Multicenter validation of an RNA-based assay to predict anti-PD-1 disease control in patients with recurrent or metastatic head and neck squamous cell carcinoma: the PREDAPT study

作者：Kevin C. Flanagan, Jon Earls, Jeffrey Hiken, Rachel L. Wellinghoff, Michelle M. Ponder, Howard L. McLeod, William H. Westra, Vera Vavinskaya, Leisa Sutton, Ida Deichaite, O. Kenneth Macdonald, Karim Youssry Welaya, James Lloyd Wade, Georges Azzi, Andrew William Pippas, Jennifer Nadine Slim, Bruce B. Bank, Xingwei Sui, Steven E. Kossman, Todd D. Shenkenberg, Warren L. Alexander, Katharine Andress Rowe Price, Jessica C. Ley, David N. Messina, Jarret I Glasscock, Alexander Dimitrios Colevas, Ezra E.W. Cohen, Douglas Adkins, Eric J. Duncavage · 发表于：Journal for ImmunoTherapy of Cancer · 年份：2024 · DOI：10.1136/jitc-2024-009573 · 被引用次数：9 · 研究领域：Head and Neck Cancer Studies、Cancer Immunotherapy and Biomarkers、Ferroptosis and cancer prognosis

BACKGROUND: Despite advances in cancer care and detection, >65% of patients with squamous cell cancer of the head and neck (HNSCC) will develop recurrent and/or metastatic disease. The prognosis for these patients is poor with a 5-year overall survival of 39%. Recent treatment advances in immunotherapy, including immune checkpoint inhibitors like pembrolizumab and nivolumab, have resulted in clinical benefit in a subset of patients. There is a critical clinical need to identify patients who benefit from these antiprogrammed cell death protein 1 (anti-PD-1) immune checkpoint inhibitors. METHODS: Here, we report findings from a multicenter observational study, PREDicting immunotherapy efficacy from Analysis of Pre-treatment Tumor biopsies (PREDAPT), conducted across 17 US healthcare systems. PREDAPT aimed to validate OncoPrism-HNSCC, a clinical biomarker assay predictive of disease control in patients with recurrent or metastatic HNSCC treated with anti-PD-1 immune checkpoint inhibitors as a single agent (monotherapy) and in combination with chemotherapy (chemo-immunotherapy). The test used RNA-sequencing data and machine learning models to score each patient and place them into groups of low, medium, or high. RESULTS: The OncoPrism-HNSCC prediction significantly correlated with disease control in both the monotherapy cohort (n=62, p=0.004) and the chemo-immunotherapy cohort (n=50, p=0.01). OncoPrism-HNSCC also significantly predicted progression-free survival in both cohorts (...