Researchers at Yale Cancer Center and Yale-New Haven Health's Smilow Cancer Hospital discovered that patients who received a combination of capecitabine and temozolomide had a higher progression-free survival rate than those who received temozolomide alone.

Patients with advanced pancreatic neuroendocrine tumours (NETs) have few treatment options. The findings will be presented on June 5 at the 2022 Annual Meeting of the American Society of Clinical Oncology (ASCO).

"This combination of drugs yielded the longest progression-free survival rates and highest response rates that have been seen in any study of this type for patients with pancreatic NETs," said Pamela Kunz, associate professor of internal medicine (medical oncology) at Yale School of Medicine, director of the Center for Gastrointestinal Cancers at Smilow Cancer Hospital and Yale Cancer Center, and principal investigator of the clinical trial.

The trial was a randomised, phase 2 clinical trial comparing the effects of temozolomide alone with those of temozolomide in combination with capecitabine. The trial, which included 144 patients, showed that the combination of capecitabine and temozolomide produced longer progression-free survival rates, higher response rates, and longer overall survival rates than temozolomide alone.

In addition, deficiency of the DNA repair enzyme methylguanine methyltransferase (MGMT) in the tumour tissue, was found to be associated with increased responses.

"This clinical trial is practice-changing and the combination of capecitabine and temozolomide should be included as a standard treatment option for patients with advanced pancreatic NETs," said Kunz.

"In addition, MGMT testing can be considered for select patients receiving temozolomide for whom the response is a primary goal of treatment. However, testing is not recommended for routine use as confirmatory studies are needed."

The findings are part of the ECOG-ACRIN E2211 trial, conducted through the National Cancer Institute and the National Clinical Trial Network.