Phase I and phase II objective response rates are correlated in pediatric cancer trials: An argument for better clinical trial efficiency

Jonathan C. Yeh, Peng Huang, Kenneth J Cohen

Research output: Contribution to journalArticle


Although many phase I trials report tumor response, formal analysis of efficacy is deferred to phase II.We reviewed paired phase I and II pediatric oncology trials to ascertain the relationship between phase I and II objective response rate (OR%). Single-agent phase I trials were paired with corresponding phase II trials (comparable study drug, dosing schedule, and population). Phase I trials without efficacy data or a matching phase II trial were excluded. OR% was tabulated for all trials, and phase II authors' subjective conclusions regarding efficacy were documented; 35 pairs of trials were analyzed. The correlation between phase I and II OR% was 0.93. Between phase II studies with a "positive" conclusion versus a "negative" one, there was a statistically significant difference in mean phase I OR% (32.0% vs. 4.5%, P

Original languageEnglish (US)
Pages (from-to)360-366
Number of pages7
JournalJournal of Pediatric Hematology/Oncology
Issue number5
StatePublished - 2016



  • Drug development
  • Pediatric oncology
  • Phase I
  • Phase II

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Oncology
  • Hematology

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