Current and Future Treatment Strategies for Rhabdomyosarcoma
Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children, and can be subcategorized histologically and/or based on PAX-FOXO1 fusion gene status. Over the last four decades, there have been no significant improvements in clinical outcomes for advanced and metastatic RMS patients, und...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2019-12-01
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| Series: | Frontiers in Oncology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/article/10.3389/fonc.2019.01458/full |
| _version_ | 1811295142727909376 |
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| author | Celine Chen Heathcliff Dorado Garcia Monika Scheer Anton G. Henssen Anton G. Henssen Anton G. Henssen Anton G. Henssen |
| author_facet | Celine Chen Heathcliff Dorado Garcia Monika Scheer Anton G. Henssen Anton G. Henssen Anton G. Henssen Anton G. Henssen |
| author_sort | Celine Chen |
| collection | DOAJ |
| description | Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children, and can be subcategorized histologically and/or based on PAX-FOXO1 fusion gene status. Over the last four decades, there have been no significant improvements in clinical outcomes for advanced and metastatic RMS patients, underscoring a need for new treatment options for these groups. Despite significant advancements in our understanding of the genomic landscape and underlying biological mechanisms governing RMS that have informed the identification of novel therapeutic targets, development of these therapies in clinical trials has lagged far behind. In this review, we summarize the current frontline multi-modality therapy for RMS according to pediatric protocols, highlight emerging targeted therapies and immunotherapies identified by preclinical studies, and discuss early clinical trial data and the implications they hold for future clinical development. |
| first_indexed | 2024-04-13T05:28:59Z |
| format | Article |
| id | doaj.art-025b8e17eb43450599d80e915a9839b6 |
| institution | Directory Open Access Journal |
| issn | 2234-943X |
| language | English |
| last_indexed | 2024-04-13T05:28:59Z |
| publishDate | 2019-12-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Oncology |
| spelling | doaj.art-025b8e17eb43450599d80e915a9839b62022-12-22T03:00:30ZengFrontiers Media S.A.Frontiers in Oncology2234-943X2019-12-01910.3389/fonc.2019.01458502837Current and Future Treatment Strategies for RhabdomyosarcomaCeline Chen0Heathcliff Dorado Garcia1Monika Scheer2Anton G. Henssen3Anton G. Henssen4Anton G. Henssen5Anton G. Henssen6Department of Pediatric Oncology/Hematology, Charité-Universitätsmedizin Berlin, Berlin, GermanyDepartment of Pediatric Oncology/Hematology, Charité-Universitätsmedizin Berlin, Berlin, GermanyPediatrics 5, Klinikum Stuttgart, Olgahospital, Stuttgart, GermanyDepartment of Pediatric Oncology/Hematology, Charité-Universitätsmedizin Berlin, Berlin, GermanyBerlin Institute of Health, Berlin, GermanyGerman Cancer Consortium (DKTK), Partner Site Berlin, German Cancer Research Center (DKFZ), Heidelberg, GermanyExperimental and Clinical Research Center (ECRC) of the MDC and Charité Berlin, Berlin, GermanyRhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children, and can be subcategorized histologically and/or based on PAX-FOXO1 fusion gene status. Over the last four decades, there have been no significant improvements in clinical outcomes for advanced and metastatic RMS patients, underscoring a need for new treatment options for these groups. Despite significant advancements in our understanding of the genomic landscape and underlying biological mechanisms governing RMS that have informed the identification of novel therapeutic targets, development of these therapies in clinical trials has lagged far behind. In this review, we summarize the current frontline multi-modality therapy for RMS according to pediatric protocols, highlight emerging targeted therapies and immunotherapies identified by preclinical studies, and discuss early clinical trial data and the implications they hold for future clinical development.https://www.frontiersin.org/article/10.3389/fonc.2019.01458/fullrhabdomyosacomapediatric oncologysoft tissue sarcomatargeted therapychildhood cancer |
| spellingShingle | Celine Chen Heathcliff Dorado Garcia Monika Scheer Anton G. Henssen Anton G. Henssen Anton G. Henssen Anton G. Henssen Current and Future Treatment Strategies for Rhabdomyosarcoma Frontiers in Oncology rhabdomyosacoma pediatric oncology soft tissue sarcoma targeted therapy childhood cancer |
| title | Current and Future Treatment Strategies for Rhabdomyosarcoma |
| title_full | Current and Future Treatment Strategies for Rhabdomyosarcoma |
| title_fullStr | Current and Future Treatment Strategies for Rhabdomyosarcoma |
| title_full_unstemmed | Current and Future Treatment Strategies for Rhabdomyosarcoma |
| title_short | Current and Future Treatment Strategies for Rhabdomyosarcoma |
| title_sort | current and future treatment strategies for rhabdomyosarcoma |
| topic | rhabdomyosacoma pediatric oncology soft tissue sarcoma targeted therapy childhood cancer |
| url | https://www.frontiersin.org/article/10.3389/fonc.2019.01458/full |
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