Case Report: Lasting complete response to pembrolizumab in mismatch repair-deficient cardiac sarcoma: a genomic characterization

Sarcomas are traditionally considered “cold” tumors with poor response to immunotherapy. However, evidence accumulating over the last years shows that immune checkpoint inhibitors (ICIs) may have a role in selected sarcoma patients according to predictive markers. Here, we report the case of a woman...

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Main Authors: Daniela A. Ferraro, Bettina Bisig, David C. Rotzinger, Fresia Pareja, Edoardo Missiaglia, Ioannis Voutsadakis, Krisztian Homicsko, Antonia Digklia
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-04-01
Series:Frontiers in Oncology
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Online Access:https://www.frontiersin.org/articles/10.3389/fonc.2025.1485386/full
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author Daniela A. Ferraro
Bettina Bisig
Bettina Bisig
David C. Rotzinger
Fresia Pareja
Edoardo Missiaglia
Edoardo Missiaglia
Ioannis Voutsadakis
Ioannis Voutsadakis
Krisztian Homicsko
Krisztian Homicsko
Antonia Digklia
Antonia Digklia
author_facet Daniela A. Ferraro
Bettina Bisig
Bettina Bisig
David C. Rotzinger
Fresia Pareja
Edoardo Missiaglia
Edoardo Missiaglia
Ioannis Voutsadakis
Ioannis Voutsadakis
Krisztian Homicsko
Krisztian Homicsko
Antonia Digklia
Antonia Digklia
author_sort Daniela A. Ferraro
collection DOAJ
description Sarcomas are traditionally considered “cold” tumors with poor response to immunotherapy. However, evidence accumulating over the last years shows that immune checkpoint inhibitors (ICIs) may have a role in selected sarcoma patients according to predictive markers. Here, we report the case of a woman diagnosed with a primary cardiac undifferentiated sarcoma. Following failure of standard first line chemotherapy, high-throughput sequencing (HTS) revealed a high tumor mutational burden (TMB), pathogenic mutations in FAT1 and NOTCH2 and a microsatellite instability (MSI)-associated signature. Immunohistochemistry confirmed mismatch repair-deficiency (MMRd) and abundant CD8+ tumor-infiltrating lymphocytes (TILs), in the absence of tertiary lymphoid structures. The patient was, therefore, treated with the ICI pembrolizumab, reaching a complete response that continues to persist at last follow-up, more than seven years from initial diagnosis and nearly six years from initiation of ICI treatment. This case illustrates the importance of performing HTS in rare sarcomas given the availability of efficient therapies, such as those for tumors displaying high TMB or MMRd/MSI. In agreement with other reports, it supports the contention that MMRd/MSI status and high numbers of TILs are valuable predictive markers of response to immunotherapy in sarcomas.
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spelling doaj-art-58d020f23c6d4e80aad3cc8ccc58b9212025-08-20T03:03:06ZengFrontiers Media S.A.Frontiers in Oncology2234-943X2025-04-011510.3389/fonc.2025.14853861485386Case Report: Lasting complete response to pembrolizumab in mismatch repair-deficient cardiac sarcoma: a genomic characterizationDaniela A. Ferraro0Bettina Bisig1Bettina Bisig2David C. Rotzinger3Fresia Pareja4Edoardo Missiaglia5Edoardo Missiaglia6Ioannis Voutsadakis7Ioannis Voutsadakis8Krisztian Homicsko9Krisztian Homicsko10Antonia Digklia11Antonia Digklia12Department of Medical Oncology, CHUV University Hospital, Lausanne, SwitzerlandInstitute of Pathology, Department of Laboratory Medicine and Pathology, CHUV University Hospital, Lausanne, SwitzerlandFaculty of Biology and Medicine, University of Lausanne, Lausanne, SwitzerlandDepartment of Radiology, CHUV University Hospital, Lausanne, SwitzerlandDepartment of Pathology, Memorial Sloan Kettering Cancer Center, New York, NY, United StatesInstitute of Pathology, Department of Laboratory Medicine and Pathology, CHUV