番茄社区

WASHINGTON -- A widely used medication for primary biliary cholangitis (PBC) is beneficial even when it does not produce a biochemical response, a researcher reported here.

In a large cohort of PBC patients, treatment with ursodeoxycholic acid (UDCA) was associated with a significant increase in survival without a liver transplant compared with no treatment, according to Adriaan van der Meer, MD, PhD, of Erasmus University Medical Center in Rotterdam, the Netherlands.

But surprisingly, there was a significant benefit even when -- on the basis of biochemical markers -- the patient was classified as having no response or an inadequate response, van der Meer reported at the Liver Meeting, the annual conference of the American Association for the Study of Liver Diseases.

Cumulative transplant-free survival for the so-called non-responders was less than what was seen for those who had a biochemical response, but still significantly greater than for patients not treated, van der Meer told 番茄社区, adding that "There is no such thing as non-response."

Patients treated with UDCA will have a lower risk of transplant and a greater chance of living longer, so doctors should "start patients on urso and never stop" until clinical trials show that they will do better on another drug, he noted.

While the drug sometimes has side effects that can be unpleasant, van Der Meer said it is safe and inexpensive. "It's something that you should consider to continue, even if there are side effects, but this is up to the individual patient's decision."

But Pierre Gholam, MD, of University Hospitals in Cleveland, was skeptical, telling 番茄社区 the results are "very different from established dogma."

Gholam, who was not involved in the study, said most clinicians today begin by putting PBC patients on UCDA and then switching them to a second-line therapy, obeticholic acid (Ocaliva), if they don't have a biochemical response.

The idea is that the second-line therapy can cut their risk of transplant or death by improving their response, essentially putting them on a par with those who responded in the first place, he said. It's "not news" that successful treatment with UDCA cuts the risk of transplant, he added.

Van der Meer's group "suggest you should just continue the urso," he said. "So where does that leave second-line therapy?"

The study was a retrospective analysis of 3,902 PBC patients from 15 large hepatology centers in the U.S. and Europe, conducted by the , an international PBC research collaboration.

The patients were followed for a median of 7.8 years, with a view to the association between UDCA treatment and a composite endpoint of liver transplant or death from any cause.

Most patients (90.4%) were given UDCA and after a year of treatment, and 29.1% did not meet the so-called GLOBE criteria for response, which combines age, bilirubin, albumin, alkaline phosphatase, and platelet counts in a risk assessment score.

Analysis showed that, overall, patients treated with UDCA had a hazard ratio of 0.46 for cumulative risk of liver transplant, compared with those who were never treated, the investigators reported.

The benefit was significant for all baseline characteristics, including such things as age, sex, and biochemical markers.

But when the researchers analyzed the treated patients by their GLOBE response, they still found a benefit for those who had not responded: an HR of 0.56 that remained significantly better than the risk faced by those who weren't treated, although it was lower than what was seen among those who had responded.

Comment