Plasma and urine biomarker algorithm versus systematic biopsy for prostate cancer detection in elderly men: a randomised trial with early termination for futility.

OBJECTIVE

This study aimed to compare test sensitivity for detecting aggressive prostate cancer and test specificity (measured by reduction in prostate biopsies) between algorithm-triage and standard systematic biopsy in elderly men with suspected prostate cancer.

METHODS

This randomised controlled trial enrolled men ≥ 70 years old suspected of prostate cancer and randomised them 1:1 to algorithm-triage or standard systematic biopsies. The algorithm arm used a 10-gene mRNA panel from urine and plasma samples, integrated with clinical characteristics and PSA measurements to predict prostate cancer with International Society of Urological Pathology grade group ≥ 2. Patient-reported outcomes measures were collected using the Functional Assessment of Cancer Therapy-Prostate (FACT-P) scores or subdomains throughout a 24-month follow-up.

CLINICALTRIALS: gov: NCT04079699.

RESULTS

A total of 202 men were included between October 2019 and September 2021. The study was terminated early due to algorithm underperformance. The algorithm-triage arm detected fewer indolent cancers (7.9% vs. 19%, absolute difference -10.9 percentage points, 95% confidence interval [CI]: -21.0 to -1.0 pp, P = 0.039) but also fewer clinically significant cancers (26% vs. 40%, absolute difference -13.9 percentage points, 95% CI: -27.6 to -0.1 pp, P = 0.051) compared to systematic biopsy. Patient-reported outcomes showed no significant between-group differences in FACT-P scores or subdomains throughout 24-month follow-up (differences 0.1-2.2 points, all P > 0.05), indicating comparable quality of life.

CONCLUSION

The biomarker-based algorithm-triage reduced biopsy numbers but also detected fewer -clinically significant prostate cancers. Quality of life was comparable between approaches.