Patient-Reported Outcomes as a Component of the Primary Endpoint in a Double-Blind, Placebo-Controlled Trial in Advanced Pancreatic Cancer

In this randomized, double-blind, placebo-controlled study comparing gemcitabine + tipifarnib (G + t) or gemcitabine + placebo (G + p) in patients with pancreatic cancer, the primary endpoint, of time to deterioration (TTD) was based primarily on patient-reported outcomes. Deterioration was defined as death or worsening of disease-related symptoms, based on patient-reported Outcomes of pain intensity and analgesic use in a daily diary, plus investigator-rated weekly performance status. Secondary endpoints included survival and safety. Two hundred and forty-four patients were treated for a total of 4780 weeks, during which the diary was completed daily. Overall, the completion of the diary was found to be feasible: patients completed approximately 95% of scheduled diary entries. Baseline characteristics were well balanced between the two treatment arms. The primary endpoint of TTD was not significantly different between the G + t arm (69 days) and the G + p arm (91 days, P = 0.40). Survival was not significantly different between the G + t arm (202 days) and the G + p arm (221 days, P = 0.66). The combination of G + t had an acceptable profile, with primarily neutropenia and thrombocytopenia. Methodologically, measurement of Patient-reported Outcomes is feasible and useful in assessing the effect of anti-cancer therapy in pancreatic cancer if comprehensive initial and. ongoing training is provided, to all people involved, including not only the patients but also the study personnel. J Pain Symptom Manage 2009;37:135-143. (c) 2009 U.S. Cancer Pain Relief Committee. Published by Elsevier Inc. All rights reserved.