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Long- term survival of participants in the CENTAUR trial of sodium phenylbutyrate- taurursodiol in amyotrophic lateral sclerosis
dc.contributor.authorPaganoni, Sabrina
dc.contributor.authorHendrix, Suzanne
dc.contributor.authorDickson, Samuel P.
dc.contributor.authorKnowlton, Newman
dc.contributor.authorMacklin, Eric A.
dc.contributor.authorBerry, James D.
dc.contributor.authorElliott, Michael A.
dc.contributor.authorMaiser, Samuel
dc.contributor.authorKaram, Chafic
dc.contributor.authorCaress, James B.
dc.contributor.authorOwegi, Margaret Ayo
dc.contributor.authorQuick, Adam
dc.contributor.authorWymer, James
dc.contributor.authorGoutman, Stephen A.
dc.contributor.authorHeitzman, Daragh
dc.contributor.authorHeiman‐patterson, Terry D.
dc.contributor.authorJackson, Carlayne E.
dc.contributor.authorQuinn, Colin
dc.contributor.authorRothstein, Jeffrey D.
dc.contributor.authorKasarskis, Edward J.
dc.contributor.authorKatz, Jonathan
dc.contributor.authorJenkins, Liberty
dc.contributor.authorLadha, Shafeeq
dc.contributor.authorMiller, Timothy M.
dc.contributor.authorScelsa, Stephen N.
dc.contributor.authorVu, Tuan H.
dc.contributor.authorFournier, Christina N.
dc.contributor.authorGlass, Jonathan D.
dc.contributor.authorJohnson, Kristin M.
dc.contributor.authorSwenson, Andrea
dc.contributor.authorGoyal, Namita A.
dc.contributor.authorPattee, Gary L.
dc.contributor.authorAndres, Patricia L.
dc.contributor.authorBabu, Suma
dc.contributor.authorChase, Marianne
dc.contributor.authorDagostino, Derek
dc.contributor.authorHall, Meghan
dc.contributor.authorKittle, Gale
dc.contributor.authorEydinov, Matthew
dc.contributor.authorMcGovern, Michelle
dc.contributor.authorOstrow, Joseph
dc.contributor.authorPothier, Lindsay
dc.contributor.authorRandall, Rebecca
dc.contributor.authorShefner, Jeremy M.
dc.contributor.authorSherman, Alexander V.
dc.contributor.authorSt Pierre, Maria E.
dc.contributor.authorTustison, Eric
dc.contributor.authorVigneswaran, Prasha
dc.contributor.authorWalker, Jason
dc.contributor.authorYu, Hong
dc.contributor.authorChan, James
dc.contributor.authorWittes, Janet
dc.contributor.authorYu, Zi‐fan
dc.contributor.authorCohen, Joshua
dc.contributor.authorKlee, Justin
dc.contributor.authorLeslie, Kent
dc.contributor.authorTanzi, Rudolph E.
dc.contributor.authorGilbert, Walter
dc.contributor.authorYeramian, Patrick D.
dc.contributor.authorSchoenfeld, David
dc.contributor.authorCudkowicz, Merit E.
dc.date.accessioned2021-01-05T18:48:52Z
dc.date.availableWITHHELD_13_MONTHS
dc.date.available2021-01-05T18:48:52Z
dc.date.issued2021-01
dc.identifier.citationPaganoni, Sabrina; Hendrix, Suzanne; Dickson, Samuel P.; Knowlton, Newman; Macklin, Eric A.; 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 E.; Quinn, Colin; Rothstein, Jeffrey D.; Kasarskis, Edward J.; Katz, Jonathan; Jenkins, Liberty; Ladha, Shafeeq; Miller, Timothy M.; Scelsa, Stephen N.; Vu, Tuan H.; Fournier, Christina N.; Glass, Jonathan D.; Johnson, Kristin M.; Swenson, Andrea; Goyal, Namita A.; Pattee, Gary L.; Andres, Patricia L.; Babu, Suma; Chase, Marianne; Dagostino, Derek; Hall, Meghan; Kittle, Gale; Eydinov, Matthew; McGovern, Michelle; Ostrow, Joseph; Pothier, Lindsay; Randall, Rebecca; Shefner, Jeremy M.; Sherman, Alexander V.; St Pierre, Maria E.; Tustison, Eric; Vigneswaran, Prasha; Walker, Jason; Yu, Hong; Chan, James; Wittes, Janet; Yu, Zi‐fan ; Cohen, Joshua; Klee, Justin; Leslie, Kent; Tanzi, Rudolph E.; Gilbert, Walter; Yeramian, Patrick D.; Schoenfeld, David; Cudkowicz, Merit E. (2021). "Long- term survival of participants in the CENTAUR trial of sodium phenylbutyrate- taurursodiol in amyotrophic lateral sclerosis." Muscle & Nerve 63(1): 31-39.
dc.identifier.issn0148-639X
dc.identifier.issn1097-4598
dc.identifier.urihttps://hdl.handle.net/2027.42/163951
dc.description.abstractAn 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.
dc.publisherJohn Wiley & Sons, Inc.
dc.subject.otheramyotrophic lateral sclerosis
dc.subject.otherCENTAUR
dc.subject.othermotor neuron disease
dc.subject.othersodium phenylbutyrate- taurursodiol
dc.subject.othersurvival analysis
dc.titleLong- term survival of participants in the CENTAUR trial of sodium phenylbutyrate- taurursodiol in amyotrophic lateral sclerosis
dc.typeArticle
dc.rights.robotsIndexNoFollow
dc.subject.hlbsecondlevelNeurosciences
dc.subject.hlbtoplevelHealth Sciences
dc.description.peerreviewedPeer Reviewed
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/163951/1/mus27091.pdf
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/163951/2/mus27091_am.pdf
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/163951/3/mus27091-sup-0002-FigureS1.pdf
dc.identifier.doi10.1002/mus.27091
dc.identifier.sourceMuscle & Nerve
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