Abstract
Primary biliary cholangitis (PBC) is a rare chronic cholestatic liver disease that may progress to biliary cirrhosis if left untreated. The first-line therapy for PBC is ursodeoxycholic acid (UDCA). Unfortunately, 1 of 3 patients does not respond to UDCA. These patients are at risk for developing clinical events, including cirrhosis, complications of portal hypertension, hepatocellular carcinoma, liver transplant, or death. Recently, the U.S. Food and Drug Administration approved obeticholic acid to be used in certain patients with PBC. Off-label therapies are also used, and several other therapies are currently under evaluation. Real-world effectiveness of newly approved and off-label therapies remains unknown. TARGET-PBC is a 5-year, longitudinal, observational study of patients with PBC that will evaluate the effectiveness of clinical practice interventions and provide practical information unobtainable in registration trials. Enrollment will take place at both academic and community sites. In addition to consenting to medical records review, participants will be asked to provide an annual blood sample and complete patient reported outcome surveys at predetermined intervals. Any available liver biopsies will be digitally pre...Continue Reading
References
May 12, 1994·The New England Journal of Medicine·R E PouponB Balkau
Sep 1, 1997·Gastroenterology·R E PouponE J Heathcote
Mar 10, 2001·Statistics in Medicine·R B Geskus
Jun 18, 2005·Gut·A JacobyD E J Jones
Mar 15, 2006·Gastroenterology·Albert ParésJuan Rodés
Apr 20, 2007·Medical Care·David CellaUNKNOWN PROMIS Cooperative Group
Dec 10, 2009·The British Journal of Dermatology·S ElmanM J Mayo
Aug 28, 2012·Medical Care·Sara AhmedUNKNOWN International Society for Quality of Life Research
Apr 2, 2013·Clinics in Liver Disease·Frank CzulCynthia Levy
May 9, 2015·World Journal of Hepatology·Treta Purohit, Mitchell S Cappell
Aug 12, 2015·Gastroenterology·Willem J LammersUNKNOWN Global PBC Study Group
Sep 15, 2015·Lancet·Elizabeth J CareyKeith D Lindor
Aug 18, 2016·The New England Journal of Medicine·Frederik NevensUNKNOWN POISE Study Group
Sep 7, 2016·PloS One·Floor A C BerdenWietske Kievit
Aug 2, 2017·Gastroenterology·Poonam MishraDebra Birnkrant
Citations
Sep 7, 2019·Digestive Diseases and Sciences·Artin GaloosianRobert J Wong
Aug 7, 2020·Liver International : Official Journal of the International Association for the Study of the Liver·Domenico AlvaroPietro Invernizzi
Jul 18, 2018·Hepatology : Official Journal of the American Association for the Study of Liver Diseases·Mehmet SayinerZobair M Younossi
Dec 19, 2019·Alimentary Pharmacology & Therapeutics·Christopher L Bowlus, Cynthia Levy
Mar 17, 2021·Clinics and Research in Hepatology and Gastroenterology·Anand V KulkarniNagaraja Rao Padaki