Is there a "trial effect" on outcome of patients with metastatic renal cell carcinoma treated with sunitinib?

Daniel Keizman, Keren Rouvinov, Avishay Sella, Maya Gottfried, Natalie Maimon, Jenny J. Kim, Mario Eisenberger, Victoria Sinibaldi, Avivit Peer, Michael A Carducci, Wilmosh Mermershtain, Raya Leibowitz-amit, Rony Weitzen, Raanan Berger

Research output: Contribution to journalArticle

Abstract

Purpose 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. Materials and 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), sex (male, 67%), Heng risk (favorable, 25%; intermediate, 59%; poor, 16%), prior nephrectomy (92%), RCC histology (clear cell 86%), pre-treatment neutrophil to lymphocyte ratio (> 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; odds ratio, 1.2). The median PFS was 10 versus 11 months (hazard ratio [HR], 0.96; p=0.84), and the median OS 23 versus 24 months (HR, 0.97; p=0.89). Conclusion In mRCC patients treated with sunitinib, the outcome of clinical trial participants was similar to that of non-participants who received standard therapy.

Original languageEnglish (US)
Pages (from-to)281-287
Number of pages7
JournalCancer Research and Treatment
Volume48
Issue number1
DOIs
StatePublished - 2016
Externally publishedYes

Fingerprint

Renal Cell Carcinoma
Clinical Trials
Disease-Free Survival
Survival
Therapeutics
Nephrectomy
sunitinib
Histology
Neutrophils
Research Design
Odds Ratio
Lymphocytes
Hypertension

Keywords

  • Clinical trial participation
  • Metastatic renal cell carcinoma
  • Outcome
  • Sunitinib

ASJC Scopus subject areas

  • Cancer Research
  • Oncology

Cite this

Keizman, D., Rouvinov, K., Sella, A., Gottfried, M., Maimon, N., Kim, J. J., ... Berger, R. (2016). Is there a "trial effect" on outcome of patients with metastatic renal cell carcinoma treated with sunitinib? Cancer Research and Treatment, 48(1), 281-287. https://doi.org/10.4143/crt.2014.289

Is there a "trial effect" on outcome of patients with metastatic renal cell carcinoma treated with sunitinib? / Keizman, Daniel; Rouvinov, Keren; Sella, Avishay; Gottfried, Maya; Maimon, Natalie; Kim, Jenny J.; Eisenberger, Mario; Sinibaldi, Victoria; Peer, Avivit; Carducci, Michael A; Mermershtain, Wilmosh; Leibowitz-amit, Raya; Weitzen, Rony; Berger, Raanan.

In: Cancer Research and Treatment, Vol. 48, No. 1, 2016, p. 281-287.

