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Primary biliary cholangitis 5(PBC5)

MedGen UID:
481832
•Concept ID:
C3280202
•
Disease or Syndrome
Synonym: Biliary cirrhosis, primary, 5
 
Monarch Initiative: MONDO:0013637
OMIM®: 614221

Definition

Primary biliary cirrhosis (PBC) is a chronic, progressive cholestatic liver disease that usually affects middle-aged women and eventually leads to liver failure (summary by Kaplan, 1996). For a discussion of genetic heterogeneity of primary biliary cirrhosis (PBC), see PBC1 (109720). [from OMIM]

Professional guidelines

PubMed

Diagnosis and management of choledochal cysts.
Brown ZJ, Baghdadi A, Kamel I, Labiner HE, Hewitt DB, Pawlik TM
HPB (Oxford) 2023 Jan;25(1):14-25. Epub 2022 Oct 5 doi: 10.1016/j.hpb.2022.09.010. PMID: 36257874
An international genome-wide meta-analysis of primary biliary cholangitis: Novel risk loci and candidate drugs.
Cordell HJ, Fryett JJ, Ueno K, Darlay R, Aiba Y, Hitomi Y, Kawashima M, Nishida N, Khor SS, Gervais O, Kawai Y, Nagasaki M, Tokunaga K, Tang R, Shi Y, Li Z, Juran BD, Atkinson EJ, Gerussi A, Carbone M, Asselta R, Cheung A, de Andrade M, Baras A, Horowitz J, Ferreira MAR, Sun D, Jones DE, Flack S, Spicer A, Mulcahy VL, Byan J, Han Y, Sandford RN, Lazaridis KN, Amos CI, Hirschfield GM, Seldin MF, Invernizzi P, Siminovitch KA, Ma X, Nakamura M, Mells GF; PBC Consortia; Canadian PBC Consortium; Chinese PBC Consortium; Italian PBC Study Group; Japan-PBC-GWAS Consortium; US PBC Consortium; UK-PBC Consortium
J Hepatol 2021 Sep;75(3):572-581. Epub 2021 May 23 doi: 10.1016/j.jhep.2021.04.055. PMID: 34033851Free PMC Article
Jaundice revisited: recent advances in the diagnosis and treatment of inherited cholestatic liver diseases.
Chen HL, Wu SH, Hsu SH, Liou BY, Chen HL, Chang MH
J Biomed Sci 2018 Oct 26;25(1):75. doi: 10.1186/s12929-018-0475-8. PMID: 30367658Free PMC Article