University Hospital, Lausanne, SwitzerlandFaculty of Biology and Medicine, University of Lausanne, Lausanne, SwitzerlandAlgoma District Cancer Program, Sault Area Hospital, Sault Ste. Marie, ON, CanadaDivision of Clinical Sciences, Section of Internal Medicine, Northern Ontario School of Medicine, Sudbury, ON, CanadaDepartment of Medical Oncology, CHUV University Hospital, Lausanne, SwitzerlandFaculty of Biology and Medicine, University of Lausanne, Lausanne, SwitzerlandDepartment of Medical Oncology, CHUV University Hospital, Lausanne, SwitzerlandFaculty of Biology and Medicine, University of Lausanne, Lausanne, SwitzerlandSarcomas are traditionally considered “cold” tumors with poor response to immunotherapy. However, evidence accumulating over the last years shows that immune checkpoint inhibitors (ICIs) may have a role in selected sarcoma patients according to predictive markers. Here, we report the case of a woman diagnosed with a primary cardiac undifferentiated sarcoma. Following failure of standard first line chemotherapy, high-throughput sequencing (HTS) revealed a high tumor mutational burden (TMB), pathogenic mutations in FAT1 and NOTCH2 and a microsatellite instability (MSI)-associated signature. Immunohistochemistry confirmed mismatch repair-deficiency (MMRd) and abundant CD8+ tumor-infiltrating lymphocytes (TILs), in the absence of tertiary lymphoid structures. The patient was, therefore, treated with the ICI pembrolizumab, reaching a complete response that continues to persist at last follow-up, more than seven years from initial diagnosis and nearly six years from initiation of ICI treatment. This case illustrates the importance of performing HTS in rare sarcomas given the availability of efficient therapies, such as those for tumors displaying high TMB or MMRd/MSI. In agreement with other reports, it supports the contention that MMRd/MSI status and high numbers of TILs are valuable predictive markers of response to immunotherapy in sarcomas.https://www.frontiersin.org/articles/10.3389/fonc.2025.1485386/fullcardiac sarcomaimmune-checkpoint inhibitors (ICI)complete response (CR)predictive markersmismatch repair deficiency (MMRd)microsatellite instability (MSI)
spellingShingle Daniela A. Ferraro
Bettina Bisig
Bettina Bisig
David C. Rotzinger
Fresia Pareja
Edoardo Missiaglia
Edoardo Missiaglia
Ioannis Voutsadakis
Ioannis Voutsadakis
Krisztian Homicsko
Krisztian Homicsko
Antonia Digklia
Antonia Digklia
Case Report: Lasting complete response to pembrolizumab in mismatch repair-deficient cardiac sarcoma: a genomic characterization
Frontiers in Oncology
cardiac sarcoma
immune-checkpoint inhibitors (ICI)
complete response (CR)
predictive markers
mismatch repair deficiency (MMRd)
microsatellite instability (MSI)
title Case Report: Lasting complete response to pembrolizumab in mismatch repair-deficient cardiac sarcoma: a genomic characterization
title_full Case Report: Lasting complete response to pembrolizumab in mismatch repair-deficient cardiac sarcoma: a genomic characterization
title_fullStr Case Report: Lasting complete response to pembrolizumab in mismatch repair-deficient cardiac sarcoma: a genomic characterization
title_full_unstemmed Case Report: Lasting complete response to pembrolizumab in mismatch repair-deficient cardiac sarcoma: a genomic characterization
title_short Case Report: Lasting complete response to pembrolizumab in mismatch repair-deficient cardiac sarcoma: a genomic characterization
title_sort case report lasting complete response to pembrolizumab in mismatch repair deficient cardiac sarcoma a genomic characterization
topic cardiac sarcoma
immune-checkpoint inhibitors (ICI)
complete response (CR)
predictive markers
mismatch repair deficiency (MMRd)
microsatellite instability (MSI)
url https://www.frontiersin.org/articles/10.3389/fonc.2025.1485386/full
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