TY - JOUR
T1 - 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
AU - Bria, Emilio
AU - Massari, Francesco
AU - Maines, Francesca
AU - Pilotto, Sara
AU - Bonomi, Maria
AU - Porta, Camillo
AU - Bracarda, Sergio
AU - Heng, Daniel
AU - Santini, Daniele
AU - Sperduti, Isabella
AU - Giannarelli, Diana
AU - Cognetti, Francesco
AU - Tortora, Giampaolo
AU - Milella, Michele
PY - 2015/1/1
Y1 - 2015/1/1
N2 - 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.
AB - 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.
KW - Correlation
KW - Overall survival
KW - Progression-free survival
KW - Renal cell carcinoma
UR - http://www.scopus.com/inward/record.url?scp=84915752188&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84915752188&partnerID=8YFLogxK
U2 - 10.1016/j.critrevonc.2014.08.001
DO - 10.1016/j.critrevonc.2014.08.001
M3 - Article
C2 - 25195095
AN - SCOPUS:84915752188
VL - 93
SP - 50
EP - 59
JO - Critical Reviews in Oncology/Hematology
JF - Critical Reviews in Oncology/Hematology
SN - 1040-8428
IS - 1
ER -