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Clinical Utility of Precision Medicine in Pediatric Oncology: A Systematic Review

Justin Lee1, Lynn Gillam2,3,4, Keshini Visvanathan1

  • 1Children's Cancer Centre, Royal Children's Hospital, Parkville, VIC, Australia.

JCO Precision Oncology
|January 7, 2022
PubMed
Abstract

Insights

Precision medicine shows feasibility in identifying molecular targets in pediatric cancer. However, limited drug access restricts its therapeutic potential, necessitating better drug availability and standardized reporting for future trials.

Area of Science:

  • Pediatric Oncology
  • Precision Medicine
  • Molecular Diagnostics

Background:

  • Precision medicine, utilizing advanced molecular techniques, is an emerging approach in pediatric oncology.
  • Limited clinical evidence exists for novel targeted drugs in children due to rare cancer types and sparse data.
  • Evaluating the feasibility and efficacy of precision medicine in pediatric oncology is crucial.

Purpose of the Study:

  • To systematically review the existing evidence on the feasibility and clinical efficacy of precision medicine in pediatric oncology.
  • To assess the identification of molecular targets and subsequent use of targeted therapies in pediatric cancer patients.
  • To identify barriers and facilitators for implementing precision medicine in this population.

Main Methods:

  • A systematic review of PubMed, Medline, and Embase databases was performed.
  • Included studies were clinical trials and observational studies using molecular assays (e.g., whole-exome sequencing) to guide targeted cancer drug allocation.
  • Studies reporting clinical outcomes were prioritized for analysis.

Main Results:

  • Twenty-one studies involving 1,408 pediatric patients were analyzed.
  • Molecular targets were identified in 46.0% of patients, but only 27.0% received a targeted drug.
  • Objective response rates were 41.7% among those treated, representing 5.2% of the total sample, with inconsistent outcome reporting.

Conclusions:

  • Precision medicine can feasibly identify molecular targets in pediatric cancer patients.
  • Inaccessibility of targeted drugs is a major barrier to realizing the therapeutic potential of precision medicine in pediatric oncology.
  • Future trials must link molecular testing with drug access and standardize outcome reporting to advance this paradigm.

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