Treatment of malignant peripheral nerve sheath tumors in pediatric NF1 disease

Enrico Martin, Uta E Flucke, J Henk Coert, Max M van Noesel

Onderzoeksoutput: Bijdrage aan tijdschriftArtikel recenserenpeer review

13 Citaten (Scopus)


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.

Originele taal-2Engels
Pagina's (van-tot)2453-2462
Aantal pagina's10
TijdschriftChild's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
Nummer van het tijdschrift10
StatusGepubliceerd - jun. 2020


Duik in de onderzoeksthema's van 'Treatment of malignant peripheral nerve sheath tumors in pediatric NF1 disease'. Samen vormen ze een unieke vingerafdruk.

Citeer dit