Recent clinical studies

Etiology

Primary biliary cholangitis drug evaluation and regulatory approval: Where do we go from here?
Jones DEJ, Beuers U, Bonder A, Carbone M, Culver E, Dyson J, Gish RG, Hansen BE, Hirschfield G, Jones R, Kowdley K, Kremer AE, Lindor K, Mayo M, Mells G, Neuberger J, Prince M, Swain M, Tanaka A, Thorburn D, Trauner M, Trivedi P, Weltman M, Yeoman A, Levy C
Hepatology 2024 Nov 1;80(5):1291-1300. Epub 2024 Mar 22 doi: 10.1097/HEP.0000000000000864. PMID: 38506926Free PMC Article
A Phase 3 Trial of Seladelpar in Primary Biliary Cholangitis.
Hirschfield GM, Bowlus CL, Mayo MJ, Kremer AE, Vierling JM, Kowdley KV, Levy C, Villamil A, Ladrón de Guevara Cetina AL, Janczewska E, Zigmond E, Jeong SH, Yilmaz Y, Kallis Y, Corpechot C, Buggisch P, Invernizzi P, Londoño Hurtado MC, Bergheanu S, Yang K, Choi YJ, Crittenden DB, McWherter CA; RESPONSE Study Group
N Engl J Med 2024 Feb 29;390(9):783-794. Epub 2024 Feb 21 doi: 10.1056/NEJMoa2312100. PMID: 38381664
An international genome-wide meta-analysis of primary biliary cholangitis: Novel risk loci and candidate drugs.
Cordell HJ, Fryett JJ, Ueno K, Darlay R, Aiba Y, Hitomi Y, Kawashima M, Nishida N, Khor SS, Gervais O, Kawai Y, Nagasaki M, Tokunaga K, Tang R, Shi Y, Li Z, Juran BD, Atkinson EJ, Gerussi A, Carbone M, Asselta R, Cheung A, de Andrade M, Baras A, Horowitz J, Ferreira MAR, Sun D, Jones DE, Flack S, Spicer A, Mulcahy VL, Byan J, Han Y, Sandford RN, Lazaridis KN, Amos CI, Hirschfield GM, Seldin MF, Invernizzi P, Siminovitch KA, Ma X, Nakamura M, Mells GF; PBC Consortia; Canadian PBC Consortium; Chinese PBC Consortium; Italian PBC Study Group; Japan-PBC-GWAS Consortium; US PBC Consortium; UK-PBC Consortium
J Hepatol 2021 Sep;75(3):572-581. Epub 2021 May 23 doi: 10.1016/j.jhep.2021.04.055. PMID: 34033851Free PMC Article
A Placebo-Controlled Trial of Bezafibrate in Primary Biliary Cholangitis.
Corpechot C, Chazouillères O, Rousseau A, Le Gruyer A, Habersetzer F, Mathurin P, Goria O, Potier P, Minello A, Silvain C, Abergel A, Debette-Gratien M, Larrey D, Roux O, Bronowicki JP, Boursier J, de Ledinghen V, Heurgue-Berlot A, Nguyen-Khac E, Zoulim F, Ollivier-Hourmand I, Zarski JP, Nkontchou G, Lemoinne S, Humbert L, Rainteau D, Lefèvre G, de Chaisemartin L, Chollet-Martin S, Gaouar F, Admane FH, Simon T, Poupon R
N Engl J Med 2018 Jun 7;378(23):2171-2181. doi: 10.1056/NEJMoa1714519. PMID: 29874528
A Placebo-Controlled Trial of Obeticholic Acid in Primary Biliary Cholangitis.
Nevens F, Andreone P, Mazzella G, Strasser SI, Bowlus C, Invernizzi P, Drenth JP, Pockros PJ, Regula J, Beuers U, Trauner M, Jones DE, Floreani A, Hohenester S, Luketic V, Shiffman M, van Erpecum KJ, Vargas V, Vincent C, Hirschfield GM, Shah H, Hansen B, Lindor KD, Marschall HU, Kowdley KV, Hooshmand-Rad R, Marmon T, Sheeron S, Pencek R, MacConell L, Pruzanski M, Shapiro D; POISE Study Group
N Engl J Med 2016 Aug 18;375(7):631-43. doi: 10.1056/NEJMoa1509840. PMID: 27532829

Diagnosis

A large-scale plasma proteome Mendelian randomization study identifies novel causal plasma proteins related to primary biliary cholangitis.
Yang H, Chen L, Liu Y
Front Immunol 2023;14:1052616. Epub 2023 Feb 7 doi: 10.3389/fimmu.2023.1052616. PMID: 36825008Free PMC Article
Impact of Pruritus on Quality of Life and Current Treatment Patterns in Patients with Primary Biliary Cholangitis.
Mayo MJ, Carey E, Smith HT, Mospan AR, McLaughlin M, Thompson A, Morris HL, Sandefur R, Kim WR, Bowlus C; TARGET-PBC Investigators, Levy C
Dig Dis Sci 2023 Mar;68(3):995-1005. Epub 2022 Jun 15 doi: 10.1007/s10620-022-07581-x. PMID: 35704252Free PMC Article
Clinical features of anti-mitochondrial M2 antibody-positive myositis: case series of 17 patients.
Nagai A, Nagai T, Yaguchi H, Fujii S, Uwatoko H, Shirai S, Horiuchi K, Iwata I, Matsushima M, Ura S, Anzai T, Yabe I
J Neurol Sci 2022 Nov 15;442:120391. Epub 2022 Aug 28 doi: 10.1016/j.jns.2022.120391. PMID: 36115217
A randomized placebo-controlled trial of elafibranor in patients with primary biliary cholangitis and incomplete response to UDCA.
Schattenberg JM, Pares A, Kowdley KV, Heneghan MA, Caldwell S, Pratt D, Bonder A, Hirschfield GM, Levy C, Vierling J, Jones D, Tailleux A, Staels B, Megnien S, Hanf R, Magrez D, Birman P, Luketic V
J Hepatol 2021 Jun;74(6):1344-1354. Epub 2021 Jan 21 doi: 10.1016/j.jhep.2021.01.013. PMID: 33484775
Fibrates for Itch (FITCH) in Fibrosing Cholangiopathies: A Double-Blind, Randomized, Placebo-Controlled Trial.
de Vries E, Bolier R, Goet J, Parés A, Verbeek J, de Vree M, Drenth J, van Erpecum K, van Nieuwkerk K, van der Heide F, Mostafavi N, Helder J, Ponsioen C, Oude Elferink R, van Buuren H, Beuers U; Netherlands Association for the Study of the Liver-Cholestasis Working Group
Gastroenterology 2021 Feb;160(3):734-743.e6. Epub 2020 Oct 5 doi: 10.1053/j.gastro.2020.10.001. PMID: 33031833

