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Precision medicine in pediatric oncology

Suzanne J Forrest1, Birgit Geoerger2,3, Katherine A Janeway1

  • 1Pediatrics, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts, USA.

Current Opinion in Pediatrics
|December 1, 2017
PubMed
Abstract

Insights

Precision medicine for pediatric cancers shows promise, with clinical trials evaluating targeted therapies based on tumor profiling. These studies are crucial for advancing treatment options for childhood cancers.

Area of Science:

  • Oncology
  • Genetics
  • Pharmacology

Background:

  • Pediatric cancers present unique challenges for treatment.
  • Tumor profiling is increasingly used to identify molecular alterations.

Purpose of the Study:

  • To review advances and challenges in pediatric precision medicine.
  • To highlight clinical trials assessing targeted therapy based on molecular profiling.
  • To discuss precision medicine trial design in pediatric oncology.

Main Methods:

  • Review of recent prospective clinical sequencing studies in pediatric oncology.
  • Analysis of completed, ongoing, and developing precision medicine trials.
  • Focus on trial design aspects relevant to pediatric populations.

Main Results:

  • Demonstrated feasibility of clinical tumor sequencing in pediatric oncology.
  • Identified a significant rate of actionable variants in childhood cancers.
  • Several precision medicine trials are in various stages of development and completion.

Conclusions:

  • Results from early pediatric precision oncology trials will enhance understanding of targeted therapy efficacy.
  • Aggregated data will drive further discoveries and expand precision medicine opportunities for children with cancer.

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