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
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 immu...
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BMJ Publishing Group
2024-11-01
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| Series: | Journal for ImmunoTherapy of Cancer |
| Online Access: | https://jitc.bmj.com/content/12/11/e009573.full |
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| author | Howard L McLeod A Dimitrios Colevas Georges Azzi Kevin C Flanagan Douglas Adkins Jessica Ley Ezra E W Cohen James Wade Jon Earls Jeffrey Hiken Rachel L Wellinghoff Michelle M Ponder William H Westra Vera Vavinskaya Leisa Sutton Ida Deichaite Orlan K Macdonald Karim Welaya Andrew W Pippas Jennifer Slim Bruce Bank Xingwei Sui Steven E Kossman Todd D Shenkenberg Warren L Alexander Katharine A Price David N Messina Jarret I Glasscock Eric J Duncavage |
| author_facet | Howard L McLeod A Dimitrios Colevas Georges Azzi Kevin C Flanagan Douglas Adkins Jessica Ley Ezra E W Cohen James Wade Jon Earls Jeffrey Hiken Rachel L Wellinghoff Michelle M Ponder William H Westra Vera Vavinskaya Leisa Sutton Ida Deichaite Orlan K Macdonald Karim Welaya Andrew W Pippas Jennifer Slim Bruce Bank Xingwei Sui Steven E Kossman Todd D Shenkenberg Warren L Alexander Katharine A Price David N Messina Jarret I Glasscock Eric J Duncavage |
| author_sort | Howard L McLeod |
| collection | DOAJ |
| description | 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 (p=0.015 and p=0.037, respectively). OncoPrism-HNSCC had more than threefold higher specificity than programmed death-ligand 1 combined positive score and nearly fourfold higher sensitivity than tumor mutational burden for predicting disease control.Conclusions Here, we demonstrate the clinical validity of the OncoPrism-HNSCC assay in identifying patients with disease control in response to anti-PD-1 immune checkpoint inhibitors.Trial registration number NCT04510129. |
| format | Article |
| id | doaj-art-5aeea84f23854b819fffb4fd1e4886a8 |
| institution | DOAJ |
| issn | 2051-1426 |
| language | English |
| publishDate | 2024-11-01 |
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| series | Journal for ImmunoTherapy of Cancer |
| spelling | doaj-art-5aeea84f23854b819fffb4fd1e4886a82025-08-20T03:07:58ZengBMJ Publishing GroupJournal for ImmunoTherapy of Cancer2051-14262024-11-01121110.1136/jitc-2024-009573Multicenter 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 studyHoward L McLeod0A Dimitrios Colevas1Georges Azzi2Kevin C Flanagan3Douglas Adkins4Jessica Ley5Ezra E W Cohen6James Wade7Jon Earls8Jeffrey Hiken9Rachel L Wellinghoff10Michelle M Ponder11William H Westra12Vera Vavinskaya13Leisa Sutton14Ida Deichaite15Orlan K Macdonald16Karim Welaya17Andrew W Pippas18Jennifer Slim19Bruce Bank20Xingwei Sui21Steven E Kossman22Todd D Shenkenberg23Warren L Alexander24Katharine A Price25David N Messina26Jarret I Glasscock27Eric J Duncavage282 Utah Tech University, St George, Utah, USA20 Stanford University, Stanford, California, USA10 Holy Cross Hospital Medical Group, Fort Lauderdale, Florida, USA1 Cofactor Genomics Inc, Saint Louis, Missouri, USA3Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine, St. Louis, MO, USA19 Division of Oncology, Department of Medicine, Washington University in St Louis School of Medicine, St Louis, Missouri, USA21 Division of Hematology and Oncology, University of California San Diego, La Jolla, California, USA9 Decatur Memorial Hospital, Decatur, Illinois, USA1 Cofactor Genomics Inc, Saint Louis, Missouri, USA1 Cofactor Genomics Inc, Saint Louis, Missouri, USA1 Cofactor Genomics Inc, Saint Louis, Missouri, USA1 Cofactor Genomics Inc, Saint Louis, Missouri, USA3 Icahn School of Medicine at Mount Sinai, New York, New York, USA4 Division of Hematology and Oncology, UCSD Moores Cancer Center, La Jolla, California, USA5 UCSD Moores Cancer Center, La Jolla, California, USA6 Radiation Medicine and Applied Sciences, UCSD Moores Cancer Center, La Jolla, California, USA7 Cancer Care Northwest, Spokane Valley, Washington, USA8 CoxHealth Medical Oncology, Springfield, Missouri, UK11 John B Amos Cancer Center, Columbus Regional Research Institute, Centricity Research, Columbus, Georgia, USA12 Multicare Institute for Research and Innovation, Tacoma, Washington, USA13 Northwest Oncology & Hematology, Elk Grove Village, Illinois, USA14 Providence Regional Cancer System, Lacey, Washington, USA15 Sharp Clinical Oncology Research, San Diego, California, USA16 Valley Cancer Associates, Harlingen, Texas, USA17 William Beaumont Army Medical Center and The Geneva Foundation, Fort Bliss, Texas, USA18 Mayo Clinic, Rochester, Minnesota, USA1 Cofactor Genomics Inc, Saint Louis, Missouri, USA1 Cofactor Genomics Inc, Saint Louis, Missouri, USA22 Department of Pathology and Immunology, Washington University in St Louis School of Medicine, St Louis, Missouri, USABackground 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 (p=0.015 and p=0.037, respectively). OncoPrism-HNSCC had more than threefold higher specificity than programmed death-ligand 1 combined positive score and nearly fourfold higher sensitivity than tumor mutational burden for predicting disease control.Conclusions Here, we demonstrate the clinical validity of the OncoPrism-HNSCC assay in identifying patients with disease control in response to anti-PD-1 immune checkpoint inhibitors.Trial registration number NCT04510129.https://jitc.bmj.com/content/12/11/e009573.full |
| spellingShingle | Howard L McLeod A Dimitrios Colevas Georges Azzi Kevin C Flanagan Douglas Adkins Jessica Ley Ezra E W Cohen James Wade Jon Earls Jeffrey Hiken Rachel L Wellinghoff Michelle M Ponder William H Westra Vera Vavinskaya Leisa Sutton Ida Deichaite Orlan K Macdonald Karim Welaya Andrew W Pippas Jennifer Slim Bruce Bank Xingwei Sui Steven E Kossman Todd D Shenkenberg Warren L Alexander Katharine A Price David N Messina Jarret I Glasscock Eric J Duncavage 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 Journal for ImmunoTherapy of Cancer |
| title | 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 |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | 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 |
| title_sort | 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 |
| url | https://jitc.bmj.com/content/12/11/e009573.full |
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