Rates of surveillance have continued to grow among US veterans with low-risk or favorable intermediate-risk prostate cancer, in line with guidelines, while thousands of patients nationally continue to receive focal therapy despite limited evidence supporting its use, according to a pair of new papers.

In one study, data from the Veterans Affairs Healthcare System showed that between 2005 and 2024, active surveillance/watchful waiting rose from 27% to 93% in men with low-risk disease, and from 14% to 61% in those with favorable intermediate-risk disease.

The other study, using data from the National Cancer Database, found that more than 15,000 patients diagnosed between 2010 and 2023 — or 1.3% of the study population — received focal therapy such as laser ablation or high-intensity focused ultrasound. However, such treatment remains investigational and is not backed by high-quality evidence.

The two studies were published online on August 13 in JAMA, alongside an accompanying editorial.

The studies “show real progress, in that surveillance is finally treated as disciplined care rather than therapeutic inaction,” Spyridon P. Basourakos, MD, of Case Western Reserve University in Cleveland, and colleagues wrote in the editorial, titled “The Emperor’s New Clothes — The Discipline of Evidence.”

The studies “also expose a growing concern, in that focal therapy is presented as elegant and individualized when it usually amounts to overtreatment or undertreatment, with no quality data to demonstrate that it is superior to other noninvasive standard of care options,” wrote Basourakos, along with coauthors Jonathan E. Shoag, MD, and Daniel E. Spratt, MD.

The increase in active surveillance is “gratifying,” said David Einstein, MD, a prostate cancer specialist at the Beth Israel Deaconess Medical Center in Boston, who was not involved in the research.

While the paper on focal therapies “raises troubling questions,” he added, the analysis “didn’t necessarily exclude cases” in which the treatment was used to “address obstructive urinary symptoms before proceeding to traditional radiation.” Considering this point and the low overall use, “I don’t necessarily think this article points to a massive problem,” Einstein said.

A Look at the Data

According to current guidelines, active surveillance is the preferred management approach for low-risk and many intermediate-risk prostate cancers, while focal therapy is recommended only for intermediate-risk disease in clinical trials or prospective registries. For low-risk disease, focal therapy may represent overtreatment, whereas for high- and very-high-risk disease, there is insufficient high-level evidence.

To characterize recent management trends, the VA study assessed use of active surveillance/watchful waiting between 2005 and 2024 in men with favorable-risk prostate cancer.

Grace Lee, MD, of the San Francisco Veterans Affairs Medical Center, and colleagues classified patients as being on active surveillance or watchful waiting if they did not receive treatment within 15 months of their diagnostic biopsy and had one or more prostate-specific antigen (PSA) values ≥ 1 ng/mL during this time, or if they received a confirmatory biopsy within 3-15 months after their diagnostic biopsy and before any active treatment.

The median age of the more than 73,000 men in the study was 65 years. As the use of surveillance as initial management rose over time, rates of radiation and surgery dropped from more than 40% and 20%, respectively, to single digits in men with low-risk disease and to just below 20% in those with favorable intermediate-risk disease.

On subgroup analysis, surveillance rates for patients with PSA levels < 10 ng/mL and Gleason grade group 2 in less than 50% of cores rose from 11% to 55%. The rates increased from 27% to 88% for men with Gleason grade group 1 and PSA level 10-20 ng/mL.

Multivariable logistic regression showed increasing age and more recent diagnosis were tied to higher odds of surveillance. By contrast, people who were Black (odds ratio [OR], 0.95 vs White; P < .001) or Hispanic (OR, 0.85 vs non-Hispanic; P = .003) or lived in more disadvantaged neighborhoods had lower odds of receiving active surveillance — a trend that “might be exacerbated outside of a VA system,” said Einstein, “so there is still room for improvement.”

The other study, by Andrea Cosenza, MD, of the University of Pittsburgh Medical Center, Pittsburgh, and colleagues, analyzed data from the National Cancer Database.

The researchers classified focal therapy as inappropriate in low-, high-, or very-high-risk disease, and reported intermediate-risk disease separately as the database “does not capture trial enrollment, registry participation, or lesion characteristics needed to classify appropriateness,” they wrote.

Among a study population of nearly 1.18 million men 50 and older, 15,672 received focal therapy. Fifty-one percent had low-, high-, or very-high-risk disease, and 49.0% had intermediate-risk disease.

More intermediate-risk patients received focal therapies in 2023 than in 2010 (2.9% vs 2.1%, P < .001), while the proportion dropped in men with unfavorable intermediate-risk (1.9% vs 2.5%), high-risk (0.9% vs 2.1%), and very-high-risk disease (0.5% vs 1.8%). Additionally, there was no significant change in the proportion of patients with low-risk disease who received focal therapies between 2010 (1.8%) and 2023 (2.2%).

The use of cryotherapy fell, whereas laser ablation and other tumor-destruction methods such as high-intensity focused ultrasound became more popular during the study period.

Focal therapy techniques, including newer ones, “deserve evaluation on their own evidence. Yet as the number of devices has grown, the evidence has not,” the editorialists wrote.

“The patterns of adoption deepen the concern,” Basourakos, Shoag, and Spratt added. The study found that, compared with radical prostatectomy, focal therapy was more common among older and sicker patients, at community programs, and at higher-volume centers.

Although the use seems limited overall, “we should certainly be cautious about overselling patients on an emerging technology,” Einstein agreed.

“I do have concerns that these focal approaches may risk messier salvage treatments in those patients at higher risk who subsequently recur and may risk over treatment in patients who should really be encouraged to pursue active surveillance,” he added.

Funding information is not available in the papers. Several study authors reported disclosures in their publications (a full list can be viewed here and here). Einstein reported receiving honoraria from several drugmakers.