Abstract A44: No tumor or very small tumor burden in on-treatment biopsies is significantly associated with response to Pembrolizumab

Background: In phase I clinical trials, baseline and on-treatment biopsies are increasingly performed to investigate biomarkers correlating, e.g., with therapy response. Prior to use these biopsies for expensive analysis, quality control (QC) is performed regarding, e.g., morphology, tumor content. However, studies to correlate the results obtained from QC evaluation with clinical outcomes are lacking. The purpose of this study was to determine if data from the QC process correlate with therapy response. Method: We investigated 64 baseline and 50 on-treatment (C1D15-21) biopsies from 67 patients participating in an ongoing phase I clinical trial receiving pembrolizumab for their rare tumors such as e.g. adrenocortical carcinoma, angiosarcoma, alveolar rhabdomyosarcoma, carcinoma of unknown primary, epithelioid hemangioendothelioma, malignant neoplasm of tunica vaginalis, paraganglioma, penile carcinoma, pituitary tumor, renal medullary carcinoma, teratoma, and others. Baseline and the on-treatment biopsies were taken in u003e90% from the same amenable tumor lesion. Matched biopsies were available in n= 48 (71.64%) and data on response was available for 54 (80.60%) patients. From all biopsies the tumor content was estimated by a pathologist on an HE 2017 Oct 1-4; Boston, MA. Philadelphia (PA): AACR; Cancer Immunol Res 2018;6(9 Suppl):Abstract nr A44.