Background: In early phase II trials in advanced pancreatic cancer, gemcitabine demonstrated modest antitumor activity. The investigators in these studies reported that gemcitabine should be studied further in view of the degree and frequency of symptomatic improvement observed, the durability of some of the remissions, and the favorable toxicity profile.
Methods: In order to quantify such symptomatic improvement, a rigorous endpoint of Clinical Benefit was developed that incorporated measures including pain intensity, analgesic consumption and performance status, which have been shown to be reliable and valid endpoints in other studies.
Results: Two trials have been conducted using this methodology in patients with advanced pancreatic carcinoma.
Conclusions: The results of these studies suggest that gemcitabine is the first cytotoxic agent with any meaningful impact on survival and disease-related symptoms in advanced pancreatic adenocarcinoma. The degree of improvement seen is one which patients with cancer often consider to be most important. Further studies will be required to define more fully the role of gemcitabine in the treatment of pancreatic cancer.