Clinical outcomes of patients with gastrointestinal malignancies participating in phase I clinical trials.

Abstract

428 Background: Early phase clinical trials play a pivotal role in drug development. However, limited data is available on the clinical outcomes of patients with gastrointestinal (GI) malignancies enrolled in phase 1 clinical trials. The aim of this study is to evaluate the clinical characteristics and survival of patients with GI cancers participating in phase 1 clinical trials. Methods: All consecutive patients with advanced GI tumors who participated in phase 1 clinical trials from 1/2007 to 12/2013 at Moffitt Cancer Center were included. Data for individual patients were extracted from the OnCore database and clinical charts. Only patients who received at least one dose of the study drug were included. Cox regression model was used to estimate multivariable adjusted hazard ratios and 95% CI. Results: 243 pts with GI malignancies who participated in phase 1 clinical trials were included. Median age was 62 years (26-82 years) with 55% of the patients aged >60 years. Patients had the following disease types: pancreas (42%), colorectal (34%), gastro-esophageal (10%), hepatobiliary (13%) and others (2%). The distribution of patients for baseline variables are: male (55%), DVT/PE (12%), ECOG ≥1 (72%), prior systemic therapies ≥2 (59%), no. of metastatic sites > 2 (31%), lung metastases (40%) and liver metastases (77%). Pts received treatment with chemotherapy only (14%), targeted therapy (41%), chemotherapy + targeted therapy (42%) and others (2%). The response rate was 4% with 38% achieving stable disease; 42% of pts had progressive disease. The median OS was 5.8 months (0.2-52.4). ECOG score of 0, prior systemic therapies < 2 and absence of liver metastases were associated with better OS on multivariate analysis. Conclusions: This is the largest study to assess clinical outcomes in this patient population. Phase 1 clinical trials provide clinical benefit to patients with advanced GI malignancies and should be recommended as a treatment option in appropriate patients. [Table: see text]