Novel "T-Dimension" Therapies for Pediatric Optic Pathway Glioma: A Timely, Targeted, and Tailored Treatment Trend

Pediatr Neurosurg. 2022;57(3):161-174. doi: 10.1159/000524873. Epub 2022 May 4.


Introduction: Novel targeted and tailored therapies can substantially improve the prognosis for optic pathway glioma (OPG), especially when implemented in a timely manner. However, their tremendous potential remains underestimated. Therefore, in this study, we provide an updated overview of the clinical trials, current trends, and future perspectives for OPG's novel therapeutic strategies.

Methods: We completed an extensive literature review using the PubMed, MEDLINE, and databases. We analyzed and reported the data following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines.

Results: Thioguanine, procarbazine, lomustine, and vincristine/vinblastine, as well as cisplatin-etoposide, provided excellent results in advanced-phase trials. Selumetinib and trametinib, two oral MEK inhibitors, have been approved for recurrent or refractory OPGs in association with the angiogenetic inhibitor bevacizumab. Among the mTOR inhibitors, everolimus and sirolimus showed the best results. Stereotactic radiosurgery and proton beam radiation therapy have advantages over conventional radiotherapy regimens. Timely treatment is imperative for acute visual symptoms with evidence of tumor progression. This latest evidence can help define a novel "T-Dimension" for pediatric OPG therapies.

Conclusion: The novel "T-Dimension" for pediatric OPGs is based on recent evidence-based treatments, including combination chemotherapy regimens, molecular targeted therapies, stereotactic radiosurgery, and proton beam radiation therapy. Additional clinical trials are essential for validating each of these new therapies.

Keywords: Bevacizumab; MEK inhibitors; Optic pathway glioma; Proton beam radiotherapy; Stereotactic radiosurgery; Target therapy; mTOR inhibitors.

Publication types

  • Systematic Review

MeSH terms

  • Child
  • Combined Modality Therapy
  • Glioma* / drug therapy
  • Humans
  • Lomustine / therapeutic use
  • Radiosurgery*
  • Vincristine / therapeutic use


  • Vincristine
  • Lomustine