ASCO 2009: First Standard of Care for Biliary Tract Cancer

Zosia Chustecka

May 19, 2009

May 19, 2009 — For the first time, a standard of care for biliary tract cancer has been proposed: chemotherapy with a combination of gemcitabine and cisplatin. The recommendation is based on results showing a survival benefit from the largest study to date in patients with this rare but difficult-to-treat cancer.

The results will be presented at the upcoming 2009 Annual Meeting of the American Society of Clinical Oncologists (ASCO), but a few details were previewed in an ASCO presscast on May 14.

Biliary tract cancer is a collective term, covering both the gall bladder and bile duct, and it is relatively rare, explained lead author Juan Valle, MD, medical oncologist at the University of Manchester in the United Kingdom. It affects about 1 to 2 people per 100,000, although the incidence is 10-fold higher in the Far East.

Surgery is the only chance for a long-term cure, but only about 5% to 10% of patients survive after 5 years, he said.

For patients with inoperable biliary tract cancer, chemotherapy based on 5-fluorouracil and gemcitabine has been used because these are the drugs that are used for pancreatic cancer. However, to date there has been no effective treatment for advanced cases.

Now a survival benefit has been shown for the first time. The results come from the UK ABC-02 trial, which involved 410 patients with inoperable, advanced, or metastatic biliary tract cancer. He noted that this population includes 86 patients from the earlier UK ABC-01 trial, the results of which were presented at the ASCO Gastrointestinal Cancers Symposium in 2006.

Benefit in Survival Without Added Toxicity

Patients were randomized to receive either the combination of gemcitabine plus cisplatin or gemcitabine alone. Initial treatment was continued for 3 months and, in the absence of disease progression, was repeated for another 3 months.

After a median follow-up of 6.1 months, there were 122 deaths (59.8%) in the combination group and 141 (68.5%) deaths in the gemcitabine-alone group.

The median survival with gemcitabine plus cisplatin was 11.7 months, compared with 8.3 months with gemcitabine alone (P = .002). The hazard ratio was 0.70 (95% confidence interval, 0.54 - 0.89), so the combination reduces the risk for death by 30%, Dr. Valle reported.

Progression-free survival was also reduced by 30%, and scans showed that there was improved tumor control in the group treated with the combination, he said.

The benefit was gained with no clinically significant added toxicity, Dr. Valle noted. The most common adverse effect was moderate neutropenia, seen in 22.6% on the combination and 17.9% on gemcitabine alone, but this was "mostly asymptomatic," he said.

"This is the first demonstration of a survival benefit in biliary tract cancer, so we now consider the combination of gemcitabine plus cisplatin to be a worldwide standard of care," Dr. Valle concluded.

It is comforting to know that we have such a clear standard of care.

Current ASCO president Richard Schilsky, MD, professor of medicine at Chicago University in Illinois, said this was a "definitive and well-conducted study in a difficult-to-treat cancer." He added that, as a physician who treats patients with biliary tract cancers, "it is comforting to know that we have such a clear standard of care that we can offer them."

Dr. Schilsky also asked about plans for future trials in this area. Dr. Valle said that there is probably little to gain in further benefit from adding on more chemotherapy, which would probably increase toxicity, so the next step is to consider adding a targeted therapy, he said.

The ABC-02 trial was funded by Cancer Research UK. The researchers did not report any conflicts of interest. Dr. Schilsky has no relevant financial relationships to disclose since taking office in June 2007, but continues to serve (with compensation) on a Data Monitoring Committee for Novartis Oncology.

2009 Annual Meeting of the American Society of Clinical Oncologists (ASCO): Abstract 4503. To be presented June 1, 2009.


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