Therapy

A Phase 3 Trial of Seladelpar in Primary Biliary Cholangitis.
Hirschfield GM, Bowlus CL, Mayo MJ, Kremer AE, Vierling JM, Kowdley KV, Levy C, Villamil A, Ladrón de Guevara Cetina AL, Janczewska E, Zigmond E, Jeong SH, Yilmaz Y, Kallis Y, Corpechot C, Buggisch P, Invernizzi P, Londoño Hurtado MC, Bergheanu S, Yang K, Choi YJ, Crittenden DB, McWherter CA; RESPONSE Study Group
N Engl J Med 2024 Feb 29;390(9):783-794. Epub 2024 Feb 21 doi: 10.1056/NEJMoa2312100. PMID: 38381664
Seladelpar efficacy and safety at 3 months in patients with primary biliary cholangitis: ENHANCE, a phase 3, randomized, placebo-controlled study.
Hirschfield GM, Shiffman ML, Gulamhusein A, Kowdley KV, Vierling JM, Levy C, Kremer AE, Zigmond E, Andreone P, Gordon SC, Bowlus CL, Lawitz EJ, Aspinall RJ, Pratt DS, Raikhelson K, Gonzalez-Huezo MS, Heneghan MA, Jeong SH, Ladrón de Guevara AL, Mayo MJ, Dalekos GN, Drenth JPH, Janczewska E, Leggett BA, Nevens F, Vargas V, Zuckerman E, Corpechot C, Fassio E, Hinrichsen H, Invernizzi P, Trivedi PJ, Forman L, Jones DEJ, Ryder SD, Swain MG, Steinberg A, Boudes PF, Choi YJ, McWherter CA; ENHANCE Study Group*
Hepatology 2023 Aug 1;78(2):397-415. Epub 2023 Apr 6 doi: 10.1097/HEP.0000000000000395. PMID: 37386786Free PMC Article
Fibrates for Itch (FITCH) in Fibrosing Cholangiopathies: A Double-Blind, Randomized, Placebo-Controlled Trial.
de Vries E, Bolier R, Goet J, Parés A, Verbeek J, de Vree M, Drenth J, van Erpecum K, van Nieuwkerk K, van der Heide F, Mostafavi N, Helder J, Ponsioen C, Oude Elferink R, van Buuren H, Beuers U; Netherlands Association for the Study of the Liver-Cholestasis Working Group
Gastroenterology 2021 Feb;160(3):734-743.e6. Epub 2020 Oct 5 doi: 10.1053/j.gastro.2020.10.001. PMID: 33031833
A Placebo-Controlled Trial of Bezafibrate in Primary Biliary Cholangitis.
Corpechot C, Chazouillères O, Rousseau A, Le Gruyer A, Habersetzer F, Mathurin P, Goria O, Potier P, Minello A, Silvain C, Abergel A, Debette-Gratien M, Larrey D, Roux O, Bronowicki JP, Boursier J, de Ledinghen V, Heurgue-Berlot A, Nguyen-Khac E, Zoulim F, Ollivier-Hourmand I, Zarski JP, Nkontchou G, Lemoinne S, Humbert L, Rainteau D, Lefèvre G, de Chaisemartin L, Chollet-Martin S, Gaouar F, Admane FH, Simon T, Poupon R
N Engl J Med 2018 Jun 7;378(23):2171-2181. doi: 10.1056/NEJMoa1714519. PMID: 29874528
A Placebo-Controlled Trial of Obeticholic Acid in Primary Biliary Cholangitis.
Nevens F, Andreone P, Mazzella G, Strasser SI, Bowlus C, Invernizzi P, Drenth JP, Pockros PJ, Regula J, Beuers U, Trauner M, Jones DE, Floreani A, Hohenester S, Luketic V, Shiffman M, van Erpecum KJ, Vargas V, Vincent C, Hirschfield GM, Shah H, Hansen B, Lindor KD, Marschall HU, Kowdley KV, Hooshmand-Rad R, Marmon T, Sheeron S, Pencek R, MacConell L, Pruzanski M, Shapiro D; POISE Study Group
N Engl J Med 2016 Aug 18;375(7):631-43. doi: 10.1056/NEJMoa1509840. PMID: 27532829

