Abstract 2669: Early-phase trials in patients with advanced gallbladder cancer and cholangiocarcinoma: The MD Anderson Clinical Center for Targeted Therapy experience

Abstract

Abstract BACKGROUND: Patients with cholangiocarcinoma (CC) and gallbladder carcinoma (GC) have few effective therapeutic options in the relapsed and/or metastatic setting. With that purpose, we analyzed the characteristics and outcomes of patients with advanced CC and GC treated on early-phase clinical trials in the Clinical Center for Targeted Therapy at MD Anderson Cancer Center. METHODS: We searched an electronic database of consecutive pts with GC and CC referred to the Clinical Center for Targeted Therapy starting in November 2004. We assessed the relationship between types of clinical trial and pts’ tumor types, progression-free survival, and mutations. RESULTS: Of the 72 patients with CC and GC referred to the clinic, 32 (44%) were not enrolled in a clinical trials mainly due to deterioration of performance status (n=25), decision to pursue alternate therapies (n=6), and insurance denial (n=1). Forty pts (5 w GC; 35 w CC) who received treatment on early clinical trials are included in this analysis. The median age was 60.2 yrs (range, 41.4-73.6 yrs). ECOG performance status (PS) was 0-1 in 36 pts (90%) and 2 in 4 (10%). Median number of prior therapies was 3 (range 0-17). Six pts were not restaged due to early disease progression. Of the 40 pts evaluable for response, 8 (20%) had stable disease (SD) > 6 months and 3 (8%) had a partial response (PR) (total SD > 6 mos/PR = 28%). Highest rates of SD > 6 mos/PR were observed in patients treated with protocols that included hepatic arterial infusion drug administration (7/17 = 41%) or anti-angiogenic agents (9/24 = 38%) (with 6 of these pts receiving treatment that included hepatic arterial infusion chemotherapy combined with a systemic anti-angiogenic agent). SD lasting 9 months was also seen in 1 pt treated with a MEK inhibitor. Median progression-free survival for the 40 pts was 2.0 months 9 (95% CI 1.7, 2.8) on early-phase clinical trials versus 3.0 months on their last FDA-approved treatment before Phase I referral (95% CI 2.3, 3.7). CONCLUSIONS: About one-third of patients with CC and GC referred for early-phase trials cannot be enrolled because of poor performance status. Twenty-eight percent of patients who met eligibility criteria achieved SD > 6 mos/PR, with responses predominantly observed in regimens that contained locoregional treatment, anti-angiogenic agents, or a targeted MEK inhibitor. Citation Format: {Authors}. {Abstract title} [abstract]. In: Proceedings of the 103rd Annual Meeting of the American Association for Cancer Research; 2012 Mar 31-Apr 4; Chicago, IL. Philadelphia (PA): AACR; Cancer Res 2012;72(8 Suppl):Abstract nr 2669. doi:1538-7445.AM2012-2669