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Updated: Nov 2, 2025

Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
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Risk and Benefit for Targeted Therapy Agents in Pediatric Phase II Trials in Oncology: A Systematic Review with a

Karolina Strzebonska1, Mateusz T Wasylewski1, Lucja Zaborowska1

  • 1Research Ethics in Medicine Study Group (REMEDY), Department of Philosophy and Bioethics, Jagiellonian University Medical College, ul. Kopernika 40, 31-501, Kraków, Poland.

Targeted Oncology
|June 10, 2021
PubMed
Abstract

Insights

This study reviewed pediatric cancer trials, finding a 24.4% response rate but also a 1.6% fatal adverse event rate. These findings help assess risks and benefits for children in targeted therapy clinical trials.

Area of Science:

  • Pediatric Oncology
  • Clinical Pharmacology
  • Translational Medicine

Background:

  • Ethical research requires a favorable risk/benefit balance.
  • Limited data exists on risk/benefit for pediatric cancer phase II targeted therapy trials.

Purpose of the Study:

  • Systematically review preliminary efficacy and safety of phase II targeted therapy trials in pediatric oncology.
  • Provide an estimate of risks and benefits for participation in these trials.

Main Methods:

  • Systematic review of EMBASE and PubMed for phase II pediatric cancer trials (2015-2020).
  • Included solid and hematological malignancy studies.
  • Measured risk by grade 3+ adverse events; benefit by response rates. Meta-analysis performed where possible.

Main Results:

  • 34 trials (1202 patients) were included.
  • Pooled overall response rate: 24.4% (lower in solid tumors: 6.4% vs. hematological malignancies: 55.1%).
  • Fatal adverse event rate: 1.6%; average grade 3/4 adverse event rate: 0.66 per person.

Conclusions:

  • Provides an estimate of risks and benefits for pediatric phase II cancer trials.
  • Data can inform communication about participation burdens and benefits in pediatric oncology research.

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