A 48-year-old woman with primary biliary cholangitis (PBC) and pruritus fails to respond to ursodeoxycholic acid (UDCA) after 1 year (inadequate biochemical response by Paris criteria). What is the MOST appropriate add-on therapy per current guidelines?
- A Colestyramine for pruritus management
- B Methotrexate immunosuppression
- C Liver transplantation immediately
- D Obeticholic acid (OCA) ✓
Explanation
For PBC patients with an inadequate biochemical response to UDCA (defined by Paris-II criteria: ALP > 1.5× ULN or AST > 1.5× ULN or bilirubin > 1× ULN after 1 year of UDCA), obeticholic acid (OCA—a farnesoid X receptor agonist) is the approved second-line agent (POISE trial) and significantly reduces ALP and bilirubin. Bezafibrate (off-label) is an alternative with emerging evidence. Colestyramine addresses pruritus symptomatically but does not improve disease progression. Liver transplantation is reserved for end-stage PBC or refractory pruritus after all medical options are exhausted.
Reference: Harrison's Principles of Internal Medicine, 21st ed.
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Written and medically reviewed by the StethoPrep medical team.