Mutational profiles of head and neck squamous cell carcinomas based upon human papillomavirus status in the Veterans Affairs National Precision Oncology Program

J Cancer Res Clin Oncol. 2023 Jan;149(1):69-77. doi: 10.1007/s00432-022-04358-7. Epub 2022 Sep 18.

Abstract

Background: Patients with advanced head and neck squamous cell carcinoma (HNSCC) associated with human papillomavirus (HPV) demonstrate favorable clinical outcomes compared to patients bearing HPV-negative HNSCC. We sought to characterize the association between HPV status and mutational profiles among patients served by the Veterans Health Administration (VHA).

Methods: We performed a retrospective analysis of all Veterans with primary HNSCC tumors who underwent next-generation sequencing (NGS) through the VHA's National Precision Oncology Program between July 2016 and February 2019. HPV status was determined by clinical pathology reports of p16 immunohistochemical staining; gene variant pathogenicity was classified using OncoKB, an online precision oncology knowledge database, and mutation frequencies were compared using Fisher's exact test.

Results: A total of 79 patients met inclusion criteria, of which 48 (60.8%) had p16-positive tumors. Patients with p16-negative HNSCC were more likely to have mutations in TP53 (p < 0.0001), and a trend towards increased mutation frequency was observed within NOTCH1 (p = 0.032) and within the composite CDK/Rb pathway (p = 0.065). Mutations in KRAS, NRAS, HRAS, and FBXW7 were exclusively identified within p16-positive tumors, and a trend towards increased frequency was observed within the PI3K pathway (p = 0.051). No difference in overall mutational burden was observed between the two groups.

Conclusions: In accordance with the previous studies, no clear molecular basis for improved prognosis among patients harboring HPV-positive disease has been elucidated. Though no targeted therapies are approved based upon HPV-status, current efforts to trial PI3K inhibitors and mTOR inhibitors across patients with HPV-positive disease bear genomic rationale based upon the current findings.

Keywords: HPV status; Head and neck cancer; Molecular profiling; Mutational frequency.

MeSH terms

  • Cyclin-Dependent Kinase Inhibitor p16 / genetics
  • Head and Neck Neoplasms* / complications
  • Head and Neck Neoplasms* / genetics
  • Human Papillomavirus Viruses
  • Humans
  • Mutation
  • Papillomaviridae / genetics
  • Papillomavirus Infections* / complications
  • Papillomavirus Infections* / genetics
  • Phosphatidylinositol 3-Kinases / genetics
  • Precision Medicine
  • Retrospective Studies
  • Squamous Cell Carcinoma of Head and Neck / complications
  • Squamous Cell Carcinoma of Head and Neck / genetics
  • Veterans*

Substances

  • Phosphatidylinositol 3-Kinases
  • Cyclin-Dependent Kinase Inhibitor p16