Prospective Phase II Trial of Definitive Chemoradiation and Concurrent Nivolumab in Locally Advanced p16+ Oropharynx Cancer

Purpose: We conducted a prospective, single-institution phase II trial to test the hypothesis that the addition of nivolumab to definitive chemoradiation would improve the progression-free survival (PFS) among patients with high-risk p16+ oropharyngeal squamous cell carcinoma (OPSCC). Methods and Ma...

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Main Authors: Samuel Nicholas Regan, MD, Jennifer Shah, MD, Krithika Suresh, PhD, Krystal A. Morales, MD, PhD, Yue Cao, PhD, Madhava Aryal, PhD, Benjamin S. Rosen, PhD, Heather Walline, PhD, Choonik Lee, PhD, Jessica Aldous, BS, Paul L. Swiecicki, MD, Keith A. Casper, MD, Steven B. Chinn, MD, Kelly M. Malloy, MD, Mark E.P. Prince, MD, Chaz L. Stucken, MD, Andrew G. Shuman, MD, Molly Heft-Neal, MD, Teresa H. Lyden, MA, CCC-SLP, Anna Blakely, MA, CCC-SLP, Madison Ambrose, MA, CCC-SLP, John Chad Brenner, PhD, Francis P. Worden, MD, Michelle L. Mierzwa, MD
Format: Article
Language:English
Published: Elsevier 2025-10-01
Series:Advances in Radiation Oncology
Online Access:http://www.sciencedirect.com/science/article/pii/S2452109425001198
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author Samuel Nicholas Regan, MD
Jennifer Shah, MD
Krithika Suresh, PhD
Krystal A. Morales, MD, PhD
Yue Cao, PhD
Madhava Aryal, PhD
Benjamin S. Rosen, PhD
Heather Walline, PhD
Choonik Lee, PhD
Jessica Aldous, BS
Paul L. Swiecicki, MD
Keith A. Casper, MD
Steven B. Chinn, MD
Kelly M. Malloy, MD
Mark E.P. Prince, MD
Chaz L. Stucken, MD
Andrew G. Shuman, MD
Molly Heft-Neal, MD
Teresa H. Lyden, MA, CCC-SLP
Anna Blakely, MA, CCC-SLP
Madison Ambrose, MA, CCC-SLP
John Chad Brenner, PhD
Francis P. Worden, MD
Michelle L. Mierzwa, MD
author_facet Samuel Nicholas Regan, MD
Jennifer Shah, MD
Krithika Suresh, PhD
Krystal A. Morales, MD, PhD
Yue Cao, PhD
Madhava Aryal, PhD
Benjamin S. Rosen, PhD
Heather Walline, PhD
Choonik Lee, PhD
Jessica Aldous, BS
Paul L. Swiecicki, MD
Keith A. Casper, MD
Steven B. Chinn, MD
Kelly M. Malloy, MD
Mark E.P. Prince, MD
Chaz L. Stucken, MD
Andrew G. Shuman, MD
Molly Heft-Neal, MD
Teresa H. Lyden, MA, CCC-SLP
Anna Blakely, MA, CCC-SLP
Madison Ambrose, MA, CCC-SLP
John Chad Brenner, PhD
Francis P. Worden, MD
Michelle L. Mierzwa, MD
author_sort Samuel Nicholas Regan, MD
collection DOAJ
description Purpose: We conducted a prospective, single-institution phase II trial to test the hypothesis that the addition of nivolumab to definitive chemoradiation would improve the progression-free survival (PFS) among patients with high-risk p16+ oropharyngeal squamous cell carcinoma (OPSCC). Methods and Materials: Patients with previously-untreated locoregionally advanced, p16+ OPSCC (clinical T4/N3, matted lymph nodes, and/or retropharyngeal lymphadenopathy) were enrolled. Patients received a priming dose of nivolumab, concurrent nivolumab and chemoradiation (70 Gy to PTVhigh, 56 Gy to PTVlow, weekly carboplatin/paclitaxel), and 4 cycles of adjuvant nivolumab over 12 weeks. The primary endpoint was 2-year PFS compared to an institutional historic control of 68%. Exploratory endpoints included associations between survival and circulating tumor DNA kinetics during treatment and pretreatment/midtreatment 18F-fluorodeoxyglucose positron-emission tomography with computed tomography and magnetic resonance imaging metrics (gross tumor volume, low blood volume tumor subvolume, low apparent diffusion coefficient tumor subvolume, and metabolic tumor volume ≥50% of maximum standardized uptake value). Results: Twenty-six patients were enrolled prior to an interim analysis; 65% cT4, 46% with matted nodes, with a median total gross tumor volume of 60 cc (range, 36-165). Estimated 2-year PFS was 65% (2-sided 90% CI, 46%-79%) and 2-year distant metastasis-free survival was 84% (66%-93%). The majority (69%) did not complete the full course of nivolumab because of toxicity, and 54% experienced grade ≥3 acute dysphagia. Statistical analyses showed that increasing values of imaging-defined primary tumor subvolumes were significant associated with inferior PFS (eg, midtreatment low apparent diffusion coefficient: HR, 5.61; P = .05) and distant metastasis-free survival (eg, pretreatment metabolic tumor volume ≥50% of maximum standardized uptake value: HR, 2.97; P = .05). Midtreatment circulating tumor DNA kinetics were not associated with survival endpoints. Conclusions: Concurrent and adjuvant nivolumab did not improve PFS in locoregionally advanced Human papillomavirus+ OPSCC and was associated with significant toxicity. Physiological magnetic resonance imaging and 18F-fluorodeoxyglucose positron-emission tomography with computed tomography imaging markers are potential biomarkers to guide future therapy escalation strategies in high-risk oropharynx cancer.