Prognosis

Clinical features of anti-mitochondrial M2 antibody-positive myositis: case series of 17 patients.
Nagai A, Nagai T, Yaguchi H, Fujii S, Uwatoko H, Shirai S, Horiuchi K, Iwata I, Matsushima M, Ura S, Anzai T, Yabe I
J Neurol Sci 2022 Nov 15;442:120391. Epub 2022 Aug 28 doi: 10.1016/j.jns.2022.120391. PMID: 36115217
Liver stiffness measurement by vibration-controlled transient elastography improves outcome prediction in primary biliary cholangitis.
Corpechot C, Carrat F, Gaouar F, Chau F, Hirschfield G, Gulamhusein A, Montano-Loza AJ, Lytvyak E, Schramm C, Pares A, Olivas I, Eaton JE, Osman KT, Dalekos G, Gatselis N, Nevens F, Cazzagon N, Zago A, Russo FP, Abbas N, Trivedi P, Thorburn D, Saffioti F, Barkai L, Roccarina D, Calvaruso V, Fichera A, Delamarre A, Medina-Morales E, Bonder A, Patwardhan V, Rigamonti C, Carbone M, Invernizzi P, Cristoferi L, van der Meer A, de Veer R, Zigmond E, Yehezkel E, Kremer AE, Deibel A, Dumortier J, Bruns T, Große K, Pageaux GP, Wetten A, Dyson J, Jones D, Chazouillères O, Hansen B, de Lédinghen V; Global & ERN Rare-Liver PBC Study Groups
J Hepatol 2022 Dec;77(6):1545-1553. Epub 2022 Jun 28 doi: 10.1016/j.jhep.2022.06.017. PMID: 35777587
An international genome-wide meta-analysis of primary biliary cholangitis: Novel risk loci and candidate drugs.
Cordell HJ, Fryett JJ, Ueno K, Darlay R, Aiba Y, Hitomi Y, Kawashima M, Nishida N, Khor SS, Gervais O, Kawai Y, Nagasaki M, Tokunaga K, Tang R, Shi Y, Li Z, Juran BD, Atkinson EJ, Gerussi A, Carbone M, Asselta R, Cheung A, de Andrade M, Baras A, Horowitz J, Ferreira MAR, Sun D, Jones DE, Flack S, Spicer A, Mulcahy VL, Byan J, Han Y, Sandford RN, Lazaridis KN, Amos CI, Hirschfield GM, Seldin MF, Invernizzi P, Siminovitch KA, Ma X, Nakamura M, Mells GF; PBC Consortia; Canadian PBC Consortium; Chinese PBC Consortium; Italian PBC Study Group; Japan-PBC-GWAS Consortium; US PBC Consortium; UK-PBC Consortium
J Hepatol 2021 Sep;75(3):572-581. Epub 2021 May 23 doi: 10.1016/j.jhep.2021.04.055. PMID: 34033851Free PMC Article
Fibrates and cholestasis.
Ghonem NS, Assis DN, Boyer JL
Hepatology 2015 Aug;62(2):635-43. Epub 2015 Mar 23 doi: 10.1002/hep.27744. PMID: 25678132Free PMC Article
Antimitochondrial antibody-negative primary biliary cirrhosis.
Hirschfield GM, Heathcote EJ
Clin Liver Dis 2008 May;12(2):323-31; viii-ix. doi: 10.1016/j.cld.2008.02.003. PMID: 18456183

