Differences in Trial and Real-world Populations in the Dutch Castration-resistant Prostate Cancer Registry

Hans M. Westgeest, Carin A. Uyl-de Groot, Reindert J.A. van Moorselaar, Ronald de Wit, Alphonsus C.M. van den Bergh, Jules L.L.M. Coenen, Harrie P. Beerlage, Mathijs P. Hendriks, Monique M.E.M. Bos, Pieter van den Berg, Agnes J. van de Wouw, Roan Spermon, Michiel O. Boerma, Maud M. Geenen, Lidwine W. Tick, Marco B. Polee, Haiko J. Bloemendal, Igor Cordia, Frank P.J. Peters, Aad I. de Vos & 3 others Joan van den Bosch, Alphonsus J.M. van den Eertwegh, Winald R. Gerritsen

Research output: Contribution to journalArticleAcademicpeer-review

Abstract

Background: Trials in castration-resistant prostate cancer (CRPC) treatment have shown improved outcomes, including survival. However, as trial populations are selected, results may not be representative for the real-world population. The aim of this study was to assess the differences between patients treated in a clinical trial versus standard care during the course of CRPC in a real-world CRPC population. Design, setting, and participants: Castration-resistant Prostate Cancer Registry is a population-based, observational, retrospective registry. CRPC patients from 20 hospitals in the Netherlands have been included from 2010 to 2013. Outcome measurements and statistical analysis: Baseline characteristics, systemic treatment, and overall survival were the main outcomes. Descriptive statistics, multivariate Cox regression, and multiple imputations with the Monte Carlo Markov Chain method were used. Results and limitations: In total, 1524 patients were enrolled of which 203 patients had participated in trials at any time. The median follow-up period was 23 mo. Patients in the trial group were significantly younger and had less comorbidities. Docetaxel treatment was more frequently used in trial patients (85% vs 40%). Despite an observed unadjusted median overall survival difference of 35 mo versus 24 mo between the trial and standard care group, this difference was not retained after adjustment for baseline characteristics and treatment effect. Conclusions: At CRPC diagnosis, the baseline characteristics of the patients who had been enrolled in trials notably differed from patients who received standard treatment options only. The survival difference between the trial and standard care group could be explained by baseline differences and treatment effects. These results indicate that trial results cannot easily be translated to real-world practice. Patient summary: We observed that patients treated in clinical trials differed from patients who were not. We concluded that this may lead to differential treatment and survival. Caution is warranted when real-world outcomes are compared with trial results. We observed that castration-resistant prostate cancer patients treated in clinical trials differ from patients who are not. We conclude that this may lead to differential treatment and survival. This warrants caution when comparing real-world outcomes to trial results.

Original languageEnglish
Pages (from-to)694-701
Number of pages8
JournalEuropean Urology Focus
Volume4
Issue number5
DOIs
Publication statusPublished - 1 Sep 2018

