THE largest real-world data (RWD) study under the PIONEER project highlights treatment trends and outcomes in metastatic hormone-sensitive prostate cancer (mHSPC).
With the number of prostate cancer treatment options rapidly increasing, it has remained unclear which approaches are most effective. The researchers aimed to examine actual treatment use and outcomes by analysing electronic health records, insurance claims, primary care data, and cancer registries from eight European and US databases covering 2016–2020.
In total, 107,438 patients with mHSPC were identified, of whom 67,909 received treatment. Most patients received androgen deprivation therapy (ADT) alone (69.4%), followed by ADT combined with an androgen receptor pathway inhibitor (ARPI) (15.2%), ADT plus chemotherapy (14%), and triplet therapy (1.2%).
Combination Therapy Outperforms ADT in Prostate Cancer
Patients receiving the combination of ADT with an ARPI demonstrated the highest treatment persistence at 53.8% and a five-year switch-free survival rate of up to 72.3%. In contrast, ADT monotherapy showed five-year switch-free survival rates ranging from 21.3% to 58.6%, with adverse event–free survival between 64.7% and 81.2%. Adding an ARPI improved switch-free survival (24.1–72.3%) but was associated with slightly lower adverse event–free survival (55.2–82.7%). Chemotherapy-based and triplet therapies produced variable outcomes without consistent survival benefits.
The study’s limitations include potential residual confounding, inconsistent adverse event reporting across databases, and possible overlap in patient data. Overall, the findings highlight a real-world gap between clinical evidence and practice, suggesting that while combination therapies improve persistence and outcomes, ADT monotherapy remains the most frequently used first-line treatment.
This is the largest RWD study of systemic treatment for mHSPC. Despite strong evidence supporting combination therapy, ADT monotherapy continues to be the most commonly used first-line therapy. Increased adoption of ADT + ARPI is associated with higher treatment persistence and improved outcomes in real-world settings, supporting its broader integration into clinical practice.
Reference
Rosella Nicoletti et al. Treatment Trajectories in Metastatic Hormone-sensitive Prostate Cancer: A PIONEER+ Big Data Analysis. Eur. Urol. Oncol. 2025;DOI: 10.1016/j.euo.2025.2287.
