Penn State Health to Study New Hematuria Urine Test in Clinical Trial

Could less be more when it comes to testing for bladder cancer? Penn State Health Milton S. Hershey Medical Center is part of a new multicenter study that will ask that question, and the answer could change the standard of care.

Under the current clinical guidelines for physicians, patients 35 and older who have blood in their urine, also known as hematuria, get an extensive workup to look for causes, like inflammation, kidney stones or—in the worst case—bladder cancer. Testing currently involves cystoscopy, computed tomography (CT), and in some cases urine cytology.

Cystoscopy can be invasive and anxiety provoking. It involves inserting a camera through the urethra into the bladder to look for tumors or other abnormalities. Meanwhile, a single CT scan delivers the radiation equivalent of 100 chest X-rays or greater.

For many low-risk patients, this battery of tests could be overkill, said Dr. Jay Raman, professor of surgery and chief of the Department of Urology at Penn State Health. When blood in the urine is visible to the naked eye, the tests identify cancer in approximately 10% of patients. That drops to only about 3% for microscopic hematuria.

Penn State Health is the first center to enroll patients into the new clinical trial, which will help to assess whether simple urine biomarker tests called Cxbladder Triage and Cxbladder Detect could replace the full workup for some lower risk patients. Cxbladder has previously been shown to be potentially superior to urine cytology for low grade bladder tumor detection.

The U.S. Food and Drug Administration-approved tests look for gene expression changes associated with bladder cancer. Cxbladder Triage combines these changes with risk factors to determine if patients have a low probability for bladder cancer, whereas Cxbladder Detect diagnoses malignancies based on the changes. Used in sequence, the two tests can thus “rule out” patients for bladder cancer.

The trial will enroll an estimated 600 adult patients with recently confirmed hematuria at eight academic and community medical centers around the United States and Canada. The researchers will randomly assign patients with a low risk of urothelial carcinoma—the most common form of bladder cancer—into two arms. The test group will receive Cxbladder Triage testing, while the control group will receive cystoscopy as standard of care.

 

In the case of a negative Cxbladder Triage test result, the physician will decide to either rule out or further investigate the patient with cystoscopy. Patients who don’t receive a negative result will be provided a Cxbladder Detect test, followed by further evaluation, which could include cystoscopy.

Six months after the trial’s completion, the researchers will look at the outcomes: Did Cxbladder reduce the number of cystoscopies and other procedures physicians ordered? How many patients were ruled out who actually had cancer? How many patients did Cxbladder correctly rule out or diagnose? And did Cxbladder reduce anxiety and pain compared with cystoscopy?

The goal is to see if patients can have the same good outcomes with less testing. And according to Dr. Raman, the findings could influence guidelines for hematuria management. “There is an understanding that perhaps we are being overly aggressive with our testing,” he said.

Cxbladder follows the overall health care trend of less invasive testing. That’s especially important with blood in the urine, a common condition. The gold standard tests are both expensive and extensive, and many patients with hematuria don’t follow through with them. “The advantage of this noninvasive testing is that nobody objects to giving a urine sample,” Dr. Raman said. “It allows us to make sure that we don’t lose some of these patients to follow-up who are deterred by more invasive testing.”

Recruitment for the study begins in October 2019, and results could be available as soon as late next year.