Pediatric brainstem gliomas: new understanding leads to potential new treatments for two very different tumors

Curr Oncol Rep. 2015 Mar;17(3):436. doi: 10.1007/s11912-014-0436-7.

Abstract

Pediatric brainstem gliomas include low-grade focal brainstem gliomas (FBSG) and high-grade diffuse intrinsic pontine gliomas (DIPG). These tumors share a crucial and eloquent area of the brain as their location, which carries common challenges for treatment. Otherwise, though, these two diseases are very different in terms of presentation, biology, treatment, and prognosis. FBSG usually present with greater than 3 months of symptoms, while DIPG are usually diagnosed within 3 months of symptom onset. Surgery remains the preferred initial treatment for FBSG, with chemotherapy used for persistent, recurrent, or inoperable disease; conversely, radiation is the only known effective treatment for DIPG. Recent developments in biological understanding of both tumors have led to new treatment possibilities. In FBSG, two genetic changes related to BRAF characterize the majority of tumors, and key differences in their biological effects are informing strategies for targeted chemotherapy use. In DIPG, widespread histone H3 and ACVR1 mutations have led to new hope for effective targeted treatments. FBSG has an excellent prognosis, while the long-term survival rate of DIPG tragically remains near zero. In this review, we cover the epidemiology, biology, presentation, imaging characteristics, multimodality treatment, and prognosis of FBSG and DIPG, with a focus on recent biological discoveries.

Publication types

  • Review

MeSH terms

  • Antineoplastic Agents / therapeutic use
  • Brain Stem Neoplasms / diagnosis
  • Brain Stem Neoplasms / therapy*
  • Chemoradiotherapy / methods
  • Child
  • Clinical Trials as Topic
  • Combined Modality Therapy
  • Glioma / diagnosis
  • Glioma / therapy*
  • Humans
  • Molecular Targeted Therapy / methods
  • Prognosis

Substances

  • Antineoplastic Agents