Research output: Contribution to journalArticle

Keizman, D, Rouvinov, K, Sella, A, Gottfried, M, Maimon, N, Kim, JJ, Eisenberger, M, Sinibaldi, V, Peer, A, Carducci, MA, Mermershtain, W, Leibowitz-amit, R, Weitzen, R & Berger, R 2016, 'Is there a "trial effect" on outcome of patients with metastatic renal cell carcinoma treated with sunitinib?', Cancer Research and Treatment, vol. 48, no. 1, pp. 281-287. https://doi.org/10.4143/crt.2014.289
Keizman, Daniel ; Rouvinov, Keren ; Sella, Avishay ; Gottfried, Maya ; Maimon, Natalie ; Kim, Jenny J. ; Eisenberger, Mario ; Sinibaldi, Victoria ; Peer, Avivit ; Carducci, Michael A ; Mermershtain, Wilmosh ; Leibowitz-amit, Raya ; Weitzen, Rony ; Berger, Raanan. / Is there a "trial effect" on outcome of patients with metastatic renal cell carcinoma treated with sunitinib?. In: Cancer Research and Treatment. 2016 ; Vol. 48, No. 1. pp. 281-287.
@article{e159da32514e4acc940e6d1f36b0caec,
title = "Is there a {"}trial effect{"} on outcome of patients with metastatic renal cell carcinoma treated with sunitinib?",
abstract = "Purpose 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. Materials and 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), sex (male, 67{\%}), Heng risk (favorable, 25{\%}; intermediate, 59{\%}; poor, 16{\%}), prior nephrectomy (92{\%}), RCC histology (clear cell 86{\%}), pre-treatment neutrophil to lymphocyte ratio (> 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; odds ratio, 1.2). The median PFS was 10 versus 11 months (hazard ratio [HR], 0.96; p=0.84), and the median OS 23 versus 24 months (HR, 0.97; p=0.89). Conclusion In mRCC patients treated with sunitinib, the outcome of clinical trial participants was similar to that of non-participants who received standard therapy.",
keywords = "Clinical trial participation, Metastatic renal cell carcinoma, Outcome, Sunitinib",
author = "Daniel Keizman and Keren Rouvinov and Avishay Sella and Maya Gottfried and Natalie Maimon and Kim, {Jenny J.} and Mario Eisenberger and Victoria Sinibaldi and Avivit Peer and Carducci, {Michael A} and Wilmosh Mermershtain and Raya Leibowitz-amit and Rony Weitzen and Raanan Berger",
year = "2016",
doi = "10.4143/crt.2014.289",
language = "English (US)",
volume = "48",
pages = "281--287",
journal = "Cancer Research and Treatment",
issn = "1598-2998",
publisher = "Korean Cancer Association",
number = "1",

}

TY - JOUR

T1 - Is there a "trial effect" on outcome of patients with metastatic renal cell carcinoma treated with sunitinib?

AU - Keizman, Daniel

AU - Rouvinov, Keren

AU - Sella, Avishay

AU - Gottfried, Maya

AU - Maimon, Natalie

AU - Kim, Jenny J.

AU - Eisenberger, Mario

AU - Sinibaldi, Victoria

AU - Peer, Avivit

AU - Carducci, Michael A

AU - Mermershtain, Wilmosh

AU - Leibowitz-amit, Raya

AU - Weitzen, Rony

AU - Berger, Raanan

PY - 2016

Y1 - 2016

N2 - Purpose 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. Materials and 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), sex (male, 67%), Heng risk (favorable, 25%; intermediate, 59%; poor, 16%), prior nephrectomy (92%), RCC histology (clear cell 86%), pre-treatment neutrophil to lymphocyte ratio (> 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; odds ratio, 1.2). The median PFS was 10 versus 11 months (hazard ratio [HR], 0.96; p=0.84), and the median OS 23 versus 24 months (HR, 0.97; p=0.89). Conclusion In mRCC patients treated with sunitinib, the outcome of clinical trial participants was similar to that of non-participants who received standard therapy.

AB - Purpose 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. Materials and 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), sex (male, 67%), Heng risk (favorable, 25%; intermediate, 59%; poor, 16%), prior nephrectomy (92%), RCC histology (clear cell 86%), pre-treatment neutrophil to lymphocyte ratio (> 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; odds ratio, 1.2). The median PFS was 10 versus 11 months (hazard ratio [HR], 0.96; p=0.84), and the median OS 23 versus 24 months (HR, 0.97; p=0.89). Conclusion In mRCC patients treated with sunitinib, the outcome of clinical trial participants was similar to that of non-participants who received standard therapy.

KW - Clinical trial participation

KW - Metastatic renal cell carcinoma

KW - Outcome

KW - Sunitinib

UR - http://www.scopus.com/inward/record.url?scp=84957566594&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84957566594&partnerID=8YFLogxK

U2 - 10.4143/crt.2014.289

DO - 10.4143/crt.2014.289

M3 - Article

C2 - 25761478

AN - SCOPUS:84957566594

VL - 48

SP - 281

EP - 287

JO - Cancer Research and Treatment

JF - Cancer Research and Treatment

SN - 1598-2998

IS - 1

ER -