PET/CT scan and biopsy-driven approach for safe anti-PD-1 therapy discontinuation in patients with advanced melanoma

Background Limited data exist on safe discontinuation of antiprogrammed cell death protein 1 (PD-1) therapy in responding patients with advanced melanoma. The use of 18fluorodeoxyglucose (18FDG)-PET/CT scan and tumor biopsy for assessment of active disease may be an effective predictive biomarker to...

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Main Authors: Michael B. Atkins, Suthee Rapisuwon, Arash Radfar, Kellie Gardner, Neil J. Shah, Geoffrey T. Gibney, Jacob Zaemes, Shelly Shand, David Swoboda, Vesna Petronic-Rosic, Michael J. Reilly, Waddah B. Al-Refaie
Format: Article
Language:English
Published: BMJ Publishing Group 2021-10-01
Series:Journal for ImmunoTherapy of Cancer
Online Access:https://jitc.bmj.com/content/9/10/e002955.full
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author Michael B. Atkins
Suthee Rapisuwon
Arash Radfar
Kellie Gardner
Neil J. Shah
Geoffrey T. Gibney
Jacob Zaemes
Shelly Shand
David Swoboda
Vesna Petronic-Rosic
Michael J. Reilly
Waddah B. Al-Refaie
author_facet Michael B. Atkins
Suthee Rapisuwon
Arash Radfar
Kellie Gardner
Neil J. Shah
Geoffrey T. Gibney
Jacob Zaemes
Shelly Shand
David Swoboda
Vesna Petronic-Rosic
Michael J. Reilly
Waddah B. Al-Refaie
author_sort Michael B. Atkins
collection DOAJ
description Background Limited data exist on safe discontinuation of antiprogrammed cell death protein 1 (PD-1) therapy in responding patients with advanced melanoma. The use of 18fluorodeoxyglucose (18FDG)-PET/CT scan and tumor biopsy for assessment of active disease may be an effective predictive biomarker to guide such treatment decisions.Methods A retrospective study of 122 patients with advanced melanoma treated with anti-PD-1 monotherapy or anti-PD-1/anticytotoxic T-lymphocyte-associated protein 4 combination therapy at Georgetown Lombardi Comprehensive Cancer Center was conducted. Uveal melanoma patients and those receiving concurrent experimental therapy were excluded. Baseline characteristics, treatment outcomes, and survival were analyzed. Patients who decided to come off treatment typically after 12 months using CT scan radiographic complete response (CR), 18FDG-PET/CT scan complete metabolic response (CMR) or tumor biopsy of a non-CR/CMR tumor site negative for active disease (possible pathological CR) were identified and compared with patients who discontinued treatment due to toxicity while their disease was in control. Event-free survival (EFS) was assessed from the last dose of anti-PD-1 therapy to progression requiring subsequent treatment (surgery, radiation, and/or systemic therapy) or referral to hospice/death due to melanoma.Results 24 (20%) patients discontinued treatment by choice with no active disease and 28 (23%) patients discontinued treatment due to toxicity with disease control after 12-month and 4-month median treatment durations, respectively. Similar baseline characteristics were observed between cohorts except higher prior receipt of ipilimumab (29% vs 7%; p=0.036) and fewer BRAF mutant positive disease (17% vs 41%; p=0.064) in patients off treatment by choice. Three-year EFS rates were 95% and 71%, respectively. No significant associations between EFS and sex, disease stage, lactate dehydrogenase elevation, BRAF status, prior systemic therapy, ECOG performance status, presence of brain metastases, or combination versus monotherapy were observed. Tumor biopsies led to alternative management in 3/10 patients due to active metastatic melanoma or second malignancy.Conclusions Anti-PD-1 therapy discontinuation after 12 months when no active disease is observed on CT scan, PET/CT scan or tumor biopsy may have low rates of disease relapse in patients with advanced melanoma. Biopsy of residual disease may frequently lead to a change in management. These findings are undergoing validation in the EA6192 trial.
