title: Patient-reported outcomes as a component of the primary endpoint in a double-blind, placebo-controlled trial in advanced pancreatic cancer.
abstract: 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 toxicity 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.
authors: Developmental Therapeutics and GI Malignancies, University of Colorado Health Sciences Center, Aurora, Colorado 80045, USA. Gail.Eckhardt@UCHSC.edu
authors: S Gail
authors: SG
authors: Eckhardt
authors: Peter
authors: P
authors: De Porre
authors: David
authors: D
authors: Smith
authors: Joan
authors: J
authors: Maurel
authors: William P
authors: WP
authors: Steward
authors: Olivier
authors: O
authors: Bouche
authors: Helgi
authors: H
authors: van de Velde
authors: Bart
authors: B
authors: Michiels
authors: Roland
authors: R
authors: Bugat
mesh_terms: D000230:Adenocarcinoma / Q000188:drug therapy* / Q000401:mortality*; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage*; D015897:Comorbidity; D003841:Deoxycytidine / Q000008:administration & dosage / Q000031:analogs & derivatives; D004311:Double-Blind Method; D005060:Europe / Q000453:epidemiology; D005260:Female; D006801:Humans; D015994:Incidence; D008297:Male; D008875:Middle Aged; D017063:Outcome Assessment, Health Care; D010146:Pain / Q000401:mortality* / Q000517:prevention & control*; D010147:Pain Measurement / Q000187:drug effects; D010190:Pancreatic Neoplasms / Q000188:drug therapy* / Q000401:mortality*; D017060:Patient Satisfaction / Q000706:statistics & numerical data*; D015990:Placebo Effect; D015363:Quinolones / Q000008:administration & dosage; D018570:Risk Assessment; D012307:Risk Factors; D016019:Survival Analysis; D015996:Survival Rate; D016896:Treatment Outcome; D014481:United States / Q000453:epidemiology; D000093542:Gemcitabine
chemical_list: D000970:Antineoplastic Agents; D015363:Quinolones; D003841:Deoxycytidine; C402769:tipifarnib; D000093542:Gemcitabine