Recent Advances in the Pharmacotherapy of Peripheral Nervous System Tumors

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Pharmacology".

Deadline for manuscript submissions: closed (3 April 2023) | Viewed by 3723

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Guest Editor
Department of Neurosurgery, Neuro-Oncological Surgery Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, 20133 Milan, Italy
Interests: PNS tumors; intraoperative fluorescence; vascular malformations
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Guest Editor
Molecular Neuro-Oncology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, 20133 Milan, Italy
Interests: familial tumor syndromes; immunotherapy; nervous system tumors

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Guest Editor
Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, 20133 Milan, Italy
Interests: neurocutaneous diseases; genetic syndromes; Nervous System tumors

Special Issue Information

Dear Colleagues,

The management of peripheral nervous system (PNS) tumors has traditionally been reserved for surgical treatment. However, not all PNS tumors are suitable for surgery due to the possible morbidity or the limited role in the case of multiple tumors. This is particularly true for neurocutaneous disease-related lesions, such as plexiform neurofibromas or malignant peripheral nerve sheath tumors (MPNSTs), in particular considering the significant limitations to conventional therapeutic options in general for such tumors. During the last years, the peculiar clinical behavior of such tumors, along with the risk of developing multiple tumors over time, has shifted the focus to the need for prevention and for symptomatic treatment in recurrent or inoperable forms. Recently, the availability of oral drugs acting as MEK inhibitors for patients with plexiform neurofibromas and neurofibromatosis type 1 has substantiated a significant advance in patients’ care. Novel drugs are also available for MPNSTs. However, no significant progress has been made for patients with either neurofibromatosis type 2 or schwannomatosis, despite the availability of anti-angiogenetic drugs for vestibular schwannomas in NF2. Despite these premises, the need for more tumor-directed therapies with tolerable short- and long-term toxicities is needed, considering the complexity of the signaling pathways involved. The journal Pharmaceuticals invites both reviews and original articles shedding light on the challenges related to pharmacological management of all types of PNS tumors, both sporadic and occurring in familial tumor syndromes.The topics of this Special Issue include, but are not limited to: ongoing and concluded clinical trials, drug repositioning, optimization of side effects, molecular pathways and new possible targets, pharmaceuticals applications, and pharmacogenomic and drug responses. 

Dr. Ignazio Gaspare Vetrano
Dr. Marica Eoli
Dr. Veronica Saletti
Guest Editors

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Keywords

  • neurofibromatosis
  • MEK inhibition
  • MPNST
  • NF1
  • NF2
  • RAShopaties
  • plexiform neurofibromas
  • schwannomatosis
  • selumetinib

Published Papers (1 paper)

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12 pages, 1956 KiB  
Systematic Review
Efficacy and Safety of Trametinib in Neurofibromatosis Type 1-Associated Plexiform Neurofibroma and Low-Grade Glioma: A Systematic Review and Meta-Analysis
by Dun Wang, Lingling Ge, Zizhen Guo, Yuehua Li, Beiyao Zhu, Wei Wang, Chengjiang Wei, Qingfeng Li and Zhichao Wang
Pharmaceuticals 2022, 15(8), 956; https://doi.org/10.3390/ph15080956 - 31 Jul 2022
Cited by 7 | Viewed by 3009
Abstract
Trametinib has been used in neurofibromatosis type 1 (NF1) patients, especially those with unresectable nerve tumors, but no systematic review based on the latest studies has been published. We conducted this meta-analysis to evaluate the effectiveness and safety of trametinib in treating NF1-related [...] Read more.
Trametinib has been used in neurofibromatosis type 1 (NF1) patients, especially those with unresectable nerve tumors, but no systematic review based on the latest studies has been published. We conducted this meta-analysis to evaluate the effectiveness and safety of trametinib in treating NF1-related nerve tumors. Original articles reporting the efficacy and safety of trametinib in NF1 patents were identified in PubMed, EMBASE, and Web of Science up to 1 June 2022. Using R software and the ‘meta’ package, the objective response rates (ORRs) and disease control rates (DCRs) were calculated to evaluate the efficacy, and the pooled proportion of adverse events (AEs) was calculated. The Grading of Recommendations, Assessment, Development and Evaluation system was used to assess the quality of evidence. Eight studies involving 92 patients were included, which had a very low to moderate quality of evidence. The pooled ORR was 45.3% (95% CI: 28.9–62.1%, I2 = 0%), and the DCR was 99.8% (95% CI: 95.5–100%, I2 = 0%). The most common AEs was paronychia, with a pooled rate of 60.7% (95% CI: 48.8–72.7%, I2 = 0%). Our results indicate the satisfactory ability to stabilize tumor progression but a more limited ability to shrink tumors of trametinib in NF1-related nerve tumors. The safety profile of trametinib is satisfactory. Full article
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