Study indicates which PBC patients may see liver function recovery
Type of complication found to predict damage reversal
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Primary biliary cholangitis (PBC) patients who have irreversible liver scarring that affects the organ’s function are more likely to experience liver function recovery if the first major manifestation of severe liver damage is a bleeding complication known as variceal hemorrhage.
That’s according to a study in China that also showed that patients who experienced severe liver dysfunction while on ursodeoxycholic acid (UDCA) — PBC’s first-line treatment, sold as Urso and Actigall in the U.S. — were less likely to have liver function recovery than those who experienced it before UDCA treatment.
Patients who experienced liver function recovery saw long-term outcomes that were generally similar to those of patients who maintained consistently adequate liver function.
The study, “Factors associated with recompensation and criteria for etiological suppression in patients with primary biliary cholangitis,” was published in the Journal of Gastroenterology.
PBC is an autoimmune disease marked by inflammation in the bile ducts, a series of tubes that carry the digestive fluid bile from the liver to the intestines. This inflammation can lead to scarring (fibrosis) in the liver, which can ultimately result in irreversible liver fibrosis (cirrhosis).
Complications of cirrhosis
Cirrhosis can be broadly classified as either compensated, in which the liver can still perform its functions despite scarring, or decompensated, when the liver no longer works as it should. This can lead to noticeable complications, including bleeding in the gastrointestinal tract due to rupture of swollen veins (variceal hemorrhage), neurological problems resulting from liver toxicity (hepatic encephalopathy), and liver failure.
Some PBC patients with decompensated cirrhosis can achieve recompensation, in which the liver’s ability to perform its essential functions improves and decompensation manifestations are resolved.
However, it’s unclear which patients are most likely to experience this phenomenon. To learn more, scientists in China analyzed data from 240 people with PBC-related cirrhosis who were seen at their center from January 2014 to June 2023. A total of 122 participants had compensated cirrhosis, and the remaining 118 had decompensated cirrhosis.
In the decompensated group, the most common manifestation of decompensated cirrhosis was ascites and/or hepatic encephalopathy (51.7%), followed by variceal hemorrhage with ascites and/or hepatic encephalopathy (33.9%), and variceal hemorrhage alone (14.4%).
Eighteen patients with decompensated cirrhosis (15.3%) experienced recompensation after a median follow-up of 50 months (about four years), defined using standard criteria that reflects stable liver function improvement without the need for certain medications to manage complications of liver decompensation.
Using a slightly less stringent definition — stable liver function improvements while allowing for low doses of certain medications — 31 patients (26.3%) experienced recompensation.
Those whose initial manifestation of decompensated cirrhosis was variceal hemorrhage alone were much more likely to experience recompensation. In fact, more than half (52.9%) of patients with this manifestation experienced recompensation, compared with fewer than one in 10 patients in the other two groups of initial manifestations.
“Our study also demonstrated that the potential for recompensation in PBC was strongly influenced by the type of index decompensation,” the researchers wrote. “We found that [variceal hemorrhage] as the presentation associated with the highest likelihood of … recompensation.”
A mainstay treatment for PBC is UDCA, which promotes bile flow. Among the participants with decompensated cirrhosis, roughly half experienced decompensation before starting UDCA.
These patients were significantly more likely to experience recompensation than those who were already taking UDCA when they experienced decompensation (23.2% vs. 8.1%). The researchers said this finding “is not unexpected, as the occurrence of decompensation despite ongoing UDCA therapy suggests an inherently poor biochemical response.”
Response to UDCA treatment, as assessed by standard clinical measures such as the Paris II and Momah/Lindor criteria, was a predictor of recompensation and other long-term outcomes, highlighting these measures as potentially useful tools to guide care decisions.
Among the 18 participants who experienced recompensation, one underwent liver transplant, and none died during follow-up. These outcomes were similar to those of patients with compensated cirrhosis. By contrast, more than 40% of participants with decompensated cirrhosis who did not experience recompensation either died or underwent transplant.
“Compensation in PBC was … translated into tangible survival benefits,” the researchers wrote. “In our [study population], patients who achieved recompensation showed significantly better liver transplant-free survival than those who remained [in] decompensation, with outcomes comparable to compensation individuals.”
Transplant-free survival refers to the time a person lives without the need for a liver transplant.
The scientists stressed that the study was limited to a relatively small group of patients at a single center, and called for further work to validate the findings.
