Randomized Trial of Adjuvant Thalidomide Versus Observation in Patients With Completely Resected High-Risk Renal Cell Carcinoma

Objectives: To evaluate the effect of adjuvant thalidomide on recurrence-free survival (RFS) after nephrectomy for high-risk metastatic renal cell carcinoma (RCC). Methods: Eligibility criteria for enrollment on this randomized trial included any histologic subtype, T2 (high grade, any N), T3/T4 (any grade, any N), or node-positive (any grade, any T) RCC. We randomized eligible patients to observation or to receive thalidomide 300 mg daily for 24 months. Patients were observed until disease recurrence or death. Results: After we enrolled 46 patients, we stopped the trial at a median follow up of 43.9 months (range, 9.7-74.2 months). Patients on the thalidomide arm had inferior 2- and 3-year probabilities of RFS, compared with controls (47.8% vs 69.3% and 28.7% vs 69.3%, respectively; P = .022). The 2- and 3-year cancer-specific survival was similar for both groups. All observed deaths were attributable to RCC (P = .392). By multivariate analysis, tumor size and grade predicted recurrence (P = .001 and .013) and kidney cancer-specific death (P = .002 and .014). Thalidomide treatment, however, was not an independent predictor of recurrence or cancer-specific mortality. Conclusions: In this small, randomized, controlled trial, adjuvant thalidomide therapy after complete resection of high-risk RCC did not improve the 2- and 3-year RFS rates or cancer-specific death rates.