Hans M Westgeest1, Carin A Uyl-de Groot2, Reindert J A van Moorselaar3, Ronald de Wit4, Alphonsus C M van den Bergh5, Jules L L M Coenen6, Harrie P Beerlage7, Mathijs P Hendriks8, Monique M E M Bos9, Pieter van den Berg10, Agnes J van de Wouw11, Roan Spermon12, Michiel O Boerma13, Maud M Geenen14, Lidwine W Tick15, Marco B Polee16, Haiko J Bloemendal17, Igor Cordia18, Frank P J Peters19, Aad I de Vos20, Joan van den Bosch21, Alphonsus J M van den Eertwegh22, Winald R Gerritsen23. 1. Department of Internal Medicine, Amphia Hospital, Breda, The Netherlands; Institute for Medical Technology Assessment, Erasmus University, Rotterdam, The Netherlands. Electronic address: hwestgeest@amphia.nl. 2. Institute for Medical Technology Assessment, Erasmus University, Rotterdam, The Netherlands. 3. Department of Urology, VU University Medical Center, Amsterdam, The Netherlands. 4. Department of Medical Oncology, ErasmusMC Cancer Institute, Rotterdam, The Netherlands. 5. Department of Radiation Oncology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands. 6. Department of Internal Medicine, Isala, Zwolle, The Netherlands. 7. Department of Urology, Jeroen Bosch Ziekenhuis, 's-Hertogenbosch, The Netherlands. 8. Department of Internal Medicine, Northwest Clinics, Alkmaar, The Netherlands. 9. Department of Internal Medicine, Reinier de Graaf Groep, Delft, The Netherlands. 10. Department of Internal Medicine, Tergooi Ziekenhuizen, Hilversum, The Netherlands. 11. Department of Internal Medicine, Viecuri Medisch Centrum, Venlo, The Netherlands. 12. Department of Urology, Diakonessen Ziekenhuis, Utrecht, The Netherlands. 13. Department of Urology, Deventer Ziekenhuis, Deventer, The Netherlands. 14. Department of Internal Medicine, OLVG Locatie West, Amsterdam, The Netherlands. 15. Department of Internal Medicine, Maxima Medisch Centrum, Eindhoven, The Netherlands. 16. Department of Internal Medicine, Medical Center, Leeuwarden, The Netherlands. 17. Department of Internal Medicine/Oncology, Meander Medical Center, Amersfoort, The Netherlands. 18. Department of Urology, MCH-Bronovo Ziekenhuis, 's-Gravenhage, The Netherlands. 19. Department of Internal Medicine, Zuyderland Medisch Centrum, Heerlen-Sittard, The Netherlands. 20. Department of Internal Medicine, van Weel Bethesda Ziekenhuis, Dirksland, The Netherlands. 21. Department of Internal Medicine, Albert Schweitzer Ziekenhuis, Dordrecht, The Netherlands. 22. Department of Medical Oncology, VU University Medical Center, Amsterdam, The Netherlands. 23. Department of Medical Oncology, Radboud University Medical Center, Nijmegen, The Netherlands.
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.
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.
Authors: Kevin M Veen; Isabel B de Angst; Mostafa M Mokhles; Hans M Westgeest; Malou Kuppen; Carin A Uyl-de Groot; Winald R Gerritsen; Paul J M Kil; Johanna J M Takkenberg Journal: J Cancer Res Clin Oncol Date: 2020-06-17 Impact factor: 4.553
Authors: Guillemette E Benoist; Inge M van Oort; David M Burger; Niven Mehra; Nielka P van Erp Journal: Cancer Chemother Pharmacol Date: 2020-02-19 Impact factor: 3.333
Authors: Peter H J Slootbeek; Iris S H Kloots; Minke Smits; Inge M van Oort; Winald R Gerritsen; Jack A Schalken; Marjolijn J L Ligtenberg; Katrien Grünberg; Leonie I Kroeze; Haiko J Bloemendal; Niven Mehra Journal: Br J Cancer Date: 2021-12-15 Impact factor: 9.075
Authors: Yashar Khoshkar; Marcus Westerberg; Jan Adolfsson; Anna Bill-Axelson; Henrik Olsson; Martin Eklund; Olof Akre; Hans Garmo; Markus Aly Journal: BJUI Compass Date: 2021-10-10
Authors: Marscha S Holleman; Simone A Huygens; Maiwenn J Al; Malou C P Kuppen; Hans M Westgeest; Alfonsus C M van den Bergh; Andries M Bergman; Alfonsus J M van den Eertwegh; Mathijs P Hendriks; Menuhin I Lampe; Niven Mehra; Reindert J A van Moorselaar; Inge M van Oort; Diederik M Somford; Ronald de Wit; Agnes J van de Wouw; Winald R Gerritsen; Carin A Uyl-de Groot Journal: Drugs Real World Outcomes Date: 2022-03-21
Authors: Jakub Radocha; Vladimír Maisnar; Luděk Pour; Ivan Špička; Jiři Minařík; Lenka Szeligová; Petr Pavlíček; Alexandra Jungová; Marta Krejčí; Tomáš Pika; Jan Straub; Lucie Brožová; Lukáš Stejskal; Adriana Heindorfer; Pavel Jindra; Petr Kessler; Peter Mikula; Michal Sýkora; Marek Wróbel; Jiří Jarkovský; Roman Hájek Journal: Cancer Med Date: 2018-06-21 Impact factor: 4.452
Authors: Peter H J Slootbeek; Marleen L Duizer; Maarten J van der Doelen; Iris S H Kloots; Malou C P Kuppen; Hans M Westgeest; Carin A Uyl-de Groot; Samhita Pamidimarri Naga; Marjolijn J L Ligtenberg; Inge M van Oort; Winald R Gerritsen; Jack A Schalken; Leonie I Kroeze; Haiko J Bloemendal; Niven Mehra Journal: Int J Cancer Date: 2020-10-03 Impact factor: 7.396