Gastroenterology· Topic 5.10 Cholestatic, inherited, and other liver disease· 5.10.3

Primary biliary cholangitis

Primary biliary cholangitis is an autoimmune, T cell-mediated destruction of small and medium intrahepatic bile ducts that presents with fatigue and pruritus in middle-aged women and a cholestatic liver chemistry pattern. Diagnosis requires two of three criteria (elevated alkaline phosphatase, positive antimitochondrial antibody, compatible biopsy), and lifelong ursodeoxycholic acid is first-line treatment, with obeticholic acid or fibrates added for inadequate biochemical response.

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What this article covers

  • Overview

    Definition: primary biliary cholangitis (PBC), previously called primary biliary cirrhosis, is an autoimmune disease in which T cell-mediated injury destroys the small and medium intrahepatic bile ducts, producing chronic cholestasis.

  • Epidemiology

    Sex predominance: women account for 90 to 95% of cases, a female-to-male ratio of about 9 to 1.

  • Risk factors

    Female sex: the dominant demographic risk factor.

  • Pathophysiology

    T cell-mediated bile duct injury: autoreactive T cells target the small and medium intrahepatic bile ducts, producing a nonsuppurative destructive cholangitis (the florid duct lesion), sometimes with granulomas.

  • Clinical features

    Often asymptomatic: more than half of patients are diagnosed from an incidentally elevated alkaline phosphatase before any symptoms appear.

  • Differential diagnosis

    Primary sclerosing cholangitis: more common in men, strongly associated with inflammatory bowel disease (especially ulcerative colitis), and diagnosed by cholangiography (MRCP or ERCP) showing…

  • Diagnosis

    Establish the diagnosis with two of three criteria, and biopsy is not required when the first two are met: (1) unexplained alkaline phosphatase at least 1.5 times the upper limit of normal, (2) AMA…

  • Management

    Start ursodeoxycholic acid 13 to 15 mg/kg/day PO, in one or two divided doses, lifelong, as soon as the diagnosis is confirmed, because earlier initiation gives better histologic and survival outcomes.

  • Complications

    Cirrhosis and decompensated liver disease: the end result of unchecked ductopenia and fibrosis in nonresponders.

  • Prognosis

    Response to ursodeoxycholic acid is the key determinant: patients with normal baseline bilirubin and albumin who achieve an adequate biochemical response (ALP below 1.67 times the upper limit of…

  • Prevention

    No primary prevention exists, so downstream prevention is early diagnosis and prompt ursodeoxycholic acid initiation to prevent progression to cirrhosis and transplant.

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