Primary biliary cholangitis (PBC), formerly primary biliary cirrhosis – autoimmune cholangiopathy, predominantly in females
Presenting features: pruritus, fatigue, jaundice; few present as advanced liver disease.
Diagnosis: cholestasis (elevated GGT, PAlc, bilirubin), high IgM, positive serology for anti-mitochondrial antibodies (AMA, 90%; or AMA-M2 subtype) or ANA (30%); 10% are AMA negative. Liver biopsy is not necessary for diagnosis in AMA positive pts. but useful for evaluation of activity and staging, and in suspicion of overlap syndrome (disproportionate elevated transaminases, elevated IgG),
Treatment:
- UDCA (ursodeoxycholic acid, ursodiol) 13-15 mg/kg/day. Treatment response is evaluated after 1 year-treatment by Paris criteria (Bilirubin < 1 mg/dl, AST < 2 x ULN, ALP < 3 x ULN) or Barcelona criteria (ALP decreased by 40% or normalisation). Adherence problems may occur because of weight gain, nausea or hair loss.
- PPAR-a agonists – fibrates: regulate bile acids synthesis and protect bile duct epithelium.
- Farnesoid X receptor agonist – obethicolic acid: choleretic, anti-apoptotic, anti-oxidant.
- Immunosuppressive agents: prednisolone, budesonide (not in cirrhosis – can cause portal vein thrombosis)
- norUDCA – side-chain shortened omologue of UDCA
- Rituximab (B cell depletion) – beneficial for fatigue
- Liver transplant – Bilirubin > 6, MELD > 12, Mayo score > 7.8
Follow-up: yearly in those with biochemical response.