Predictive Role of TP53, PIK3CA and MLL2 in ER+ HER2+ Breast Bancer: Biomarker Analysis of Neo-ALL-IN [NCT 01275859]

Anticancer Res. 2020 Oct;40(10):5883-5893. doi: 10.21873/anticanres.14608.

Abstract

Background/aim: Somatic mutations were investigated in 21 patients with postmenopausal estrogen receptor (ER)-positive and human epidermal growth factor receptor-2 (HER-2)-positive (ER+HER2+) breast cancer (BC) treated with neoadjuvant letrozole and lapatinib, to identify their distinct molecular landscape.

Patients and methods: We used tissue samples of 21 patients from phase II Neo ALL-IN cohort, and somatic alterations were examined using targeted exome sequencing performed in Foundation Medicine, Inc. (FMI).

Results: TP53 (61.9%) and PIK3CA (57.1%) were the two most frequently mutated genes that were inter-correlated (p=0.026). They were associated with unfavorable clinical outcomes, particularly when accompanying PIK3CA mutations at exon 9 in helical domains. Meanwhile, MLL2 alteration was negatively associated with mutations of TP53 or PIK3CA, and it tended to be present in patients with low KI-67 levels and no initial nodal involvement. Moreover, patients with MLL2 mutations numerically showed more favorable overall response rates (ORR) (80% vs. 56.2%) and better 5-year event-free survival (EFS) rates (100% vs. 87.5%) compared to the wild-type.

Conclusion: Mutations in TP53 and PIK3CA hotspot at exon 9 may be potential negative predictors of ER+HER2+ BC treated with neoadjuvant letrozole and lapatinib, while MLL2 inactivating mutation might confer therapeutic benefit in these patients.

Keywords: ER+/HER2+; MLL2; Neo-ALL-IN; PIK3CA; next-generation sequencing; triple-positive breast cancer.

Publication types

  • Clinical Trial, Phase II

MeSH terms

  • Aged
  • Biomarkers, Tumor / genetics
  • Breast / drug effects
  • Breast / pathology
  • Breast Neoplasms / drug therapy*
  • Breast Neoplasms / genetics
  • Breast Neoplasms / pathology
  • Class I Phosphatidylinositol 3-Kinases / genetics*
  • DNA-Binding Proteins / genetics*
  • Estrogen Receptor alpha / genetics
  • Female
  • Gene Expression Regulation, Neoplastic / drug effects
  • Humans
  • Lapatinib / administration & dosage
  • Letrozole / administration & dosage
  • Middle Aged
  • Mutation / genetics
  • Neoplasm Proteins / genetics*
  • Progression-Free Survival
  • Receptor, ErbB-2 / genetics
  • Tumor Suppressor Protein p53 / genetics*

Substances

  • Biomarkers, Tumor
  • DNA-Binding Proteins
  • ESR1 protein, human
  • Estrogen Receptor alpha
  • KMT2D protein, human
  • Neoplasm Proteins
  • TP53 protein, human
  • Tumor Suppressor Protein p53
  • Lapatinib
  • Letrozole
  • Class I Phosphatidylinositol 3-Kinases
  • PIK3CA protein, human
  • ERBB2 protein, human
  • Receptor, ErbB-2