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PA-CRM: A continuous reassessment method for pediatric phase I oncology trials with concurrent adult trials

Yimei Li1, Ying Yuan2

  • 1Department of Biostatistics, Epidemiology and Informatics, University of Pennsylvania & The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.

Biometrics
|January 18, 2020
PubMed

Insights

A new pediatric-continuous reassessment method (PA-CRM) improves pediatric phase I oncology trials. This method enhances efficiency and accuracy in determining the maximum tolerated dose by adaptively using adult and pediatric data.

Area of Science:

  • * Oncology
  • * Clinical Trials
  • * Biostatistics

Background:

  • * Pediatric phase I trials often commence during adult trials, necessitating protocol amendments based on interim adult data.
  • * Current amendment practices are often ad hoc, disrupting patient accrual and trial efficiency.
  • * Finding the maximum tolerated dose (MTD) in pediatric oncology trials is critical but challenging.

Purpose of the Study:

  • * To develop a streamlined and rigorous method for pediatric phase I oncology trials.
  • * To introduce the pediatric-continuous reassessment method (PA-CRM) for efficient dose escalation.
  • * To improve the determination of the maximum tolerated dose (MTD) in pediatric cancer patients.

Main Methods:

  • * Developed a novel pediatric-continuous reassessment method (PA-CRM).
  • * Utilized a discounted joint likelihood incorporating both adult and pediatric trial data.
  • * Employed Bayesian model averaging to adaptively update a discount parameter for information borrowing.

Main Results:

  • * The PA-CRM significantly improves the efficiency and accuracy of pediatric phase I trials.
  • * The method demonstrates robustness across various model assumptions.
  • * Streamlines the dose-finding process, reducing trial interruptions.

Conclusions:

  • * The PA-CRM offers a more efficient and rigorous approach to pediatric dose-finding studies.
  • * Adaptive information borrowing enhances the precision of MTD estimation.
  • * This method represents a significant advancement for pediatric oncology clinical trials.

Keywords:
Bayesian model averagingadaptive designdose findinginformation borrowingphase i trials; power prior

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