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spelling doaj-art-b4f0141e65744dfd84ef84b34a621d8c2025-08-20T03:05:52ZengElsevierAdvances in Radiation Oncology2452-10942025-10-01101010183210.1016/j.adro.2025.101832Prospective Phase II Trial of Definitive Chemoradiation and Concurrent Nivolumab in Locally Advanced p16+ Oropharynx CancerSamuel Nicholas Regan, MD0Jennifer Shah, MD1Krithika Suresh, PhD2Krystal A. Morales, MD, PhD3Yue Cao, PhD4Madhava Aryal, PhD5Benjamin S. Rosen, PhD6Heather Walline, PhD7Choonik Lee, PhD8Jessica Aldous, BS9Paul L. Swiecicki, MD10Keith A. Casper, MD11Steven B. Chinn, MD12Kelly M. Malloy, MD13Mark E.P. Prince, MD14Chaz L. Stucken, MD15Andrew G. Shuman, MD16Molly Heft-Neal, MD17Teresa H. Lyden, MA, CCC-SLP18Anna Blakely, MA, CCC-SLP19Madison Ambrose, MA, CCC-SLP20John Chad Brenner, PhD21Francis P. Worden, MD22Michelle L. Mierzwa, MD23Department of Radiation Oncology, University of Michigan, Ann Arbor, MichiganDepartment of Radiation Oncology, University of Michigan, Ann Arbor, MichiganDepartment of Radiation Oncology, University of Michigan, Ann Arbor, Michigan; Department of Biostatistics, School of Public Health, University of Michigan, Ann Arbor, MichiganDepartment of Radiation Oncology, University of Michigan, Ann Arbor, MichiganDepartment of Radiation Oncology, University of Michigan, Ann Arbor, MichiganDepartment of Radiation Oncology, University of Michigan, Ann Arbor, MichiganDepartment of Radiation Oncology, University of Michigan, Ann Arbor, MichiganDepartment of Otolaryngology - Head and Neck Surgery, University of Michigan, Ann Arbor, MichiganDepartment of Radiation Oncology, University of Michigan, Ann Arbor, MichiganDepartment of Biostatistics, School of Public Health, University of Michigan, Ann Arbor, MichiganDepartment of Internal Medicine, Division of Hematology/Oncology, University of Michigan, Ann Arbor, MichiganDepartment of Otolaryngology - Head and Neck Surgery, University of Michigan, Ann Arbor, MichiganDepartment of Otolaryngology - Head and Neck Surgery, University of Michigan, Ann Arbor, MichiganDepartment of Otolaryngology - Head and Neck Surgery, University of Michigan, Ann Arbor, MichiganDepartment of Otolaryngology - Head and Neck Surgery, University of Michigan, Ann Arbor, MichiganDepartment of Otolaryngology - Head and Neck Surgery, University of Michigan, Ann Arbor, MichiganDepartment of Otolaryngology - Head and Neck Surgery, University of Michigan, Ann Arbor, MichiganDepartment of Otolaryngology - Head and Neck Surgery, University of Michigan, Ann Arbor, MichiganDepartment of Speech and Language Pathology, University of Michigan, Ann Arbor, MichiganDepartment of Speech and Language Pathology, University of Michigan, Ann Arbor, MichiganDepartment of Speech and Language Pathology, University of Michigan, Ann Arbor, MichiganDepartment of Otolaryngology - Head and Neck Surgery, University of Michigan, Ann Arbor, MichiganDepartment of Internal Medicine, Division of Hematology/Oncology, University of Michigan, Ann Arbor, MichiganDepartment of Radiation Oncology, University of Michigan, Ann Arbor, Michigan; Corresponding author: Michelle L. Mierzwa, MD.Purpose: We conducted a prospective, single-institution phase II trial to test the hypothesis that the addition of nivolumab to definitive chemoradiation would improve the progression-free survival (PFS) among patients with high-risk p16+ oropharyngeal squamous cell carcinoma (OPSCC). Methods and Materials: Patients with previously-untreated locoregionally advanced, p16+ OPSCC (clinical T4/N3, matted lymph nodes, and/or retropharyngeal lymphadenopathy) were enrolled. Patients received a priming dose