Progression-free survival as primary endpoint in randomized clinical trials of targeted agents for advanced renal cell carcinoma. Correlation with overall survival, benchmarking and power analysis

Emilio Bria, Francesco Massari, Francesca Maines, Sara Pilotto, Maria Bonomi, Camillo Porta, Sergio Bracarda, Daniel Heng, Daniele Santini, Isabella Sperduti, Diana Giannarelli, Francesco Cognetti, Giampaolo Tortora, Michele Milella

Research output: Contribution to journalArticlepeer-review

Abstract

A correlation, power and benchmarking analysis between progression-free and overall survival (PFS, OS) of randomized trials with targeted agents or immunotherapy for advanced renal cell carcinoma (RCC) was performed to provide a practical tool for clinical trial design. Results: For 1st-line of treatment, a significant correlation was observed between 6-month PFS and 12-month OS, between 3-month PFS and 9-month OS and between the distributions of the cumulative PFS and OS estimates. According to the regression equation derived for 1st-line targeted agents, 7859, 2873, 712, and 190 patients would be required to determine a 3%, 5%, 10% and 20% PFS advantage at 6 months, corresponding to an absolute increase in 12-month OS rates of 2%, 3%, 6% and 11%, respectively. Conclusions: These data support PFS as a reliable endpoint for advanced RCC receiving up-front therapies. Benchmarking and power analyses, on the basis of the updated survival expectations, may represent practical tools for future trial' design.

Original languageEnglish
Pages (from-to)50-59
Number of pages10
JournalCritical Reviews in Oncology/Hematology
Volume93
Issue number1
DOIs
Publication statusPublished - Jan 1 2015

Keywords

  • Correlation
  • Overall survival
  • Progression-free survival
  • Renal cell carcinoma

ASJC Scopus subject areas

  • Oncology
  • Hematology
  • Geriatrics and Gerontology
  • Medicine(all)

Fingerprint Dive into the research topics of 'Progression-free survival as primary endpoint in randomized clinical trials of targeted agents for advanced renal cell carcinoma. Correlation with overall survival, benchmarking and power analysis'. Together they form a unique fingerprint.

Cite this