PURPOSE: 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.
Written by:
Bria E, Massari F, Maines F, Pilotto S, Bonomi M, Porta C, Bracarda S, Heng D, Santini D, Sperduti I, Giannarelli D, Cognetti F, Tortora G, Milella M. Are you the author?
Medical Oncology, Azienda Ospedaliera Universitaria Integrata, University of Verona, Verona, Italy; Medical Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy; Medical Oncology Ospedale San Donato, Istituto Toscano Tumori, Arezzo, Italy; Medical Oncology, University of Calgary, Tom Baker Cancer Center, Calgary, Alberta, Canada; Medical Oncology, University Campus Bio-medico, Roma, Italy; Biostatistics, Regina Elena National Cancer Institute, Roma, Italy; Medical Oncology, Regina Elena National Cancer Institute, Roma, Italy. ; ; ; ; ; ; ; ; ; ; ; francesca.maines@gmail;
Reference: Crit Rev Oncol Hematol. 2014 Aug 18. pii: S1040-8428(14)00133-4.
doi: 10.1016/j.critrevonc.2014.08.001
PubMed Abstract
PMID: 25195095