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Treatment Trajectories in Metastatic Hormone-sensitive Prostate Cancer : A PIONEER+ Big Data Analysis

Nicoletti, Rossella ; Liu, Alex Qinyang ; Evans-Axelsson, Susan ; Golozar, Asieh ; Beyer, Katharina ; Meulder, Bertrand De ; Campi, Riccardo ; Gacci, Mauro ; Teoh, Jeremy Yuen Chun and Steinbesser, Carl , et al. (2025) In European Urology Oncology 8(5). p.1340-1349
Abstract

BACKGROUND AND OBJECTIVE: The treatment landscape for metastatic hormone-sensitive prostate cancer (mHSPC) is evolving rapidly. Real-world data (RWD) are essential to understand actual treatment use and outcomes. This study aimed to describe treatment trajectories and clinical outcomes in a large, multicenter RWD cohort under the PIONEER project. METHODS: Eight European and US databases (2016-2020), including electronic health records, insurance claims, primary care data, and cancer registries, were standardized to the Observational Medical Outcome Partnership Common Data Model. Patients diagnosed with mHSPC and those receiving treatment were identified. The compared and analyzed outcomes included treatment switch, symptomatic... (More)

BACKGROUND AND OBJECTIVE: The treatment landscape for metastatic hormone-sensitive prostate cancer (mHSPC) is evolving rapidly. Real-world data (RWD) are essential to understand actual treatment use and outcomes. This study aimed to describe treatment trajectories and clinical outcomes in a large, multicenter RWD cohort under the PIONEER project. METHODS: Eight European and US databases (2016-2020), including electronic health records, insurance claims, primary care data, and cancer registries, were standardized to the Observational Medical Outcome Partnership Common Data Model. Patients diagnosed with mHSPC and those receiving treatment were identified. The compared and analyzed outcomes included treatment switch, symptomatic progression, adverse events, and death. KEY FINDINGS AND LIMITATIONS: In total, 107 438 patients with mHSPC were identified, and 67 909 received treatment. Most of the patients received androgen deprivation therapy (ADT) monotherapy (69.4%), followed by ADT + an androgen receptor pathway inhibitor (ARPI; 15.2%), ADT + chemotherapy (14.0%), and triplet therapy (1.2%). The use of ARPIs increased over time. ADT + ARPI showed the highest persistence (53.8%) and a 5-yr switch-free survival rate of up to 72.3%. ADT monotherapy had 5-yr switch-free survival rates of 21.3-58.6% and adverse event-free survival rates of 64.7-81.2%. Addition of an ARPI improved switch-free survival (24.1-72.3%) but lowered adverse event-free survival (55.2-82.7%). Chemotherapy-based and triplet therapies showed variable results without consistent survival benefit. Limitations include residual confounding, inconsistent adverse event reporting, and possible data overlap. CONCLUSIONS AND CLINICAL IMPLICATIONS: This is the largest RWD study of systemic mHSPC treatment. Despite evidence, ADT monotherapy remains the most used first-line therapy. Increased use ADT + ARPI is associated with better persistence and improved outcomes in the real-world setting, supporting its broader adoption in clinical practice.

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organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Big data analysis, Metastatic prostate cancer, Prostate cancer, Real-world evidence
in
European Urology Oncology
volume
8
issue
5
pages
10 pages
publisher
Elsevier
external identifiers
  • scopus:105023441829
  • pmid:41062343
ISSN
2588-9311
DOI
10.1016/j.euo.2025.08.007
language
English
LU publication?
yes
id
d035bf99-4651-473b-9789-8041f2184701
date added to LUP
2026-01-30 16:13:03
date last changed
2026-01-30 16:13:47
@article{d035bf99-4651-473b-9789-8041f2184701,
  abstract     = {{<p>BACKGROUND AND OBJECTIVE: The treatment landscape for metastatic hormone-sensitive prostate cancer (mHSPC) is evolving rapidly. Real-world data (RWD) are essential to understand actual treatment use and outcomes. This study aimed to describe treatment trajectories and clinical outcomes in a large, multicenter RWD cohort under the PIONEER project. METHODS: Eight European and US databases (2016-2020), including electronic health records, insurance claims, primary care data, and cancer registries, were standardized to the Observational Medical Outcome Partnership Common Data Model. Patients diagnosed with mHSPC and those receiving treatment were identified. The compared and analyzed outcomes included treatment switch, symptomatic progression, adverse events, and death. KEY FINDINGS AND LIMITATIONS: In total, 107 438 patients with mHSPC were identified, and 67 909 received treatment. Most of the patients received androgen deprivation therapy (ADT) monotherapy (69.4%), followed by ADT + an androgen receptor pathway inhibitor (ARPI; 15.2%), ADT + chemotherapy (14.0%), and triplet therapy (1.2%). The use of ARPIs increased over time. ADT + ARPI showed the highest persistence (53.8%) and a 5-yr switch-free survival rate of up to 72.3%. ADT monotherapy had 5-yr switch-free survival rates of 21.3-58.6% and adverse event-free survival rates of 64.7-81.2%. Addition of an ARPI improved switch-free survival (24.1-72.3%) but lowered adverse event-free survival (55.2-82.7%). Chemotherapy-based and triplet therapies showed variable results without consistent survival benefit. Limitations include residual confounding, inconsistent adverse event reporting, and possible data overlap. CONCLUSIONS AND CLINICAL IMPLICATIONS: This is the largest RWD study of systemic mHSPC treatment. Despite evidence, ADT monotherapy remains the most used first-line therapy. Increased use ADT + ARPI is associated with better persistence and improved outcomes in the real-world setting, supporting its broader adoption in clinical practice.</p>}},
  author       = {{Nicoletti, Rossella and Liu, Alex Qinyang and Evans-Axelsson, Susan and Golozar, Asieh and Beyer, Katharina and Meulder, Bertrand De and Campi, Riccardo and Gacci, Mauro and Teoh, Jeremy Yuen Chun and Steinbesser, Carl and Hijazy, Ayman and Harbachou, Artem and Brash, James T. and Willemse, Peter Paul M. and Murtola, Teemu and Roobol, Monique J. and Sierra, Jesus Moreno and Bjartell, Anders and Merseburger, Axel S. and Rajwa, Pawel and Cornford, Philip and Abbott, Thomas and Ndow, James and Rivas, Juan Gomez and Davies, Eleanor and Feng, Qi and Snijder, Robert}},
  issn         = {{2588-9311}},
  keywords     = {{Big data analysis; Metastatic prostate cancer; Prostate cancer; Real-world evidence}},
  language     = {{eng}},
  number       = {{5}},
  pages        = {{1340--1349}},
  publisher    = {{Elsevier}},
  series       = {{European Urology Oncology}},
  title        = {{Treatment Trajectories in Metastatic Hormone-sensitive Prostate Cancer : A PIONEER+ Big Data Analysis}},
  url          = {{http://dx.doi.org/10.1016/j.euo.2025.08.007}},
  doi          = {{10.1016/j.euo.2025.08.007}},
  volume       = {{8}},
  year         = {{2025}},
}