Infantile/Congenital High-Grade Gliomas: Molecular Features and Therapeutic Perspectives

Giulia Ceglie1, Maria Vinci1, Andrea Carai2

  • 1Department of Onco-Hematology and Cell and Gene Therapy, Bambino Gesù Children's Hospital (IRCCS), Piazza Sant'Onofrio 4, 00146 Rome, Italy.

Insights

High-grade gliomas (HGGs) in infants are rare but aggressive brain tumors. Advances in molecular understanding, particularly gene fusions, offer new targeted therapy options to improve infant brain tumor treatment outcomes.

Area of Science:

  • Pediatric Oncology
  • Neuro-oncology
  • Molecular Pathology

Background:

  • Infant brain tumors represent less than 10% of pediatric nervous system tumors.
  • High-grade gliomas (HGGs) in infants present unique clinical challenges compared to adult or older pediatric counterparts.
  • Current treatment relies mainly on surgery, with limited roles for chemotherapy and radiotherapy due to potential long-term effects.

Purpose of the Study:

  • To review recent diagnostic and therapeutic advancements in infant high-grade gliomas.
  • To emphasize the significance of the molecular landscape in understanding and treating these tumors.
  • To explore future clinical applications of molecular findings in infant HGGs.

Main Methods:

  • Review of current literature on infant high-grade gliomas.
  • Focus on molecular characterization, including gene fusions and receptor tyrosine kinase mutations.
  • Analysis of emerging targeted therapies and their potential impact.

Main Results:

  • Molecular characterization of infant HGGs is crucial for understanding their unique biology.
  • Neurotrophic tyrosine receptor kinase (NTRK) gene fusions are identified in infant HGGs.
  • Targeted therapies, including those against NTRK fusions, show promise for improved outcomes.

Conclusions:

  • Molecular insights are transforming the therapeutic approach to infant high-grade gliomas.
  • Targeted therapies represent a paradigm shift, potentially improving long-term prognoses.
  • Further research into the molecular landscape will guide future clinical strategies for infant brain tumors.

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