LAS VEGAS – Evidence is mounting for several adjuvant treatments that may be appropriate for patients with primary biliary cholangitis who are not responding to first-line ursodeoxycholic acid (UDCA), according to Cynthia Levy, MD.
Given these new potential treatment options, it’s important for clinicians to assess biochemical response to first-line UDCA, said Dr. Levy, assistant director for the Schiff Center for Liver Diseases at the University of Miami.
“Up until recently, we didn’t have anything to offer to nonresponders,” Dr. Levy said at the inaugural Perspectives in Digestive Diseases meeting held by Global Academy for Medical Education. “Now we know at 1 year, we need to restratify to evaluate the need for adjuvant therapy.”
Many UDCA-treated patients will not respond to that first-line treatment, putting them at risk for progression to hepatocellular carcinoma and end-stage liver disease, according to Dr. Levy.
Obeticholic acid, a farnesoid X receptor agonist, is now a Food and Drug Administration–approved option for these patients, while fibrates and budesonide have recent data supporting their use and are available off-label, she added.
The conditional FDA approval for obeticholic acid, granted in May 2016, is for treatment as monotherapy in patients who do not tolerate UDCA or in combination with UDCA for patients who had had incomplete responses to that treatment for at least a year. Improvement in survival without liver transplantation has not yet been demonstrated for this agent, though studies are ongoing, Dr. Levy noted.
In the meantime, research is progressing with the peroxisome proliferator-activated receptor alpha (PPAR-alpha) agonists fenofibrate and bezafibrate.