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Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial.
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.
Afiliação
  • Paganoni S; Harvard Medical School, Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, Massachusetts, USA spaganoni@mgh.harvard.edu.
  • Hendrix S; Department of PM & R, Spaulding Rehabilitation Hospital, Charlestown, Massachusetts, USA.
  • Dickson SP; Pentara Corporation, Millcreek, Utah, USA.
  • Knowlton N; Pentara Corporation, Millcreek, Utah, USA.
  • Berry JD; Pentara Corporation, Millcreek, Utah, USA.
  • Elliott MA; Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, Massachusetts, USA.
  • Maiser S; Swedish Neuroscience Institute, Seattle, Washington, USA.
  • Karam C; Department of Neurology, Hennepin Healthcare, Minneapolis, Minnesota, USA.
  • Caress JB; Department of Neurology, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA.
  • Owegi MA; Department of Neurology, Wake Forest University School of Medicine, Winston-Salem, North Carolina, USA.
  • Quick A; Department of Neurology, University of Massachusetts Memorial Medical Center, Worcester, Massachusetts, USA.
  • Wymer J; Department of Neurology, Ohio State University, Columbus, Ohio, USA.
  • Goutman SA; Department of Neurology, College of Medicine, University of Florida, Gainesville, Florida, USA.
  • Heitzman D; Department of Neurology, University of Michigan, Ann Arbor, Michigan, USA.
  • Heiman-Patterson TD; ALS Clinic, Texas Neurology, Dallas, Texas, USA.
  • Jackson C; Department of Neurology, Lewis Katz School of Medicine at Temple University, Philadelphia, Pennsylvania, USA.
  • Quinn C; Department of Neurology, The University of Texas Health Science Center at San Antonio, San Antonio, Texas, USA.
  • Rothstein JD; Department of Neurology, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA.
  • Kasarskis EJ; Department of Neurology, Johns Hopkins University Brain Science Institute, Baltimore, Maryland, USA.
  • Katz J; Department of Neurology, University of Kentucky, Lexington, Kentucky, USA.
  • Jenkins L; California Pacific Medical Center Research Institute and Forbes Norris MDA/ALS Research and Treatment Center, San Francisco, California, USA.
  • Ladha SS; California Pacific Medical Center Research Institute and Forbes Norris MDA/ALS Research and Treatment Center, San Francisco, California, USA.
  • Miller TM; Department of Neurology, Barrow Neurological Institute, Phoenix, Arizona, USA.
  • Scelsa SN; Hope Center for Neurological Disorders, Washington University in Saint Louis School of Medicine, Saint Louis, Missouri, USA.
  • Vu TH; Department of Neurology, Mount Sinai Beth Israel, Icahn School of Medicine at Mount Sinai, New York, New York, USA.
  • Fournier C; Department of Neurology, University of South Florida Morsani College of Medicine, Tampa, Florida, USA.
  • Johnson KM; Department of Neurology, Emory University, Atlanta, Georgia, USA.
  • Swenson A; Department of Neurology, Ochsner Health System, New Orleans, Louisiana, USA.
  • Goyal N; Department of Neurology, University of Iowa Health Care, Iowa City, Iowa, USA.
  • Pattee GL; Department of Neurology, University of California Irvine School of Medicine, Irvine, California, USA.
  • Babu S; Neurology Associates, Lincoln, Nebraska, USA.
  • Chase M; Harvard Medical School, Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, Massachusetts, USA.
  • Dagostino D; Harvard Medical School, Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, Massachusetts, USA.
  • Hall M; Harvard Medical School, Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, Massachusetts, USA.
  • Kittle G; Department of Neurology, Barrow Neurological Institute, Phoenix, Arizona, USA.
  • Eydinov M; Department of Neurology, Barrow Neurological Institute, Phoenix, Arizona, USA.
  • Ostrow J; Harvard Medical School, Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, Massachusetts, USA.
  • Pothier L; Harvard Medical School, Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, Massachusetts, USA.
  • Randall R; Harvard Medical School, Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, Massachusetts, USA.
  • Shefner JM; Worldwide Clinical Trials, Research Triangle Park, North Carolina, USA.
  • Sherman AV; Formerly With Gregory W. Fulton ALS Center, Barrow Neurological Institute, Phoenix, Arizona, USA.
  • Tustison E; Department of Neurology, Barrow Neurological Institute, Phoenix, Arizona, USA.
  • Vigneswaran P; Harvard Medical School, Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, Massachusetts, USA.
  • Yu H; Harvard Medical School, Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, Massachusetts, USA.
  • Cohen J; Harvard Medical School, Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, Massachusetts, USA.
  • Klee J; Harvard Medical School, Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, Massachusetts, USA.
  • Tanzi R; Amylyx Pharmaceuticals Inc, Cambridge, Massachusetts, USA.
  • Gilbert W; Amylyx Pharmaceuticals Inc, Cambridge, Massachusetts, USA.
  • Yeramian P; Department of Neurology, Genetics and Aging Research Unit, McCance Center for Brain Health, Massachusetts General Hospital, Harvard University, Boston, Massachusetts, USA.
  • Cudkowicz M; Carl M. Loeb University Professor Emeritus and Chair of the Society of Fellows at Harvard, Harvard University, Cambridge, Massachusetts, USA.
J Neurol Neurosurg Psychiatry ; 2022 May 16.
Article em En | MEDLINE | ID: mdl-35577511
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.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Clinical_trials Idioma: En Revista: J Neurol Neurosurg Psychiatry Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
Adicionar na Minha BVS
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Clinical_trials Idioma: En Revista: J Neurol Neurosurg Psychiatry Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Estados Unidos