PITTSBURGH — As focal therapy gains popularity as a less invasive treatment option for some men with prostate cancer , a national study led by researchers at the University of Pittsburgh found that some men may be receiving treatment that is either unnecessary or not aggressive enough for their cancer.
The findings, published today in JAMA , suggest that about half of patients receiving the procedure fell outside the patient groups for whom its use is supported by current clinical guidelines.
"For appropriately selected patients with intermediate-risk disease, focal therapy is an appealing treatment option because it aims to preserve quality of life while treating the cancer," said senior author Quoc-Dien Trinh, M.D., M.B.A ., professor and chair of the Department of Urology at the University of Pittsburgh School of Medicine. "But as promising new technologies become more widely available, it is important to understand which patients are most likely to benefit so that treatment decisions are guided by evidence and aligned with clinical guidelines."
Prostate cancer is the most diagnosed cancer among men, affecting about one in eight during their lifetime, according to the U.S Centers for Disease Control and Prevention. For some carefully selected patients with intermediate-risk disease, focal therapy has emerged as an alternative to treating or removing the entire prostate. Instead, the procedure targets only the cancerous portion of the gland, with the goal of preserving healthy tissue and reducing side effects such as urinary incontinence and sexual dysfunction, which can occur after surgery or radiation therapy.
To understand how focal therapy is being used in practice, using the National Cancer Database. Researchers analyzed data from 1,179,384 men, age 50 and older, diagnosed with nonmetastatic prostate cancer between 2010 and 2023. Among the 15,672 patients who received focal therapy, about half belonged to groups with low-risk disease, high risk or very high risk disease, for whom focal therapy treatment was not recommended by the contemporary guidelines and for whom the treatment may not always be the best option.
For men with low-risk prostate cancer, focal therapy may constitute unnecessary treatment, as many can be safely monitored through active surveillance. In contrast, patients with high- and very high-risk disease may require more aggressive therapies with stronger evidence for long-term cancer control than focal therapy can currently provide.
The researchers emphasize that the study does not argue against focal therapy. Rather, it highlights a broader challenge in cancer care: ensuring that promising new treatments are adopted in ways that are supported by evidence and targeted to the patients most likely to benefit.
While focal therapy may be appropriate for some patients, important questions remain about long-term cancer outcomes, retreatment rates, treatment-related side effects and quality of life. "Innovation is critical in prostate cancer care," said Trinh. "Our findings highlight the need to ensure that promising new treatments are adopted in ways that are supported by evidence and matched to the patients most likely to benefit."
As the use of focal therapy continues to expand, the researchers say additional data will help clarify those patients who are most likely to benefit from the approach. While the National Cancer Database provides a broad national picture of treatment patterns, it does not capture participation in clinical trials or prospective registries, nor does it include information on long-term cancer outcomes, quality of life, side effects, retreatment or cost.
Future studies examining these outcomes could help refine patient selection and inform the ongoing evolution of clinical guidelines. In addition, focal therapy performed outside Commission on Cancer-accredited centers may not have been captured in the analysis, meaning the number of patients who received focal therapy might be larger.
"Our main goal is to ensure that each patient receives the treatment that best matches the characteristics of their cancer and their individual needs," added Trinh.
Additional authors on the study are Andrea Cosenza, M.D., David-Dan Nguyen, M.D.C.M., M.P.H., Ph.D.(c), Benjamin J. Davies, M.D., Bruce L. Jacobs, M.D., M.P.H., and Adam Olson, M.D all of UPMC or the University of Pittsburgh School of Medicine; Giorgio Gandaglia, M.D., and Andrea Cosenza, M.D., both of IRCCS San Raffaele Scientific Institute and Vita-Salute San Raffaele University; David-Dan Nguyen, M.D.C.M., M.P.H., Ph.D.(c), of the University of Toronto, Zhiyu Qian, M.D. and Alexander P. Cole, M.D., both of Harvard Medical School; and Rui Bernardino, M.D., of Cleveland Clinic.
JAMA