Cite this

Westgeest, Hans M. ; Uyl-de Groot, Carin A. ; van Moorselaar, Reindert J.A. ; de Wit, Ronald ; van den Bergh, Alphonsus C.M. ; Coenen, Jules L.L.M. ; Beerlage, Harrie P. ; Hendriks, Mathijs P. ; Bos, Monique M.E.M. ; van den Berg, Pieter ; van de Wouw, Agnes J. ; Spermon, Roan ; Boerma, Michiel O. ; Geenen, Maud M. ; Tick, Lidwine W. ; Polee, Marco B. ; Bloemendal, Haiko J. ; Cordia, Igor ; Peters, Frank P.J. ; de Vos, Aad I. ; van den Bosch, Joan ; van den Eertwegh, Alphonsus J.M. ; Gerritsen, Winald R. / Differences in Trial and Real-world Populations in the Dutch Castration-resistant Prostate Cancer Registry. In: European Urology Focus. 2018 ; Vol. 4, No. 5. pp. 694-701.
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title = "Differences in Trial and Real-world Populations in the Dutch Castration-resistant Prostate Cancer Registry",
abstract = "Background: Trials in castration-resistant prostate cancer (CRPC) treatment have shown improved outcomes, including survival. However, as trial populations are selected, results may not be representative for the real-world population. The aim of this study was to assess the differences between patients treated in a clinical trial versus standard care during the course of CRPC in a real-world CRPC population. Design, setting, and participants: Castration-resistant Prostate Cancer Registry is a population-based, observational, retrospective registry. CRPC patients from 20 hospitals in the Netherlands have been included from 2010 to 2013. Outcome measurements and statistical analysis: Baseline characteristics, systemic treatment, and overall survival were the main outcomes. Descriptive statistics, multivariate Cox regression, and multiple imputations with the Monte Carlo Markov Chain method were used. Results and limitations: In total, 1524 patients were enrolled of which 203 patients had participated in trials at any time. The median follow-up period was 23 mo. Patients in the trial group were significantly younger and had less comorbidities. Docetaxel treatment was more frequently used in trial patients (85{\%} vs 40{\%}). Despite an observed unadjusted median overall survival difference of 35 mo versus 24 mo between the trial and standard care group, this difference was not retained after adjustment for baseline characteristics and treatment effect. Conclusions: At CRPC diagnosis, the baseline characteristics of the patients who had been enrolled in trials notably differed from patients who received standard treatment options only. The survival difference between the trial and standard care group could be explained by baseline differences and treatment effects. These results indicate that trial results cannot easily be translated to real-world practice. Patient summary: We observed that patients treated in clinical trials differed from patients who were not. We concluded that this may lead to differential treatment and survival. Caution is warranted when real-world outcomes are compared with trial results. We observed that castration-resistant prostate cancer patients treated in clinical trials differ from patients who are not. We conclude that this may lead to differential treatment and survival. This warrants caution when comparing real-world outcomes to trial results.",
keywords = "Castration-resistant prostate cancer, Docetaxel, Outcomes research, Population based, Real-world outcomes, Registry, Registry of outcomes, Treatment, Trial population",
author = "Westgeest, {Hans M.} and {Uyl-de Groot}, {Carin A.} and {van Moorselaar}, {Reindert J.A.} and {de Wit}, Ronald and {van den Bergh}, {Alphonsus C.M.} and Coenen, {Jules L.L.M.} and Beerlage, {Harrie P.} and Hendriks, {Mathijs P.} and Bos, {Monique M.E.M.} and {van den Berg}, Pieter and {van de Wouw}, {Agnes J.} and Roan Spermon and Boerma, {Michiel O.} and Geenen, {Maud M.} and Tick, {Lidwine W.} and Polee, {Marco B.} and Bloemendal, {Haiko J.} and Igor Cordia and Peters, {Frank P.J.} and {de Vos}, {Aad I.} and {van den Bosch}, Joan and {van den Eertwegh}, {Alphonsus J.M.} and Gerritsen, {Winald R.}",
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Westgeest, HM, Uyl-de Groot, CA, van Moorselaar, RJA, de Wit, R, van den Bergh, ACM, Coenen, JLLM, Beerlage, HP, Hendriks, MP, Bos, MMEM, van den Berg, P, van de Wouw, AJ, Spermon, R, Boerma, MO, Geenen, MM, Tick, LW, Polee, MB, Bloemendal, HJ, Cordia, I, Peters, FPJ, de Vos, AI, van den Bosch, J, van den Eertwegh, AJM & Gerritsen, WR 2018, 'Differences in Trial and Real-world Populations in the Dutch Castration-resistant Prostate Cancer Registry' European Urology Focus, vol. 4, no. 5, pp. 694-701. https://doi.org/10.1016/j.euf.2016.09.008

Differences in Trial and Real-world Populations in the Dutch Castration-resistant Prostate Cancer Registry. / Westgeest, Hans M.; Uyl-de Groot, Carin A.; van Moorselaar, Reindert J.A.; de Wit, Ronald; van den Bergh, Alphonsus C.M.; Coenen, Jules L.L.M.; Beerlage, Harrie P.; Hendriks, Mathijs P.; Bos, Monique M.E.M.; van den Berg, Pieter; van de Wouw, Agnes J.; Spermon, Roan; Boerma, Michiel O.; Geenen, Maud M.; Tick, Lidwine W.; Polee, Marco B.; Bloemendal, Haiko J.; Cordia, Igor; Peters, Frank P.J.; de Vos, Aad I.; van den Bosch, Joan; van den Eertwegh, Alphonsus J.M.; Gerritsen, Winald R.

In: European Urology Focus, Vol. 4, No. 5, 01.09.2018, p. 694-701.

Research output: Contribution to journalArticleAcademicpeer-review

TY - JOUR

T1 - Differences in Trial and Real-world Populations in the Dutch Castration-resistant Prostate Cancer Registry

AU - Westgeest, Hans M.

AU - Uyl-de Groot, Carin A.

AU - van Moorselaar, Reindert J.A.

AU - de Wit, Ronald

AU - van den Bergh, Alphonsus C.M.

AU - Coenen, Jules L.L.M.

AU - Beerlage, Harrie P.

AU - Hendriks, Mathijs P.

AU - Bos, Monique M.E.M.

AU - van den Berg, Pieter

AU - van de Wouw, Agnes J.

AU - Spermon, Roan

AU - Boerma, Michiel O.

