Gemcitabine or Gemcitabine Plus Oxaliplatin in the First-Line Treatment of Patients With Advanced Transitional Cell Carcinoma of the Urothelium Unfit for Cisplatin-Based Chemotherapy: A Randomized Phase 2 Study of the French Genitourinary Tumor Group (GETUG V01)

Abstract Background The optimal chemotherapy for patients with advanced transitional cell carcinoma of the urothelium who are not eligible for cisplatin remains to be defined. Objective To assess the activity of gemcitabine alone (GEM) or in combination with oxaliplatin (GEMOX) in a randomized phase 2 trial. Design, setting, and participants The primary end point was the objective response rate according to Response Evaluation Criteria in Solid Tumors criteria. The sample size was based on a two-stage Fleming design with p0 = 35% and p1 = 55%. At the end of the first stage designed to register 20 patients on each treatment arm, the observation of seven or more objective responses would have led to the inclusion of 30 more patients in each arm. Results and limitations From July 2004 to March 2009, 44 patients in 10 centers were randomly assigned into the GEM or the GEMOX arm, 22 on each treatment arm. The median age was 76 yr. Seven patients were included for a performance status (PS) of 2 only. The remaining 37 patients had an impaired renal function, 11 of whom also had a PS of 2. The median creatinine clearance was 45 ml/min (range: 30–80 ml/min). The trial was closed after the first part because the GEMOX arm did not reach the targeted objective response rate to proceed further. Conclusions Oxaliplatin does not add any significant activity (in terms of response rates) compared with gemcitabine alone in patients with advanced transitional cell carcinoma of the urothelium who are ineligible for cisplatin.