of nivolumab, concurrent nivolumab and chemoradiation (70 Gy to PTVhigh, 56 Gy to PTVlow, weekly carboplatin/paclitaxel), and 4 cycles of adjuvant nivolumab over 12 weeks. The primary endpoint was 2-year PFS compared to an institutional historic control of 68%. Exploratory endpoints included associations between survival and circulating tumor DNA kinetics during treatment and pretreatment/midtreatment 18F-fluorodeoxyglucose positron-emission tomography with computed tomography and magnetic resonance imaging metrics (gross tumor volume, low blood volume tumor subvolume, low apparent diffusion coefficient tumor subvolume, and metabolic tumor volume ≥50% of maximum standardized uptake value). Results: Twenty-six patients were enrolled prior to an interim analysis; 65% cT4, 46% with matted nodes, with a median total gross tumor volume of 60 cc (range, 36-165). Estimated 2-year PFS was 65% (2-sided 90% CI, 46%-79%) and 2-year distant metastasis-free survival was 84% (66%-93%). The majority (69%) did not complete the full course of nivolumab because of toxicity, and 54% experienced grade ≥3 acute dysphagia. Statistical analyses showed that increasing values of imaging-defined primary tumor subvolumes were significant associated with inferior PFS (eg, midtreatment low apparent diffusion coefficient: HR, 5.61; P = .05) and distant metastasis-free survival (eg, pretreatment metabolic tumor volume ≥50% of maximum standardized uptake value: HR, 2.97; P = .05). Midtreatment circulating tumor DNA kinetics were not associated with survival endpoints. Conclusions: Concurrent and adjuvant nivolumab did not improve PFS in locoregionally advanced Human papillomavirus+ OPSCC and was associated with significant toxicity. Physiological magnetic resonance imaging and 18F-fluorodeoxyglucose positron-emission tomography with computed tomography imaging markers are potential biomarkers to guide future therapy escalation strategies in high-risk oropharynx cancer.http://www.sciencedirect.com/science/article/pii/S2452109425001198
spellingShingle Samuel Nicholas Regan, MD
Jennifer Shah, MD
Krithika Suresh, PhD
Krystal A. Morales, MD, PhD
Yue Cao, PhD
Madhava Aryal, PhD
Benjamin S. Rosen, PhD
Heather Walline, PhD
Choonik Lee, PhD
Jessica Aldous, BS
Paul L. Swiecicki, MD
Keith A. Casper, MD
Steven B. Chinn, MD
Kelly M. Malloy, MD
Mark E.P. Prince, MD
Chaz L. Stucken, MD
Andrew G. Shuman, MD
Molly Heft-Neal, MD
Teresa H. Lyden, MA, CCC-SLP
Anna Blakely, MA, CCC-SLP
Madison Ambrose, MA, CCC-SLP
John Chad Brenner, PhD
Francis P. Worden, MD
Michelle L. Mierzwa, MD
Prospective Phase II Trial of Definitive Chemoradiation and Concurrent Nivolumab in Locally Advanced p16+ Oropharynx Cancer
Advances in Radiation Oncology
title Prospective Phase II Trial of Definitive Chemoradiation and Concurrent Nivolumab in Locally Advanced p16+ Oropharynx Cancer
title_full Prospective Phase II Trial of Definitive Chemoradiation and Concurrent Nivolumab in Locally Advanced p16+ Oropharynx Cancer
title_fullStr Prospective Phase II Trial of Definitive Chemoradiation and Concurrent Nivolumab in Locally Advanced p16+ Oropharynx Cancer
title_full_unstemmed Prospective Phase II Trial of Definitive Chemoradiation and Concurrent Nivolumab in Locally Advanced p16+ Oropharynx Cancer
title_short Prospective Phase II Trial of Definitive Chemoradiation and Concurrent Nivolumab in Locally Advanced p16+ Oropharynx Cancer
title_sort prospective phase ii trial of definitive chemoradiation and concurrent nivolumab in locally advanced p16 oropharynx cancer
url http://www.sciencedirect.com/science/article/pii/S2452109425001198
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