Benefit of Avasopasem Manganese on Severe Oral Mucositis in Head and Neck Cancer in the ROMAN Trial: Unplanned Secondary Analysis

Purpose: Oral mucositis (OM) is a debilitating side effect of cisplatin and intensity-modulated radiation therapy (IMRT) in patients with head and neck cancer. The phase 3 ROMAN trial showed avasopasem manganese (AVA) significantly decreased individual endpoints of incidence and duration of severe o...

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Main Authors: Carryn Anderson, MD, Samuel Salvaggio, PhD, Mickaël De Backer, PhD, Jean-Christophe Chiem, PhD, Gary Walker, MD, MPH, MS, Deborah Saunders, DMD, BSc, Christopher M. Lee, MD, Neal Dunlap, MD, Eugene Kennedy, MD, Robert Beardsley, PhD, Benton Schoen, BA, Marc Buyse, ScD
Format: Article
Language:English
Published: Elsevier 2025-01-01
Series:Advances in Radiation Oncology
Online Access:http://www.sciencedirect.com/science/article/pii/S2452109424002379
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author Carryn Anderson, MD
Samuel Salvaggio, PhD
Mickaël De Backer, PhD
Jean-Christophe Chiem, PhD
Gary Walker, MD, MPH, MS
Deborah Saunders, DMD, BSc
Christopher M. Lee, MD
Neal Dunlap, MD
Eugene Kennedy, MD
Robert Beardsley, PhD
Benton Schoen, BA
Marc Buyse, ScD
author_facet Carryn Anderson, MD
Samuel Salvaggio, PhD
Mickaël De Backer, PhD
Jean-Christophe Chiem, PhD
Gary Walker, MD, MPH, MS
Deborah Saunders, DMD, BSc
Christopher M. Lee, MD
Neal Dunlap, MD
Eugene Kennedy, MD
Robert Beardsley, PhD
Benton Schoen, BA
Marc Buyse, ScD
author_sort Carryn Anderson, MD
collection DOAJ
description Purpose: Oral mucositis (OM) is a debilitating side effect of cisplatin and intensity-modulated radiation therapy (IMRT) in patients with head and neck cancer. The phase 3 ROMAN trial showed avasopasem manganese (AVA) significantly decreased individual endpoints of incidence and duration of severe oral mucositis (SOM, World Health Organization [WHO] grade 3-4), with nominal decrease in severity (WHO grade 4) and significant increase in the delay in onset of SOM. We sought to determine the Net Treatment Benefit (NTB) of AVA versus placebo (PBO) using the generalized pairwise comparisons (GPC) method. Methods and Materials: GPC is a statistical method that permits simultaneous analysis of several prioritized outcomes, comparing all possible pairs of a patient in the active (ie, AVA) group and a patient from the control (ie, PBO) group. NTB is the net benefit across all the outcomes for AVA compared to PBO. Key clinically relevant outcomes from ROMAN were prioritized: (1) WHO grade 4 OM incidence; (2) SOM incidence; (3) days of SOM; (4) days to SOM onset, with 7 days difference defined as the clinical relevance threshold for SOM days and SOM onset. Results: GPC analysis of 407 patients (AVA = 241, placebo = 166) stratified by cisplatin schedule and treatment setting resulted in 13,969 pairwise comparisons. AVA showed statistically significant net benefit on all 4 key outcomes with a 53.9% probability that AVA would benefit patients versus a 35.0% probability that PBO would; the difference between these probabilities was a NTB of 18.9% (P = .0012), translating to an AVA number needed to treat of 5.3 patients. All outcomes contributed to NTB, reflecting improvements in SOM incidence, onset and duration, and in grade 4 OM incidence seen in the original ROMAN analysis. Conclusions: This GPC analysis shows compelling evidence from the ROMAN trial of AVA's clinical benefit across key parameters of SOM burden.