Clinical prediction guides

A Phase 3 Trial of Seladelpar in Primary Biliary Cholangitis.
Hirschfield GM, Bowlus CL, Mayo MJ, Kremer AE, Vierling JM, Kowdley KV, Levy C, Villamil A, Ladrón de Guevara Cetina AL, Janczewska E, Zigmond E, Jeong SH, Yilmaz Y, Kallis Y, Corpechot C, Buggisch P, Invernizzi P, Londoño Hurtado MC, Bergheanu S, Yang K, Choi YJ, Crittenden DB, McWherter CA; RESPONSE Study Group
N Engl J Med 2024 Feb 29;390(9):783-794. Epub 2024 Feb 21 doi: 10.1056/NEJMoa2312100. PMID: 38381664
Seladelpar efficacy and safety at 3 months in patients with primary biliary cholangitis: ENHANCE, a phase 3, randomized, placebo-controlled study.
Hirschfield GM, Shiffman ML, Gulamhusein A, Kowdley KV, Vierling JM, Levy C, Kremer AE, Zigmond E, Andreone P, Gordon SC, Bowlus CL, Lawitz EJ, Aspinall RJ, Pratt DS, Raikhelson K, Gonzalez-Huezo MS, Heneghan MA, Jeong SH, Ladrón de Guevara AL, Mayo MJ, Dalekos GN, Drenth JPH, Janczewska E, Leggett BA, Nevens F, Vargas V, Zuckerman E, Corpechot C, Fassio E, Hinrichsen H, Invernizzi P, Trivedi PJ, Forman L, Jones DEJ, Ryder SD, Swain MG, Steinberg A, Boudes PF, Choi YJ, McWherter CA; ENHANCE Study Group*
Hepatology 2023 Aug 1;78(2):397-415. Epub 2023 Apr 6 doi: 10.1097/HEP.0000000000000395. PMID: 37386786Free PMC Article
An international genome-wide meta-analysis of primary biliary cholangitis: Novel risk loci and candidate drugs.
Cordell HJ, Fryett JJ, Ueno K, Darlay R, Aiba Y, Hitomi Y, Kawashima M, Nishida N, Khor SS, Gervais O, Kawai Y, Nagasaki M, Tokunaga K, Tang R, Shi Y, Li Z, Juran BD, Atkinson EJ, Gerussi A, Carbone M, Asselta R, Cheung A, de Andrade M, Baras A, Horowitz J, Ferreira MAR, Sun D, Jones DE, Flack S, Spicer A, Mulcahy VL, Byan J, Han Y, Sandford RN, Lazaridis KN, Amos CI, Hirschfield GM, Seldin MF, Invernizzi P, Siminovitch KA, Ma X, Nakamura M, Mells GF; PBC Consortia; Canadian PBC Consortium; Chinese PBC Consortium; Italian PBC Study Group; Japan-PBC-GWAS Consortium; US PBC Consortium; UK-PBC Consortium
J Hepatol 2021 Sep;75(3):572-581. Epub 2021 May 23 doi: 10.1016/j.jhep.2021.04.055. PMID: 34033851Free PMC Article
A Placebo-Controlled Trial of Bezafibrate in Primary Biliary Cholangitis.
Corpechot C, Chazouillères O, Rousseau A, Le Gruyer A, Habersetzer F, Mathurin P, Goria O, Potier P, Minello A, Silvain C, Abergel A, Debette-Gratien M, Larrey D, Roux O, Bronowicki JP, Boursier J, de Ledinghen V, Heurgue-Berlot A, Nguyen-Khac E, Zoulim F, Ollivier-Hourmand I, Zarski JP, Nkontchou G, Lemoinne S, Humbert L, Rainteau D, Lefèvre G, de Chaisemartin L, Chollet-Martin S, Gaouar F, Admane FH, Simon T, Poupon R
N Engl J Med 2018 Jun 7;378(23):2171-2181. doi: 10.1056/NEJMoa1714519. PMID: 29874528
A Placebo-Controlled Trial of Obeticholic Acid in Primary Biliary Cholangitis.
Nevens F, Andreone P, Mazzella G, Strasser SI, Bowlus C, Invernizzi P, Drenth JP, Pockros PJ, Regula J, Beuers U, Trauner M, Jones DE, Floreani A, Hohenester S, Luketic V, Shiffman M, van Erpecum KJ, Vargas V, Vincent C, Hirschfield GM, Shah H, Hansen B, Lindor KD, Marschall HU, Kowdley KV, Hooshmand-Rad R, Marmon T, Sheeron S, Pencek R, MacConell L, Pruzanski M, Shapiro D; POISE Study Group
N Engl J Med 2016 Aug 18;375(7):631-43. doi: 10.1056/NEJMoa1509840. PMID: 27532829

