PITX2 DNA methylation: A prognostic/predictive biomarker for anthracycline-based chemotherapy

Background: Published data on the basis of a research-use-only PITX2 methylation assay using fresh-frozen tissue showed that the PITX2 DNA methylation may be a predictive marker for response to (neo) adjuvant anthracycline-based chemotherapy in breast cancer patients, including high-risk lymph node positive, estrogen receptor-positive, HER2-negative breast cancer and triple-negative breast cancer (TNBC). A retrospective study showed that high-risk ER-positive patients with a high PITX2 methylation ratio (PMR\u003e12) had a poor outcome after anthracycline-based chemotherapy [HR 2,28; 95%-Cl 1,49 - 3,49; p Summary: In this hypothesis generating study DNA-methylation of PITX2 identifies with high statistical significance anthracycline-treated patients with poor vs good survival. Recurrence risk increases with increasing PITX2 DNA-methylation. PITX2 DNA-methylation shows no prognostic, but significant predictive value for anthracycline treatment in TNBC patients in this study. To confirm conclusively the predictive value of PITX2 DNA-methylation, the results warrant a confirmatory study with tumor specimens from a prospective trial of anthracycline-treated patients vs. anthracycline-free treated patients. Citation Format: Marion Kiechle, Gabriele Schricker, Rudolf Napieralski, Michaela Aubele, Gert Auer, Kurt Ulm, Jonathan Perkins, Stefan Paepke, Moritz Hamann, Olaf G. Wilhelm. PITX2 DNA methylation: A prognostic/predictive biomarker for anthracycline-based chemotherapy [abstract]. In: Proceedings of the 2019 San Antonio Breast Cancer Symposium; 2019 Dec 10-14; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2020;80(4 Suppl):Abstract nr P3-08-65.