Sustained response to erlotinib and rapamycin in a patient with pediatric anaplastic oligodendroglioma

Pediatr Blood Cancer. 2021 Jan;68(1):e28750. doi: 10.1002/pbc.28750. Epub 2020 Oct 1.

Abstract

One goal of precision medicine is to identify mutations within individual tumors to design targeted treatment approaches. This report details the use of genomic testing to select a targeted therapy regimen of erlotinib and rapamycin for a pediatric anaplastic oligodendroglioma refractory to standard treatment, achieving a 33-month sustained response. Immunohistochemical analysis of total and phosphorylated protein isoforms showed abnormal signaling consistent with detected mutations, while revealing heterogeneity in per-cell activation of signaling pathways in multiple subpopulations of tumor cells throughout the course of disease. This case highlights molecular features that may be relevant to designing future targeted treatments.

Keywords: EGFR; brain tumor; mTOR; neuro-oncology; pediatric oncology; targeted therapy.

Publication types

  • Case Reports
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Child
  • Erlotinib Hydrochloride / administration & dosage
  • Female
  • Humans
  • Oligodendroglioma / drug therapy*
  • Prognosis
  • Remission Induction
  • Sirolimus / administration & dosage

Substances

  • Erlotinib Hydrochloride
  • Sirolimus