Photodynamic Diagnosis with Oral 5-Aminolevulinic Acid for Upper Urinary Tract Carcinoma: A Prospective Clinical Trial

In this study, we evaluated the effectiveness and safety of photodynamic diagnosis (PDD) of upper urinary tract carcinoma (UTUC) using oral 5-aminolevulinic acid (5-ALA). Consecutive adult patients with suspected UTUC based on imaging and urine cytology findings were prospectively enrolled from May 2018 to January 2019. 5-ALA (20 mg/kg) was orally administered 2 to 3 hours before diagnostic ureteroscopy (URS). Sixty-three biopsy samples were collected from 20 eligible patients using white light (WL) and PDD-URS. The primary endpoint was sensitivity, and the secondary endpoints were specificity, positive predictive value (PPV) and negative predictive value (NPV), diagnostic accuracy, and safety. The sensitivity of photodynamic URS was significantly higher than that of WL-URS (93.8% 62.5%, = 0.0025). Although the specificity and PPV were not different between the two procedures, PDD-URS showed a significantly higher NPV (92.3% 69.2%, = 0.027) and accuracy (0.86 0.75, = 0.0297) than WL-URS. With respect to safety, no grade ≥3 adverse events related to 5-ALA administration occurred in any patients. We found that PDD-URS with oral 5-ALA is a safe and superior diagnostic tool for detection of UTUC compared with conventional WL-URS.