Recent systematic reviews

Incidence of Hepatocellular Carcinoma in Patients With Nonalcoholic Fatty Liver Disease: A Systematic Review, Meta-analysis, and Meta-regression.
Orci LA, Sanduzzi-Zamparelli M, Caballol B, Sapena V, Colucci N, Torres F, Bruix J, Reig M, Toso C
Clin Gastroenterol Hepatol 2022 Feb;20(2):283-292.e10. Epub 2021 May 28 doi: 10.1016/j.cgh.2021.05.002. PMID: 33965578
An international genome-wide meta-analysis of primary biliary cholangitis: Novel risk loci and candidate drugs.
Cordell HJ, Fryett JJ, Ueno K, Darlay R, Aiba Y, Hitomi Y, Kawashima M, Nishida N, Khor SS, Gervais O, Kawai Y, Nagasaki M, Tokunaga K, Tang R, Shi Y, Li Z, Juran BD, Atkinson EJ, Gerussi A, Carbone M, Asselta R, Cheung A, de Andrade M, Baras A, Horowitz J, Ferreira MAR, Sun D, Jones DE, Flack S, Spicer A, Mulcahy VL, Byan J, Han Y, Sandford RN, Lazaridis KN, Amos CI, Hirschfield GM, Seldin MF, Invernizzi P, Siminovitch KA, Ma X, Nakamura M, Mells GF; PBC Consortia; Canadian PBC Consortium; Chinese PBC Consortium; Italian PBC Study Group; Japan-PBC-GWAS Consortium; US PBC Consortium; UK-PBC Consortium
J Hepatol 2021 Sep;75(3):572-581. Epub 2021 May 23 doi: 10.1016/j.jhep.2021.04.055. PMID: 34033851Free PMC Article
Risk factors for intrahepatic and extrahepatic cholangiocarcinoma: A systematic review and meta-analysis.
Clements O, Eliahoo J, Kim JU, Taylor-Robinson SD, Khan SA
J Hepatol 2020 Jan;72(1):95-103. Epub 2019 Sep 16 doi: 10.1016/j.jhep.2019.09.007. PMID: 31536748
Long-Term Impact of Iatrogenic Bile Duct Injury.
Schreuder AM, Busch OR, Besselink MG, Ignatavicius P, Gulbinas A, Barauskas G, Gouma DJ, van Gulik TM
Dig Surg 2020;37(1):10-21. Epub 2019 Jan 17 doi: 10.1159/000496432. PMID: 30654363Free PMC Article
Meta-analysis: antibiotic prophylaxis for cirrhotic patients with upper gastrointestinal bleeding - an updated Cochrane review.
Chavez-Tapia NC, Barrientos-Gutierrez T, Tellez-Avila F, Soares-Weiser K, Mendez-Sanchez N, Gluud C, Uribe M
Aliment Pharmacol Ther 2011 Sep;34(5):509-18. Epub 2011 Jun 27 doi: 10.1111/j.1365-2036.2011.04746.x. PMID: 21707680

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