title: 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.
abstract: OBJECTIVE
  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.
authors.affiliation: Medical Oncology, Azienda Ospedaliera Universitaria Integrata, University of Verona, Verona, Italy. Electronic address: emiliobria@yahoo.it.
authors.forename: Emilio
authors.initials: E
authors.lastname: Bria
authors.affiliation: Medical Oncology, Azienda Ospedaliera Universitaria Integrata, University of Verona, Verona, Italy. Electronic address: fmassari79@gmail.com.
authors.forename: Francesco
authors.initials: F
authors.lastname: Massari
authors.affiliation: Medical Oncology, Azienda Ospedaliera Universitaria Integrata, University of Verona, Verona, Italy. Electronic address: francesca.maines@gmail.com.
authors.forename: Francesca
authors.initials: F
authors.lastname: Maines
authors.affiliation: Medical Oncology, Azienda Ospedaliera Universitaria Integrata, University of Verona, Verona, Italy. Electronic address: sara.pilotto.85@alice.it.
authors.forename: Sara
authors.initials: S
authors.lastname: Pilotto
authors.affiliation: Medical Oncology, Azienda Ospedaliera Universitaria Integrata, University of Verona, Verona, Italy. Electronic address: mari.bonomi0429@gmail.com.
authors.forename: Maria
authors.initials: M
authors.lastname: Bonomi
authors.affiliation: Medical Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy. Electronic address: c.porta@smatteo.pv.it.
authors.forename: Camillo
authors.initials: C
authors.lastname: Porta
authors.affiliation: Medical Oncology Ospedale San Donato, Istituto Toscano Tumori, Arezzo, Italy. Electronic address: sergio.bracarda@usl8.toscana.it.
authors.forename: Sergio
authors.initials: S
authors.lastname: Bracarda
authors.affiliation: Medical Oncology, University of Calgary, Tom Baker Cancer Center, Calgary, Alberta, Canada. Electronic address: daniel.heng@albertahealthservices.ca.
authors.forename: Daniel
authors.initials: D
authors.lastname: Heng
authors.affiliation: Medical Oncology, University Campus Bio-medico, Roma, Italy. Electronic address: d.santini@unicampus.it.
authors.forename: Daniele
authors.initials: D
authors.lastname: Santini
authors.affiliation: Biostatistics, Regina Elena National Cancer Institute, Roma, Italy. Electronic address: isperduti@yahoo.it.
authors.forename: Isabella
authors.initials: I
authors.lastname: Sperduti
authors.affiliation: Biostatistics, Regina Elena National Cancer Institute, Roma, Italy. Electronic address: giannarelli@ifo.it.
authors.forename: Diana
authors.initials: D
authors.lastname: Giannarelli
authors.affiliation: Medical Oncology, Regina Elena National Cancer Institute, Roma, Italy. Electronic address: cognetti@ifo.it.
authors.forename: Francesco
authors.initials: F
authors.lastname: Cognetti
authors.affiliation: Medical Oncology, Azienda Ospedaliera Universitaria Integrata, University of Verona, Verona, Italy. Electronic address: giampaolo.tortora@univr.it.
authors.forename: Giampaolo
authors.initials: G
authors.lastname: Tortora
authors.affiliation: Medical Oncology, Regina Elena National Cancer Institute, Roma, Italy.
authors.forename: Michele
authors.initials: M
authors.lastname: Milella
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D019985:Benchmarking / Q000379:methods*; D002292:Carcinoma, Renal Cell / Q000188:drug therapy* / Q000401:mortality; D018572:Disease-Free Survival; D006801:Humans; D007680:Kidney Neoplasms / Q000188:drug therapy* / Q000401:mortality; D058990:Molecular Targeted Therapy; D016032:Randomized Controlled Trials as Topic / Q000592:standards*
chemical_list: D000970:Antineoplastic Agents
keywords: Correlation; Overall survival; Progression-free survival; Renal cell carcinoma