial in ALS (CENTAUR). Herein we report results of a long‐term survival analysis of participants in CENTAUR. In CENTAUR, adults with ALS were randomized 2:1 to PB‐TURSO or placebo. Participants completing the 6‐month (24‐week) randomized phase were eligible to receive PB‐TURSO in the open‐label extension. An all‐cause mortality analysis (35‐month maximum follow‐up post‐randomization) incorporated all randomized participants. Participants and site investigators were blinded to treatment assignments through the duration of follow‐up of this analysis. Vital status was obtained for 135 of 137 participants originally randomized in CENTAUR. Median overall survival was 25.0 months among participants originally randomized to PB‐TURSO and 18.5 months among those originally randomized to placebo (hazard ratio, 0.56; 95% confidence interval, 0.34‐0.92; P = .023). Initiation of PB‐TURSO treatment at baseline resulted in a 6.5‐month longer median survival as compared with placebo. Combined with results from CENTAUR, these results suggest that PB‐TURSO has both functional and survival benefits in ALS. |
| Databáze: | OpenAIRE |
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