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spelling doaj-art-da787bd08d4c48ef8ac5864ccea25a5d2025-08-20T03:04:57ZengBMJ Publishing GroupJournal for ImmunoTherapy of Cancer2051-14262021-10-0191010.1136/jitc-2021-002955PET/CT scan and biopsy-driven approach for safe anti-PD-1 therapy discontinuation in patients with advanced melanomaMichael B. Atkins0Suthee Rapisuwon1Arash Radfar2Kellie Gardner3Neil J. Shah4Geoffrey T. Gibney5Jacob Zaemes6Shelly Shand7David Swoboda8Vesna Petronic-Rosic9Michael J. Reilly10Waddah B. Al-Refaie11Lombardi Comprehensive Cancer Center, MedStar Georgetown University Hospital, Washington, District of Columbia, USALombardi Comprehensive Cancer Center, MedStar Georgetown University Hospital, Washington, District of Columbia, USAInform Diagnostics, Irving, Texas, USAMedStar Georgetown University Hospital, Washington, District of Columbia, USAMemorial Sloan Kettering Cancer Center, New York, New York, USALombardi Comprehensive Cancer Center, MedStar Georgetown University Hospital, Washington, District of Columbia, USALombardi Comprehensive Cancer Center, MedStar Georgetown University Hospital, Washington, District of Columbia, USACharleston Oncology, Charleston, South Carolina, USAMoffitt Cancer Center, Tampa, Florida, USAGeorgetown University Medical Center, Washington, District of Columbia, USAMedStar Georgetown University Hospital, Washington, District of Columbia, USAMedStar Georgetown University Hospital, Washington, District of Columbia, USABackground Limited data exist on safe discontinuation of antiprogrammed cell death protein 1 (PD-1) therapy in responding patients with advanced melanoma. The use of 18fluorodeoxyglucose (18FDG)-PET/CT scan and tumor biopsy for assessment of active disease may be an effective predictive biomarker to guide such treatment decisions.Methods A retrospective study of 122 patients with advanced melanoma treated with anti-PD-1 monotherapy or anti-PD-1/anticytotoxic T-lymphocyte-associated protein 4 combination therapy at Georgetown Lombardi Comprehensive Cancer Center was conducted. Uveal melanoma patients and those receiving concurrent experimental therapy were excluded. Baseline characteristics, treatment outcomes, and survival were analyzed. Patients who decided to come off treatment typically after 12 months using CT scan radiographic complete response (CR), 18FDG-PET/CT scan complete metabolic response (CMR) or tumor biopsy of a non-CR/CMR tumor site negative for active disease (possible pathological CR) were identified and compared with patients who discontinued treatment due to toxicity while their disease was in control. Event-free survival (EFS) was assessed from the last dose of anti-PD-1 therapy to progression requiring subsequent treatment (surgery, radiation, and/or systemic therapy) or referral to hospice/death due to melanoma.Results 24 (20%) patients discontinued treatment by choice with no active disease and 28 (23%) patients discontinued treatment due to toxicity with disease control after 12-month and 4-month median treatment durations, respectively. Similar baseline characteristics were observed between cohorts except higher prior receipt of ipilimumab (29% vs 7%; p=0.036) and fewer BRAF mutant positive disease (17% vs 41%; p=0.064) in patients off treatment by choice. Three-year EFS rates were 95% and 71%, respectively. No significant associations between EFS and sex, disease stage, lactate dehydrogenase elevation, BRAF status, prior systemic therapy, ECOG performance status, presence of brain metastases, or combination versus monotherapy were observed. Tumor biopsies led to alternative management in 3/10 patients due to active metastatic melanoma or second malignancy.Conclusions Anti-PD-1 therapy discontinuation after 12 months when no active disease is observed on CT scan, PET/CT scan or tumor biopsy may have low rates of disease relapse in patients with advanced melanoma. Biopsy of residual disease may frequently lead to a change in management. These findings are undergoing validation in the EA6192 trial.https://jitc.bmj.com/content/9/10/e002955.full
spellingShingle Michael B. Atkins
Suthee Rapisuwon
Arash Radfar
Kellie Gardner
Neil J. Shah
Geoffrey T. Gibney
Jacob Zaemes
Shelly Shand
David Swoboda
Vesna Petronic-Rosic
Michael J. Reilly
Waddah B. Al-Refaie
PET/CT scan and biopsy-driven approach for safe anti-PD-1 therapy discontinuation in patients with advanced melanoma
Journal for ImmunoTherapy of Cancer
title PET/CT scan and biopsy-driven approach for safe anti-PD-1 therapy discontinuation in patients with advanced melanoma
title_full PET/CT scan and biopsy-driven approach for safe anti-PD-1 therapy discontinuation in patients with advanced melanoma
title_fullStr PET/CT scan and biopsy-driven approach for safe anti-PD-1 therapy discontinuation in patients with advanced melanoma
title_full_unstemmed PET/CT scan and biopsy-driven approach for safe anti-PD-1 therapy discontinuation in patients with advanced melanoma
title_short PET/CT scan and biopsy-driven approach for safe anti-PD-1 therapy discontinuation in patients with advanced melanoma
title_sort pet ct scan and biopsy driven approach for safe anti pd 1 therapy discontinuation in patients with advanced melanoma
url https://jitc.bmj.com/content/9/10/e002955.full
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