Treatment of neurofibromatosis 1-associated malignant peripheral nerve sheath tumors: a systematic review

Neurosurg Rev. 2020 Aug;43(4):1039-1046. doi: 10.1007/s10143-019-01135-y. Epub 2019 Jun 17.


Malignant peripheral nerve sheath tumors (MPNST) are a rare and aggressive group of tumors that are challenging to treat. Neurofibromatosis type 1 (NF-1)-associated MPNSTs have been associated with poorer clinical outcomes. The treatment options for NF-1-associated MPNSTs broadly include surgery (SG), chemotherapy (CT), and adjuvant radiotherapy (RT). Overall, the role and efficacy of CT and RT are unclear. Examination of existing literature for studies reporting on NF-1-associated MPNSTs and respective treatment-related outcomes was conducted. We conducted a systematic review according to PRISMA guidelines in PubMed/Medline and Cochrane databases of studies which reported treatment-specific outcomes in NF-1-associated MPNSTs. The literature search found 444 records after removal of duplicates. The present study included 50 patients across 12 observational studies. All of the included studies reported data on overall survival (OS 52%, n = 26/50) but mean follow-up in months among the studies and among patients varied widely, between 10.85 (SD, ± 10.38) and 192 (SD, ± 98.22). From the included studies, patients underwent either SG alone (n = 21), SG + CT (n = 10), SG + RT (n = 7), or SG + CT + RT (n = 12). The quality of evidence in the literature regarding optimal treatment options for NF-1-associated MPNSTs remains tenuous. Future retrospective and prospective comparative trials should consider adherence to a set of reporting guidelines to improve the quality of evidence in the literature with respect to individual treatment-related outcomes. The need for prospective multi-institutional efforts cannot be overstated.

Keywords: Chemotherapy; MPNST; Malignant peripheral nerve sheath tumor; NF-1; Neurofibromatosis 1; Publishing guidelines; Radiotherapy.

Publication types

  • Systematic Review

MeSH terms

  • Humans
  • Nerve Sheath Neoplasms / etiology*
  • Nerve Sheath Neoplasms / surgery
  • Nerve Sheath Neoplasms / therapy*
  • Neurofibromatosis 1 / complications*
  • Neurofibromatosis 1 / therapy*
  • Neurosurgical Procedures