Comprehensive genomic profiling of ESR1, PIK3CA, AKT1, and PTEN in HR(+)HER2(-) metastatic breast cancer: prevalence along treatment course and predictive value for endocrine therapy resistance in real-world practice

Breast Cancer Res Treat. 2024 Oct;207(3):599-609. doi: 10.1007/s10549-024-07376-w. Epub 2024 Jun 14.

Abstract

Background: The treatment landscape for HR(+)HER2(-) metastatic breast cancer (MBC) is evolving for patients with ESR1 mutations (mut) and PI3K/AKT pathway genomic alterations (GA). We sought to inform clinical utility for comprehensive genomic profiling (CGP) using tissue (TBx) and liquid biopsies (LBx) in HR(+)HER2(-) MBC.

Methods: Records from a de-identified breast cancer clinicogenomic database for patients who underwent TBx/LBx testing at Foundation Medicine during routine clinical care at ~ 280 US cancer clinics between 01/2011 and 09/2023 were assessed. GA prevalence [ESR1mut, PIK3CAmut, AKT1mut, PTENmut, and PTEN homozygous copy loss (PTENloss)] were calculated in TBx and LBx [stratified by ctDNA tumor fraction (TF)] during the first three lines of therapy. Real-world progression-free survival (rwPFS) and overall survival (rwOS) were compared between groups by Cox models adjusted for prognostic factors.

Results: ~ 60% of cases harbored 1 + GA in 1st-line TBx (1266/2154) or LBx TF ≥ 1% (80/126) and 26.5% (43/162) in LBx TF < 1%. ESR1mut was found in 8.1% TBx, 17.5% LBx TF ≥ 1%, and 4.9% LBx TF < 1% in 1st line, increasing to 59% in 3rd line (LBx TF ≥ 1%). PTENloss was detected at higher rates in TBx (4.3%) than LBx (1% in TF ≥ 1%). Patients receiving 1st-line aromatase inhibitor + CDK4/6 inhibitor (n = 573) with ESR1mut had less favorable rwPFS and rwOS versus ESR1 wild-type; no differences were observed for fulvestrant + CDK4/6 inhibitor (n = 348).

Conclusion: Our study suggests obtaining TBx for CGP at time of de novo/recurrent diagnosis, followed by LBx for detecting acquired GA in 2nd + lines. Reflex TBx should be considered when ctDNA TF < 1%.

Keywords: AKT inhibitor; ESR1; Genomic sequencing; HR(+)HER2(−); Metastatic breast cancer.

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Agents, Hormonal / therapeutic use
  • Biomarkers, Tumor* / genetics
  • Breast Neoplasms* / drug therapy
  • Breast Neoplasms* / genetics
  • Breast Neoplasms* / mortality
  • Breast Neoplasms* / pathology
  • Class I Phosphatidylinositol 3-Kinases* / genetics
  • Drug Resistance, Neoplasm* / genetics
  • Estrogen Receptor alpha* / genetics
  • Estrogen Receptor alpha* / metabolism
  • Female
  • Genomics / methods
  • Humans
  • Middle Aged
  • Mutation
  • Neoplasm Metastasis
  • PTEN Phosphohydrolase* / genetics
  • Prevalence
  • Prognosis
  • Proto-Oncogene Proteins c-akt* / genetics
  • Proto-Oncogene Proteins c-akt* / metabolism
  • Receptor, ErbB-2* / genetics
  • Receptor, ErbB-2* / metabolism
  • Receptors, Estrogen / metabolism
  • Receptors, Progesterone / metabolism

Substances

  • PTEN Phosphohydrolase
  • PIK3CA protein, human
  • PTEN protein, human
  • Estrogen Receptor alpha
  • Class I Phosphatidylinositol 3-Kinases
  • ESR1 protein, human
  • Proto-Oncogene Proteins c-akt
  • AKT1 protein, human
  • Receptor, ErbB-2
  • Biomarkers, Tumor
  • ERBB2 protein, human
  • Receptors, Estrogen
  • Antineoplastic Agents, Hormonal
  • Receptors, Progesterone