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spelling doaj-art-e98caa25f385439f830d743c53eed5312024-11-28T04:34:47ZengElsevierAdvances in Radiation Oncology2452-10942025-01-01101101674Benefit of Avasopasem Manganese on Severe Oral Mucositis in Head and Neck Cancer in the ROMAN Trial: Unplanned Secondary AnalysisCarryn Anderson, MD0Samuel Salvaggio, PhD1Mickaël De Backer, PhD2Jean-Christophe Chiem, PhD3Gary Walker, MD, MPH, MS4Deborah Saunders, DMD, BSc5Christopher M. Lee, MD6Neal Dunlap, MD7Eugene Kennedy, MD8Robert Beardsley, PhD9Benton Schoen, BA10Marc Buyse, ScD11Department of Radiation Oncology University of Iowa Hospitals & Clinics, Iowa City, Iowa; Corresponding author: Carryn M. Anderson, MDInternational Drug Development Institute (IDDI), Louvain-la-Neuve, BelgiumInternational Drug Development Institute (IDDI), Louvain-la-Neuve, BelgiumInternational Drug Development Institute (IDDI), Louvain-la-Neuve, BelgiumBanner MD Anderson Cancer Center, Gilbert, ArizonaNortheast Cancer Centre of Health Sciences North, Health Sciences North, Northern Ontario School of Medicine, Sudbury, CanadaCancer Care Northwest, Spokane, WashingtonUniversity of Louisville/James Graham Brown Cancer Center, Louisville, KentuckyGalera Therapeutics, Inc, Malvern, PennsylvaniaGalera Therapeutics, Inc, Malvern, PennsylvaniaGalera Therapeutics, Inc, Malvern, PennsylvaniaInternational Drug Development Institute (IDDI), Louvain-la-Neuve, BelgiumPurpose: Oral mucositis (OM) is a debilitating side effect of cisplatin and intensity-modulated radiation therapy (IMRT) in patients with head and neck cancer. The phase 3 ROMAN trial showed avasopasem manganese (AVA) significantly decreased individual endpoints of incidence and duration of severe oral mucositis (SOM, World Health Organization [WHO] grade 3-4), with nominal decrease in severity (WHO grade 4) and significant increase in the delay in onset of SOM. We sought to determine the Net Treatment Benefit (NTB) of AVA versus placebo (PBO) using the generalized pairwise comparisons (GPC) method. Methods and Materials: GPC is a statistical method that permits simultaneous analysis of several prioritized outcomes, comparing all possible pairs of a patient in the active (ie, AVA) group and a patient from the control (ie, PBO) group. NTB is the net benefit across all the outcomes for AVA compared to PBO. Key clinically relevant outcomes from ROMAN were prioritized: (1) WHO grade 4 OM incidence; (2) SOM incidence; (3) days of SOM; (4) days to SOM onset, with 7 days difference defined as the clinical relevance threshold for SOM days and SOM onset. Results: GPC analysis of 407 patients (AVA = 241, placebo = 166) stratified by cisplatin schedule and treatment setting resulted in 13,969 pairwise comparisons. AVA showed statistically significant net benefit on all 4 key outcomes with a 53.9% probability that AVA would benefit patients versus a 35.0% probability that PBO would; the difference between these probabilities was a NTB of 18.9% (P = .0012), translating to an AVA number needed to treat of 5.3 patients. All outcomes contributed to NTB, reflecting improvements in SOM incidence, onset and duration, and in grade 4 OM incidence seen in the original ROMAN analysis. Conclusions: This GPC analysis shows compelling evidence from the ROMAN trial of AVA's clinical benefit across key parameters of SOM burden.http://www.sciencedirect.com/science/article/pii/S2452109424002379
spellingShingle Carryn Anderson, MD
Samuel Salvaggio, PhD
Mickaël De Backer, PhD
Jean-Christophe Chiem, PhD
Gary Walker, MD, MPH, MS
Deborah Saunders, DMD, BSc
Christopher M. Lee, MD
Neal Dunlap, MD
Eugene Kennedy, MD
Robert Beardsley, PhD
Benton Schoen, BA
Marc Buyse, ScD
Benefit of Avasopasem Manganese on Severe Oral Mucositis in Head and Neck Cancer in the ROMAN Trial: Unplanned Secondary Analysis
Advances in Radiation Oncology
title Benefit of Avasopasem Manganese on Severe Oral Mucositis in Head and Neck Cancer in the ROMAN Trial: Unplanned Secondary Analysis
title_full Benefit of Avasopasem Manganese on Severe Oral Mucositis in Head and Neck Cancer in the ROMAN Trial: Unplanned Secondary Analysis
title_fullStr Benefit of Avasopasem Manganese on Severe Oral Mucositis in Head and Neck Cancer in the ROMAN Trial: Unplanned Secondary Analysis
title_full_unstemmed Benefit of Avasopasem Manganese on Severe Oral Mucositis in Head and Neck Cancer in the ROMAN Trial: Unplanned Secondary Analysis
title_short Benefit of Avasopasem Manganese on Severe Oral Mucositis in Head and Neck Cancer in the ROMAN Trial: Unplanned Secondary Analysis
title_sort benefit of avasopasem manganese on severe oral mucositis in head and neck cancer in the roman trial unplanned secondary analysis
url http://www.sciencedirect.com/science/article/pii/S2452109424002379
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