AU - Geenen, Maud M.

AU - Tick, Lidwine W.

AU - Polee, Marco B.

AU - Bloemendal, Haiko J.

AU - Cordia, Igor

AU - Peters, Frank P.J.

AU - de Vos, Aad I.

AU - van den Bosch, Joan

AU - van den Eertwegh, Alphonsus J.M.

AU - Gerritsen, Winald R.

PY - 2018/9/1

Y1 - 2018/9/1

N2 - Background: Trials in castration-resistant prostate cancer (CRPC) treatment have shown improved outcomes, including survival. However, as trial populations are selected, results may not be representative for the real-world population. The aim of this study was to assess the differences between patients treated in a clinical trial versus standard care during the course of CRPC in a real-world CRPC population. Design, setting, and participants: Castration-resistant Prostate Cancer Registry is a population-based, observational, retrospective registry. CRPC patients from 20 hospitals in the Netherlands have been included from 2010 to 2013. Outcome measurements and statistical analysis: Baseline characteristics, systemic treatment, and overall survival were the main outcomes. Descriptive statistics, multivariate Cox regression, and multiple imputations with the Monte Carlo Markov Chain method were used. Results and limitations: In total, 1524 patients were enrolled of which 203 patients had participated in trials at any time. The median follow-up period was 23 mo. Patients in the trial group were significantly younger and had less comorbidities. Docetaxel treatment was more frequently used in trial patients (85% vs 40%). Despite an observed unadjusted median overall survival difference of 35 mo versus 24 mo between the trial and standard care group, this difference was not retained after adjustment for baseline characteristics and treatment effect. Conclusions: At CRPC diagnosis, the baseline characteristics of the patients who had been enrolled in trials notably differed from patients who received standard treatment options only. The survival difference between the trial and standard care group could be explained by baseline differences and treatment effects. These results indicate that trial results cannot easily be translated to real-world practice. Patient summary: We observed that patients treated in clinical trials differed from patients who were not. We concluded that this may lead to differential treatment and survival. Caution is warranted when real-world outcomes are compared with trial results. We observed that castration-resistant prostate cancer patients treated in clinical trials differ from patients who are not. We conclude that this may lead to differential treatment and survival. This warrants caution when comparing real-world outcomes to trial results.

AB - Background: Trials in castration-resistant prostate cancer (CRPC) treatment have shown improved outcomes, including survival. However, as trial populations are selected, results may not be representative for the real-world population. The aim of this study was to assess the differences between patients treated in a clinical trial versus standard care during the course of CRPC in a real-world CRPC population. Design, setting, and participants: Castration-resistant Prostate Cancer Registry is a population-based, observational, retrospective registry. CRPC patients from 20 hospitals in the Netherlands have been included from 2010 to 2013. Outcome measurements and statistical analysis: Baseline characteristics, systemic treatment, and overall survival were the main outcomes. Descriptive statistics, multivariate Cox regression, and multiple imputations with the Monte Carlo Markov Chain method were used. Results and limitations: In total, 1524 patients were enrolled of which 203 patients had participated in trials at any time. The median follow-up period was 23 mo. Patients in the trial group were significantly younger and had less comorbidities. Docetaxel treatment was more frequently used in trial patients (85% vs 40%). Despite an observed unadjusted median overall survival difference of 35 mo versus 24 mo between the trial and standard care group, this difference was not retained after adjustment for baseline characteristics and treatment effect. Conclusions: At CRPC diagnosis, the baseline characteristics of the patients who had been enrolled in trials notably differed from patients who received standard treatment options only. The survival difference between the trial and standard care group could be explained by baseline differences and treatment effects. These results indicate that trial results cannot easily be translated to real-world practice. Patient summary: We observed that patients treated in clinical trials differed from patients who were not. We concluded that this may lead to differential treatment and survival. Caution is warranted when real-world outcomes are compared with trial results. We observed that castration-resistant prostate cancer patients treated in clinical trials differ from patients who are not. We conclude that this may lead to differential treatment and survival. This warrants caution when comparing real-world outcomes to trial results.

KW - Castration-resistant prostate cancer

KW - Docetaxel

KW - Outcomes research

KW - Population based

KW - Real-world outcomes

KW - Registry

KW - Registry of outcomes

KW - Treatment

KW - Trial population

UR - http://www.scopus.com/inward/record.url?scp=85008622704&partnerID=8YFLogxK

U2 - 10.1016/j.euf.2016.09.008

DO - 10.1016/j.euf.2016.09.008

M3 - Article

VL - 4

SP - 694

EP - 701

JO - European Urology Focus

JF - European Urology Focus

SN - 2405-4569

IS - 5

ER -