Treatment of malignant peripheral nerve sheath tumors in pediatric NF1 disease
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SPECIAL ANNUAL ISSUE
Treatment of malignant peripheral nerve sheath tumors in pediatric NF1 disease Enrico Martin 1
&
Uta E. Flucke 2,3
&
J. Henk Coert 1
&
Max M. van Noesel 2
Received: 30 April 2020 / Accepted: 14 May 2020 # The Author(s) 2020
Abstract 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. Keywords Emerging therapies . MPNST . NF1 . Radiotherapy . Surgical treatment . Systemic treatment
Introduction Children with neurofibromatosis type I (NF1) are at an increased risk of developing multiple tumors including neurofibromas as a result of neurofibromin inhibition involved in the Ras pathway [1]. NF1 disease is a frequent genetic disease, affecting 1 in 3000 to 5000 people. The cumulative life time risk of developing malignant peripheral nerve sheath tumors
* Enrico Martin [email protected] 1
Department of Plastic and Reconstructive Surgery, University Medical Center Utrecht, G04.126, PO Box 85060, 3508, AB Utrecht, the Netherlands
2
Department of Solid Tumors, Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands
3
Department of Pathology, Radboud University Medical Center, Nijmegen, the Netherlands
(MPNST) is 8–13% [2–5]. MPNSTs are rare in the general population encompassing only 2–4% of all soft tissue sarcomas [6]. Less than 10% of those patients are children [7, 8], but MPNST is the third most common nonrhabdomyosarcoma STS (NRSTS) in children [9]. Of all MP
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