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 study suggests that about half of patients receiving the procedure fell outside the patient groups for whom its use is supported by current clinical guidelines.
The findings were published in JAMA.
“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,” Quoc-Dien Trinh, senior author, professor, and chair of the Department of Urology at the University of Pittsburgh School of Medicine, said in a statement. “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.”
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,” said Trinh.
