Annotation-free deep learning algorithm trained on hematoxylin & eosin images predicts epithelial-to-mesenchymal transition phenotype and endocrine response in estrogen receptor-positive breast cancer

Breast Cancer Res. 2025 Jan 12;27(1):6. doi: 10.1186/s13058-025-01959-1.

Abstract

Recent evidence indicates that endocrine resistance in estrogen receptor-positive (ER+) breast cancer is closely correlated with phenotypic characteristics of epithelial-to-mesenchymal transition (EMT). Nonetheless, identifying tumor tissues with a mesenchymal phenotype remains challenging in clinical practice. In this study, we validated the correlation between EMT status and resistance to endocrine therapy in ER+ breast cancer from a transcriptomic perspective. To confirm the presence of morphological discrepancies in tumor tissues of ER+ breast cancer classified as epithelial- and mesenchymal-phenotypes according to EMT-related transcriptional features, we trained deep learning algorithms based on EfficientNetV2 architecture to assign the phenotypic status for each patient utilizing hematoxylin & eosin (H&E)-stained slides from The Cancer Genome Atlas database. Our classifier model accurately identified the precise phenotypic status, achieving an area under the curve (AUC) of 0.886 at the tile-level and an AUC of 0.910 at the slide-level. Furthermore, we evaluated the efficacy of the classifier in predicting endocrine response using data from an independent ER+ breast cancer patient cohort. Our classifier achieved a predicting accuracy of 81.25%, and 88.7% slides labeled as endocrine resistant were predicted as the mesenchymal-phenotype, while 75.6% slides labeled as sensitive were predicted as the epithelial-phenotype. Our work introduces an H&E-based framework capable of accurately predicting EMT phenotype and endocrine response for ER+ breast cancer, demonstrating its potential for clinical application and benefit.

Keywords: Deep learning; Digital pathology; Endocrine therapy response; Epithelial-to-mesenchymal transition; Estrogen receptor-positive breast cancer; Phenotypic classifier.

MeSH terms

  • Algorithms
  • Antineoplastic Agents, Hormonal / pharmacology
  • Antineoplastic Agents, Hormonal / therapeutic use
  • Biomarkers, Tumor / metabolism
  • Breast Neoplasms* / genetics
  • Breast Neoplasms* / metabolism
  • Breast Neoplasms* / pathology
  • Deep Learning*
  • Drug Resistance, Neoplasm
  • Eosine Yellowish-(YS)*
  • Epithelial-Mesenchymal Transition*
  • Female
  • Gene Expression Profiling / methods
  • Gene Expression Regulation, Neoplastic
  • Hematoxylin
  • Humans
  • Phenotype
  • Receptors, Estrogen* / metabolism

Substances

  • Receptors, Estrogen
  • Eosine Yellowish-(YS)
  • Hematoxylin
  • Antineoplastic Agents, Hormonal
  • Biomarkers, Tumor