Three-factor tool may predict response to UDCA in PBC: Study
ID'ing patients less likely to respond could allow faster changes in treatment
A predictive tool that incorporates several blood biomarkers may help to identify primary biliary cholangitis (PBC) patients who are less likely to respond to treatment with ursodeoxycholic acid, known as UDCA, during the early stages of the disease, a study has found.
The data showed that three biomarkers — high total cholesterol, alkaline phosphatase, which is a marker of damage to the liver, and the neutrophil-to-lymphocyte ratio, a marker of inflammation — were independent risk factors for a poor response to UDCA, which was linked to more complications and shorter survival.
The study, “Development and external validation of an early prediction model to identify irresponsive patients and prognosis of UDCA treatment in primary biliary cholangitis,” was published in the journal Scientific Reports by a team of researchers in China.
“Predicting … response to UDCA allows for timely adjustments to the treatment plan, which can improve patient prognosis,” or the likely course of the disease, the researchers wrote.
UDCA is standard first-line treatment for PBC
PBC is an autoimmune disease that causes cholangitis, or inflammation that damages the bile ducts that empty digestive bile into the liver. As a result, bile acids accumulate in the liver and leak into the bloodstream, leading to itching and a range of other disease symptoms.
UDCA, marketed in the U.S. as Actigall among others, is the standard first-line treatment for PBC. However, as many as 20% to 40% of patients have an inadequate response to the therapy and “remain at risk of disease progression.”
There is an urgent clinical need for … a tool to guide clinical decision-making.
According to the researchers, having a tool that can distinguish early on those patients more prone to have an inadequate response to UDCA could help doctors choose other treatments that are more likely to succeed.
“There is an urgent clinical need for such a tool to guide clinical decision-making,” the researchers wrote.
To develop a predictive tool for response to UDCA, the team of researchers drew on data from 257 people with PBC, who had a mean age of 56. The vast majority — 223, or 87% — were women, and nearly a third (32%) had cirrhosis, or permanent scarring of the liver.
Among these patients, one-third, or 87 in total, did not respond to UDCA. Compared with those who responded to treatment within a year of follow-up, these individuals had more complications, including swollen veins in the esophagus or stomach (35.6% vs. 14.7%), bleeding (16.1% vs. 5.3%), and fluid buildup in the abdomen (32.2% vs. 15.3%). They also had lower survival rates (7.77 vs. 8.93 years).
When the researchers compared blood data from patients who responded to UDCA with those who did not, they identified total cholesterol, alkaline phosphatase, and neutrophil-to-lymphocyte ratio at the time of diagnosis as independent risk factors for poor response to the treatment.
Used together, these three risk factors had “good discriminative ability,” the researchers wrote. When tested in a separate group of 71 patients, the tool was even more accurate at predicting patient prognosis, the study noted.
According to the team, this predictive tool could help healthcare providers identify patients at risk of not responding to UDCA treatment at an early stage — before starting therapy. By recognizing these high-risk individuals, clinicians could closely monitor their condition and intervene with alternative treatment strategies in a timely manner, if needed. That would ultimately improve overall patient outcomes, the researchers noted.
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