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Treatment of malignant peripheral nerve sheath tumors in pediatric NF1 disease

BACKGROUND: Malignant peripheral nerve sheath tumors (MPNSTs) are rare yet highly aggressive soft tissue sarcomas. Children with neurofibromatosis type 1 (NF1) have a 10% lifetime risk for development of MPNST. Prognosis remains poor and survival seems worse for NF1 patients. METHODS: This narrative...

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Autores principales: Martin, Enrico, Flucke, Uta E., Coert, J. Henk, van Noesel, Max M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7575473/
https://www.ncbi.nlm.nih.gov/pubmed/32494969
http://dx.doi.org/10.1007/s00381-020-04687-3
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author Martin, Enrico
Flucke, Uta E.
Coert, J. Henk
van Noesel, Max M.
author_facet Martin, Enrico
Flucke, Uta E.
Coert, J. Henk
van Noesel, Max M.
author_sort Martin, Enrico
collection PubMed
description BACKGROUND: Malignant peripheral nerve sheath tumors (MPNSTs) are rare yet highly aggressive soft tissue sarcomas. Children with neurofibromatosis type 1 (NF1) have a 10% lifetime risk for development of MPNST. Prognosis remains poor and survival seems worse for NF1 patients. METHODS: This narrative review highlights current practices and pitfalls in the management of MPNST in pediatric NF1 patients. RESULTS: Preoperative diagnostics can be challenging, but PET scans have shown to be useful tools. More recently, functional MRI holds promise as well. Surgery remains the mainstay treatment for these patients, but careful planning is needed to minimize postoperative morbidity. Functional reconstructions can play a role in improving functional status. Radiotherapy can be administered to enhance local control in selected cases, but care should be taken to minimize radiation effects as well as reduce the risk of secondary malignancies. The exact role of chemotherapy has yet to be determined. Reports on the efficacy of chemotherapy vary as some report lower effects in NF1 populations. Promisingly, survival seems to ameliorate in the last few decades and response rates of chemotherapy may increase in NF1 populations when administering it as part of standard of care. However, in metastasized disease, response rates remain poor. New systemic therapies are therefore desperately warranted and multiple trials are currently investigating the role of drugs. Targeted drugs are nevertheless not yet included in first line treatment. CONCLUSION: Both research and clinical efforts benefit from multidisciplinary approaches with international collaborations in this rare malignancy.
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spelling pubmed-75754732020-10-21 Treatment of malignant peripheral nerve sheath tumors in pediatric NF1 disease Martin, Enrico Flucke, Uta E. Coert, J. Henk van Noesel, Max M. Childs Nerv Syst Special Annual Issue BACKGROUND: Malignant peripheral nerve sheath tumors (MPNSTs) are rare yet highly aggressive soft tissue sarcomas. Children with neurofibromatosis type 1 (NF1) have a 10% lifetime risk for development of MPNST. Prognosis remains poor and survival seems worse for NF1 patients. METHODS: This narrative review highlights current practices and pitfalls in the management of MPNST in pediatric NF1 patients. RESULTS: Preoperative diagnostics can be challenging, but PET scans have shown to be useful tools. More recently, functional MRI holds promise as well. Surgery remains the mainstay treatment for these patients, but careful planning is needed to minimize postoperative morbidity. Functional reconstructions can play a role in improving functional status. Radiotherapy can be administered to enhance local control in selected cases, but care should be taken to minimize radiation effects as well as reduce the risk of secondary malignancies. The exact role of chemotherapy has yet to be determined. Reports on the efficacy of chemotherapy vary as some report lower effects in NF1 populations. Promisingly, survival seems to ameliorate in the last few decades and response rates of chemotherapy may increase in NF1 populations when administering it as part of standard of care. However, in metastasized disease, response rates remain poor. New systemic therapies are therefore desperately warranted and multiple trials are currently investigating the role of drugs. Targeted drugs are nevertheless not yet included in first line treatment. CONCLUSION: Both research and clinical efforts benefit from multidisciplinary approaches with international collaborations in this rare malignancy. Springer Berlin Heidelberg 2020-06-03 2020 /pmc/articles/PMC7575473/ /pubmed/32494969 http://dx.doi.org/10.1007/s00381-020-04687-3 Text en © The Author(s) 2020 Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/.
spellingShingle Special Annual Issue
Martin, Enrico
Flucke, Uta E.
Coert, J. Henk
van Noesel, Max M.
Treatment of malignant peripheral nerve sheath tumors in pediatric NF1 disease
title Treatment of malignant peripheral nerve sheath tumors in pediatric NF1 disease
title_full Treatment of malignant peripheral nerve sheath tumors in pediatric NF1 disease
title_fullStr Treatment of malignant peripheral nerve sheath tumors in pediatric NF1 disease
title_full_unstemmed Treatment of malignant peripheral nerve sheath tumors in pediatric NF1 disease
title_short Treatment of malignant peripheral nerve sheath tumors in pediatric NF1 disease
title_sort treatment of malignant peripheral nerve sheath tumors in pediatric nf1 disease
topic Special Annual Issue
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7575473/
https://www.ncbi.nlm.nih.gov/pubmed/32494969
http://dx.doi.org/10.1007/s00381-020-04687-3
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