New Frontiers in Therapy of Peripheral Nerve Sheath Tumors in Patients With Neurofibromatosis Type 1: Latest Evidence and Clinical Implications

Anticancer Res. 2020 Apr;40(4):1817-1831. doi: 10.21873/anticanres.14136.

Abstract

Almost all individuals with neurofibromatosis type 1 (NF1) develop peripheral nerve sheath tumors (PNSTs), mainly benign neurofibromas, however about 10% of PNSTs will undergo transformation to malignant peripheral nerve sheath tumors (MPNSTs). Surgical treatment of PNSTs has traditionally been regarded as a standard approach. The availability of new agents that target specific molecular pathways involved in the pathogenesis of PNST has led to a number of clinical trials, which resulted in increased chances for better survival and quality of life. This review presents the latest evidence and clinical implications for new therapies of PNSTs in patients with NF1 emphasizing the potential benefit from the use of Ras/MAPK pathway inhibitors, immunotherapy, chemotherapy or radiation therapy. We present evaluation of current knowledge on available treatment modalities.

Keywords: MEK inhibitors; MPNST; NF1; Peripheral nerve sheath tumor; neurofibromatosis type 1; plexiform neurofibroma; review.

Publication types

  • Review

MeSH terms

  • Humans
  • MAP Kinase Signaling System / drug effects
  • MAP Kinase Signaling System / genetics
  • Neoplasms / complications
  • Neoplasms / drug therapy*
  • Neoplasms / genetics
  • Neoplasms / pathology
  • Nerve Sheath Neoplasms / complications
  • Nerve Sheath Neoplasms / drug therapy*
  • Nerve Sheath Neoplasms / genetics
  • Nerve Sheath Neoplasms / pathology
  • Neurofibromatosis 1 / complications
  • Neurofibromatosis 1 / drug therapy*
  • Neurofibromatosis 1 / genetics
  • Neurofibromatosis 1 / pathology
  • Protein Kinase Inhibitors / therapeutic use*
  • Signal Transduction / drug effects
  • Tomography, X-Ray Computed
  • ras Proteins / antagonists & inhibitors
  • ras Proteins / genetics

Substances

  • Protein Kinase Inhibitors
  • ras Proteins