The primary endpoint of each study was progression-free survival, defined as the time between the start of the research until evidence of cancer progression or death from any cause.
"This is not the garden variety pancreatic cancer we see commonly. It's a rare tumor but definitely a health care issue," said Dr. Bhoomi Mehrotra, section head of the medical oncology and stem cell transplantation program at North Shore-Long Island Jewish Medical Center in New Hyde Park, N.Y.
"Obviously, this is very encouraging, particularly for patients who are having disease progression," Mehrotra said.
Citing ethical considerations, the everolimus researchers allowed 148 placebo patients whose cancers had spread during the study to switch to the active drug. About 64 percent of everolimus patients experienced some degree of tumor shrinkage from the drug, compared with 21 percent receiving the placebo.
Sunitinib performance was similarly impressive, prompting the researchers to discontinue the study early after noting the radical increase in progression-free survival for those on the active drug.
"There certainly is the potential for patients to be on this [everolimus] for a long time," Yao said, noting that he does not yet know the drug's potential cost. "About 34 percent of patients were alive and progression-free after 18 months, compared to 9 percent of untreated patients. So the curve separated very early."
Side effects, some of them severe, were common with both drugs and included diarrhea, fatigue, anemia and low white blood cell counts.
Noting that the medications might be able to be taken indefinitely if still effective, Mehrotra questioned whether the side effects would outweigh the benefits for patients with either no symptoms or stable cancers.<
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