Efficacy and Safety of Elafibranor in Primary Biliary Cholangitis

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dc.contributor.authorKowdley, Kris V.
dc.contributor.authorBowlus, Christopher L.
dc.contributor.authorLevy, Cynthia
dc.contributor.authorAkarca, Ulus S.
dc.contributor.authorAlvares-da-Silva, Mario Reis
dc.contributor.authorAndreone, Pietro
dc.contributor.authorArrese, Marco
dc.contributor.authorCorpechot, Christophe
dc.contributor.authorFrancque, Sven M.
dc.contributor.authorHeneghan, Michael A.
dc.contributor.authorInvernizzi, Pietro
dc.contributor.authorJones, David
dc.contributor.authorKruger, Frederik C.
dc.contributor.authorLawitz, Eric
dc.contributor.authorMayo, Marlyn J.
dc.contributor.authorShiffman, Mitchell L.
dc.contributor.authorSwain, Mark G.
dc.contributor.authorValera, Jose Miguel
dc.contributor.authorVargas, Victor
dc.contributor.authorVierling, John M.
dc.contributor.authorVillamil, Alejandra
dc.contributor.authorAddy, Carol
dc.contributor.authorDietrich, Julie
dc.contributor.authorGermain, Jean-Michel
dc.contributor.authorMazain, Sarah
dc.contributor.authorRafailovic, Dragutin
dc.contributor.authorTadde, Bachirou
dc.contributor.authorMiller, Benjamin
dc.contributor.authorShu, Jianfen
dc.contributor.authorZein, Claudia O.
dc.contributor.authorSchattenberg, Jorn M.
dc.date.accessioned2025-01-20T17:09:16Z
dc.date.available2025-01-20T17:09:16Z
dc.date.issued2024
dc.description.abstractBackground Primary biliary cholangitis is a rare, chronic cholestatic liver disease characterized by the destruction of interlobular bile ducts, leading to cholestasis and liver fibrosis. Whether elafibranor, an oral, dual peroxisome proliferator-activated receptor (PPAR) alpha and delta agonist, may have benefit as a treatment for primary biliary cholangitis is unknown.
dc.description.abstractMethods In this multinational, phase 3, double-blind, placebo-controlled trial, we randomly assigned (in a 2:1 ratio) patients with primary biliary cholangitis who had had an inadequate response to or unacceptable side effects with ursodeoxycholic acid to receive once-daily elafibranor, at a dose of 80 mg, or placebo. The primary end point was a biochemical response (defined as an alkaline phosphatase level of <1.67 times the upper limit of the normal range, with a reduction of >= 15% from baseline, and normal total bilirubin levels) at week 52. Key secondary end points were normalization of the alkaline phosphatase level at week 52 and a change in pruritus intensity from baseline through week 52 and through week 24, as measured on the Worst Itch Numeric Rating Scale (WI-NRS; scores range from 0 [no itch] to 10 [worst itch imaginable]).
dc.description.abstractResults A total of 161 patients underwent randomization. A biochemical response (the primary end point) was observed in 51% of the patients (55 of 108) who received elafibranor and in 4% (2 of 53) who received placebo, for a difference of 47 percentage points (95% confidence interval [CI], 32 to 57; P<0.001). The alkaline phosphatase level normalized in 15% of the patients in the elafibranor group and in none of the patients in the placebo group at week 52 (difference, 15 percentage points; 95% CI, 6 to 23; P=0.002). Among patients who had moderate-to-severe pruritus (44 patients in the elafibranor group and 22 in the placebo group), the least-squares mean change from baseline through week 52 on the WI-NRS did not differ significantly between the groups (-1.93 vs. -1.15; difference, -0.78; 95% CI, -1.99 to 0.42; P=0.20). Adverse events that occurred more frequently with elafibranor than with placebo included abdominal pain, diarrhea, nausea, and vomiting.
dc.description.abstractConclusions Treatment with elafibranor resulted in significantly greater improvements in relevant biochemical indicators of cholestasis than placebo. (Funded by GENFIT and Ipsen; ELATIVE ClinicalTrials.gov number, NCT04526665.)
dc.fuente.origenWOS
dc.identifier.doi10.1056/NEJMoa2306185
dc.identifier.eissn1533-4406
dc.identifier.issn0028-4793
dc.identifier.urihttps://doi.org/10.1056/NEJMoa2306185
dc.identifier.urihttps://repositorio.uc.cl/handle/11534/91027
dc.identifier.wosidWOS:001149847900001
dc.issue.numero9
dc.language.isoen
dc.pagina.final805
dc.pagina.inicio795
dc.revistaNew england journal of medicine
dc.rightsacceso restringido
dc.subject.ods03 Good Health and Well-being
dc.subject.odspa03 Salud y bienestar
dc.titleEfficacy and Safety of Elafibranor in Primary Biliary Cholangitis
dc.typeartículo
dc.volumen390
sipa.indexWOS
sipa.trazabilidadWOS;2025-01-12
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