Baseline and change from baseline in tumor burden compared with patient outcomes
e17530 Background: The impact of baseline tumor burden (bTB) and percentage reduction in tumor burden (dTB) on patient outcome has not been systematically studied in oncology clinical trials. We assess the relationship between both bTB and dTB and objective response (OR), progression free and overall survival (PFS and OS) in bevacizumab (B) and trastuzumab (T) trials. Methods: We analyzed 3 B and 1 T Ph 3 mBC and mCRC trials of > 1800 patients total. We used correlations and Cox models for the relationships between pairs of variables and multivariate Cox models to evaluate TB and PFS as independent predictors of OS. Here, we present the minimum dTB value until progression. Results: bTB is not related to dTB, OR rate or PFS time; it is negatively related to OS. dTB shows a strong positive relationship with PFS and OS. TB and PFS are independent predictors of OS, with PFS the better predictor. Results at earlier dTB assessments and within treatment arms are similar. Conclusions: Patients with high bTB are equally likely to respond to treatment and to have prolonged PFS as those with lower bTB, but high bTB is a negative predictor for OS. In contrast, greater percentage reductions in TB are strongly associated with better PFS and OS outcomes. Tumor burden and PFS are independent predictors of OS. Owing to these relationships, assessing bTB and dTB, as well as OR, PFS and OS, may improve the interpretation of phase II data. [Table: see text] [Table: see text]