Diagnosis and treatment of primary biliary cholangitis

TY - JOUR

T1 - Diagnosis and treatment of primary biliary cholangitis

AU - Laschtowitz, Alena

AU - de Veer, Rozanne C

AU - Van der Meer, Adriaan J

AU - Schramm, Christoph

PY - 2020/7

Y1 - 2020/7

N2 - Primary biliary cholangitis is a cholestatic, chronic autoimmune liver disease with a wide individual variation in disease progression. The diagnosis is predominantly based on chronic elevation of alkaline phosphatase and the presence of anti-mitochondrial antibodies or other specific antinuclear antibodies (i.e. anti-gp210 and anti-sp100). Even in early-stage disease, health-related quality of life can be severely impaired by symptoms such as pruritus, fatigue, and sicca syndrome and metabolic bone disease should be assessed and treated. The prognosis of the disease is, however, largely determined by the development of cirrhosis and its complications. Ursodeoxycholic acid is associated with an improved prognosis and should be initiated and continued in all patients. Clinical outcome is related to the biochemical response to ursodeoxycholic acid, but the prognosis of those with an incomplete response is still better than those who remain untreated. Obeticholic acid was recently approved as second-line treatment and bezafibrate may serve as an adequate off-label alternative, particularly in patients with pruritus. Preliminary data suggest an additive effect of triple therapy with ursodeoxycholic acid, obeticholic acid, and bezafibrate, whereas other promising drugs are being evaluated in clinical trials.

AB - Primary biliary cholangitis is a cholestatic, chronic autoimmune liver disease with a wide individual variation in disease progression. The diagnosis is predominantly based on chronic elevation of alkaline phosphatase and the presence of anti-mitochondrial antibodies or other specific antinuclear antibodies (i.e. anti-gp210 and anti-sp100). Even in early-stage disease, health-related quality of life can be severely impaired by symptoms such as pruritus, fatigue, and sicca syndrome and metabolic bone disease should be assessed and treated. The prognosis of the disease is, however, largely determined by the development of cirrhosis and its complications. Ursodeoxycholic acid is associated with an improved prognosis and should be initiated and continued in all patients. Clinical outcome is related to the biochemical response to ursodeoxycholic acid, but the prognosis of those with an incomplete response is still better than those who remain untreated. Obeticholic acid was recently approved as second-line treatment and bezafibrate may serve as an adequate off-label alternative, particularly in patients with pruritus. Preliminary data suggest an additive effect of triple therapy with ursodeoxycholic acid, obeticholic acid, and bezafibrate, whereas other promising drugs are being evaluated in clinical trials.

KW - Autoantigens/immunology

KW - Autoimmune Diseases/diagnosis

KW - Bezafibrate/therapeutic use

KW - Biomarkers/blood

KW - Biopsy

KW - Chenodeoxycholic Acid/analogs & derivatives

KW - Cholagogues and Choleretics/therapeutic use

KW - Disease Progression

KW - Drug Therapy, Combination/methods

KW - Elasticity Imaging Techniques

KW - End Stage Liver Disease/diagnosis

KW - Fatigue/diagnosis

KW - Female

KW - Humans

KW - Immunoglobulin M/blood

KW - Liver Cirrhosis, Biliary/diagnosis

KW - Liver Function Tests

KW - Liver Transplantation

KW - Liver/diagnostic imaging

KW - Middle Aged

KW - Off-Label Use

KW - Prognosis

KW - Pruritus/diagnosis

KW - Quality of Life

KW - Severity of Illness Index

KW - Sjogren's Syndrome/diagnosis

KW - Survival Rate

KW - Treatment Outcome

KW - Ursodeoxycholic Acid/therapeutic use

U2 - 10.1177/2050640620919585

DO - 10.1177/2050640620919585

M3 - SCORING: Review article

C2 - 32299307

VL - 8

SP - 667

EP - 674

JO - UNITED EUR GASTROENT

JF - UNITED EUR GASTROENT

SN - 2050-6406

IS - 6

ER -