Low objective response and high toxicity to single-agent mitotane in patients with metastatic adrenocortical carcinoma (ACC): A 25 year experience at MSKCC.

Abstract

4105 Background: ACC is a rare but devastating disease, with short life expectancy in the setting of metastatic, unresectable disease. Mitotane, an oral adrenocorticolytic agent, is often used as a systemic treatment though its therapeutic window is narrow. We retrospectively assessed outcomes of patients (pts) with metastatic ACC who received single-agent mitotane therapy to determine treatment toxicities and effectiveness. Methods: In this retrospective IRB-approved study, we identified 35 pts with metastatic ACC treated with single-agent mitotane at MSKCC from 3/15/1989-9/18/2014. All pts had follow-up at MSKCC and all imaging was reviewed according to RECIST 1.1 by reference radiologists. We reviewed pt demographics, clinical symptoms, toxicity (based on CTCAE Version 4), and treatment outcomes. Results: 35 pts were identified with mean age of 53 years (range, 23-87), 60% female. 18/35 tumors (51%) were functional: estradiol (n = 1), cortisol (n = 12), testosterone (n = 3), or both testosterone and co...