title: Is there a "Trial Effect" on Outcome of Patients with Metastatic Renal Cell Carcinoma Treated with Sunitinib?
abstract: OBJECTIVE
  Studies suggested the existence of a 'trial effect', in which for a given treatment, participation in a clinical trial is associated with a better outcome. Sunitinib is a standard treatment for metastatic renal cell carcinoma (mRCC). We aimed to study the effect of clinical trial participation on the outcome of mRCC patients treated with sunitinib, which at present, is poorly defined.
  
  
  METHODS
  The records of mRCC patients treated with sunitinib between 2004-2013 in 7 centers across 2 countries were reviewed. We compared the response rate (RR), progression free survival (PFS), and overall survival (OS), between clinical trial participants (n=49) and a matched cohort of non-participants (n=49) who received standard therapy. Each clinical trial participant was individually matched with a non-participant by clinicopathologic factors. PFS and OS were determined by Cox regression.
  
  
  RESULTS
  The groups were matched by age (median 64), gender (male 67%), Heng risk (favorable 25%, intermediate 59%, poor 16%), prior nephrectomy (92%), RCC histology (clear cell 86%), pre-treatment NLR (>3 in 55%, n=27), sunitinib induced hypertension (45%), and sunitinib dose reduction/treatment interruption (41%). In clinical trial participants versus non-participants, RR was partial response/stable disease 80% (n=39) versus 74% (n=36), and progressive disease 20% (n=10) versus 26% (n=13) (p=0.63, OR 1.2). The median PFS was 10 versus 11 months (HR=0.96, p=0.84), and the median OS 23 versus 24 months (HR=0.97, p=0.89).
  
  
  CONCLUSIONS
  In mRCC patients treated with sunitinib, the outcome of clinical trial participants was similar to that of non-participants who received standard therapy.
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Daniel
authors.initials: D
authors.lastname: Keizman
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Keren
authors.initials: K
authors.lastname: Rouvinov
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Avishay
authors.initials: A
authors.lastname: Sella
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Maya
authors.initials: M
authors.lastname: Gottfried
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Natalie
authors.initials: N
authors.lastname: Maimon
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Jenny J
authors.initials: JJ
authors.lastname: Kim
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Mario A
authors.initials: MA
authors.lastname: Eisenberger
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Victoria
authors.initials: V
authors.lastname: Sinibaldi
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Avivit
authors.initials: A
authors.lastname: Peer
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Michael A
authors.initials: MA
authors.lastname: Carducci
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Wilmosh
authors.initials: W
authors.lastname: Mermershtain
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Raya
authors.initials: R
authors.lastname: Leibowitz-Amit
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Rony
authors.initials: R
authors.lastname: Weitzen
authors.affiliation: Genitourinary Oncology Service, Institute of Oncology, Meir Medical Center, Sackler School of Medicine, Tel Aviv University, Kfar Saba, Israel.
authors.forename: Raanan
authors.initials: R
authors.lastname: Berger
mesh_terms: D000328:Adult; D000368:Aged; D020533:Angiogenesis Inhibitors / Q000627:therapeutic use; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D016477:Artifacts; D002292:Carcinoma, Renal Cell / Q000188:drug therapy* / Q000523:psychology; D002986:Clinical Trials as Topic / Q000523:psychology*; D018450:Disease Progression; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007211:Indoles / Q000627:therapeutic use*; D007680:Kidney Neoplasms / Q000188:drug therapy* / Q000523:psychology; D008297:Male; D008875:Middle Aged; D011758:Pyrroles / Q000627:therapeutic use*; D015203:Reproducibility of Results; D012189:Retrospective Studies; D000077210:Sunitinib; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D020533:Angiogenesis Inhibitors; D000970:Antineoplastic Agents; D007211:Indoles; D011758:Pyrroles; D000077210:Sunitinib
keywords: Clinical trial participation; Metastatic renal cell carcinoma; Outcome; Sunitinib