Bile duct cancer, also known as cholangiocarcinoma, is a rare and difficult to treat malignancy affecting the part of the digestive system that connects the liver, gallbladder, and small intestine. Approximately 2,300 people are diagnosed with bile duct cancer in the United Kingdom each year, and until recently there had been very little progress in improving outcomes following surgery. A landmark clinical trial has now produced the first clear evidence that adjuvant chemotherapy with capecitabine significantly improves survival in patients who have undergone surgical resection for bile duct cancer.
What the BILCAP Trial Found About Bile Duct Cancer Survival
The BILCAP trial, funded by Cancer Research UK and led by the University of Southampton, is one of the first randomized trials to evaluate post surgical treatment options for bile duct cancer. The trial enrolled approximately 450 patients, with half receiving capecitabine for six months following surgery and the other half undergoing surgery alone without additional treatment.
The results, presented at the 2017 ASCO Annual Meeting in Chicago, were striking. Three year survival improved by almost a quarter, representing a 23 percent increase, in patients who received capecitabine compared to those who had surgery only. Average survival in the capecitabine group increased to 53 months compared to 36 months in the surgery alone group, a difference of 17 months. The researchers concluded that capecitabine should become standard of care for all bile duct cancer patients following surgery.
Why Clinical Trials for Bile Duct Cancer Are So Rare
One of the most significant barriers to progress in bile duct cancer research has been the difficulty of recruiting enough patients to conduct adequately powered clinical trials. Because the condition is rare, assembling a large enough cohort to generate statistically meaningful results requires international collaboration and a sustained multi center effort over many years. The BILCAP trial represents exactly this kind of coordinated investment, and its results demonstrate what becomes possible when the research community organizes around rare cancers with the same rigor applied to more common malignancies.
Professor Peter Johnson, Cancer Research UK’s chief clinician and Professor of Medical Oncology at the University of Southampton, noted that rare cancers have historically been a difficult problem but that large, internationally coordinated trials are beginning to change the picture. Accelerating progress in these areas will depend on both better laboratory science to understand the cellular drivers of rare cancers and well designed trials to translate that knowledge into clinical benefit.
A Patient Perspective on the Bile Duct Cancer Trial
Nikki Archer, 42, from Exeter, was first diagnosed with bile duct cancer in 2008. Following surgery, she enrolled in the BILCAP trial and received capecitabine for six months. She described feeling privileged and lucky to have had the opportunity to participate. Her treating team accommodated a break in treatment to allow her to get married. Since completing treatment, she has had a second child and no longer requires follow up care. Her experience illustrates both the human stakes of bile duct cancer research and the life changing potential of well designed clinical trials for patients who would otherwise have had no adjuvant treatment options.
What This Means for Bile Duct Cancer Treatment Going Forward
Lead researcher Professor John Primrose of the University of Southampton called for capecitabine to be offered to all bile duct cancer patients following surgery, citing the clear survival benefit and the favorable side effect profile observed in the trial. The drug is already widely used in other gastrointestinal cancers, which means its safety and tolerability profile in clinical practice is well established.
The BILCAP results represent a meaningful step forward for a patient population that had seen very little therapeutic progress. For patients and families facing a bile duct cancer diagnosis, clinical trial participation remains one of the most important pathways to accessing emerging treatments and contributing to the evidence base that will continue to improve outcomes.
To read more about oncology research, visit the FOMAT blog. FOMAT conducts oncology clinical trials at sites across the United States. To learn more about active studies, visit FOMAT’s patient studies page.
For the full source, see the original article at DDDmag.com.