1. A Placebo-Controlled Trial of Obeticholic Acid in Primary Biliary Cholangitis
- Author
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Nevens, F, Andreone, P, Mazzella, G, Strasser, S, Bowlus, C, Drenth, J, Pockros, P, Regula, J, Beuers, U, Trauner, M, Jones, D, Floreani, A, Hohenester, S, Luketic, V, Shiffman, M, van Erpecum, K, Vargas, V, Vincent, C, Hirschfield, G, Shah, H, Hansen, B, Lindor, K, Marschall, H, Kowdley, K, Hooshmand Rad, R, Marmon, T, Sheeron, S, Pencek, R, Macconell, L, Pruzanski, M, Shapiro, D. Collaborator: Carbone, INVERNIZZI, PIETRO, DIPARTIMENTO DI SCIENZE MEDICHE E CHIRURGICHE, Facolta' di MEDICINA e CHIRURGIA, AREA MIN. 06 - Scienze mediche, Gastroenterology & Hepatology, AGEM - Amsterdam Gastroenterology Endocrinology Metabolism, Gastroenterology and Hepatology, Graduate School, Other departments, Nevens, F., Andreone, P., Mazzella, G., Strasser, S.I., Bowlus, C., Invernizzi, P., Drenth, J.P.H., Pockros, P.J., Regula, J., Beuers, U., Trauner, M., Jones, D.E., Floreani, A., Hohenester, S., Luketic, V., Shiffman, M., Van Erpecum, K.J., Vargas, V., Vincent, C., Hirschfield, G.M., Shah, H., Hansen, B., Lindor, K.D., Marschall, H.-U., Kowdley, K.V., Hooshmand-Rad, R., Marmon, T., Sheeron, S., Pencek, R., Macconell, L., Pruzanski, M., Shapiro, D., Nevens, F, Andreone, P, Mazzella, G, Strasser, S, Bowlus, C, Invernizzi, P, Drenth, J, Pockros, P, Regula, J, Beuers, U, Trauner, M, Jones, D, Floreani, A, Hohenester, S, Luketic, V, Shiffman, M, van Erpecum, K, Vargas, V, Vincent, C, Hirschfield, G, Shah, H, Hansen, B, Lindor, K, Marschall, H, Kowdley, K, Hooshmand Rad, R, Marmon, T, Sheeron, S, Pencek, R, Macconell, L, Pruzanski, M, Shapiro, and D., C
- Subjects
Liver Cirrhosis ,Male ,0301 basic medicine ,Cirrhosis ,Fibroblast Growth Factor ,medicine.medical_treatment ,Clinical Trial, Phase III ,Placebo-controlled study ,Liver transplantation ,Gastroenterology ,Pruritu ,chemistry.chemical_compound ,0302 clinical medicine ,Primary biliary cirrhosis ,Bone Density ,Chenodeoxycholic acid ,Adult ,Aged ,Alkaline Phosphatase ,Bile Acids and Salts ,Chenodeoxycholic Acid ,Double-Blind Method ,Female ,Fibroblast Growth Factors ,Humans ,Liver Cirrhosis, Biliary ,Middle Aged ,Pruritus ,Non-U.S. Gov't ,Medicine (all) ,Research Support, Non-U.S. Gov't ,Biliary ,Obeticholic acid ,General Medicine ,Clinical Trial ,Bile Acids and Salt ,Multicenter Study ,Randomized Controlled Trial ,030211 gastroenterology & hepatology ,Human ,medicine.medical_specialty ,Randomization ,Liver Cirrhosi ,Research Support ,Placebo ,03 medical and health sciences ,Phase III ,Internal medicine ,Journal Article ,medicine ,business.industry ,medicine.disease ,Surgery ,Renal disorders Radboud Institute for Molecular Life Sciences [Radboudumc 11] ,030104 developmental biology ,chemistry ,business - Abstract
none 32 BACKGROUND Primary biliary cholangitis (formerly called primary biliary cirrhosis) can progress to cirrhosis and death despite ursodiol therapy. Alkaline phosphatase and bilirubin levels correlate with the risk of liver transplantation or death. Obeticholic acid, a farnesoid X receptor agonist, has shown potential benefit in patients with this disease. METHODS In this 12-month, double-blind, placebo-controlled, phase 3 trial, we randomly assigned 217 patients who had an inadequate response to ursodiol or who found the side effects of ursodiol unacceptable to receive obeticholic acid at a dose of 10 mg (the 10-mg group), obeticholic acid at a dose of 5 mg with adjustment to 10 mg if applicable (the 5-10-mg group), or placebo. The primary end point was an alkaline phosphatase level of less than 1.67 times the upper limit of the normal range, with a reduction of at least 15% from baseline, and a normal total bilirubin level. RESULTS Of 216 patients who underwent randomization and received at least one dose of obeticholic acid or placebo, 93% received ursodiol as background therapy. The primary end point occurred in more patients in the 5-10-mg group (46%) and the 10-mg group (47%) than in the placebo group (10%; P
- Published
- 2016