Background and Purpose: While nephroureterectomy (NU) remains the gold-standard treatment for upper-tract carcinoma in situ (UT-CIS), it may be unnecessarily aggressive in comparison with the treatment of bladder CIS. Upper-tract administration of bacillus Calmette-Guérin (BCG) has shown promise for UT-CIS, but with limited reports and varied results. Furthermore, all previous reports included patients with positive cytology results without biopsy-proven CIS, or combined BCG with other topical therapies that are used for bladder CIS. We report our experience using a novel technique to directly instill BCG with interferon-α2B (BCG/IFN) into the upper-tract in patients with biopsy-proven UT-CIS.

Patients and Methods: Patients who received a diagnosis of isolated, biopsy-proven UT-CIS from September 2003 to January 2012 were included. After biopsy, all patients received a 6-week induction course of BCG/IFN, administered via an open-ended ureteral catheter. Initial follow-up was scheduled 1 month after the completion of the intrarenal therapy and consisted of flexible ureteroscopy, selective urinary cytology, retrograde pyelography, and rebiopsy of the upper tract. Complete response (CR) was defined as the absence of visualized lesions on ureteroscopy, negative selective cytology results, and absence of clinical progression. Absence of visualized lesions with persistently positive cytology results or persistence of lesions after induction was considered no response (NR). New upper-tract lesions after an initial CR were considered recurrences. Patients with a CR were placed on maintenance therapy for 2 years. Surveillance was performed every 3 months with ureteroscopy, selective cytology, and imaging.

Results: Eleven patients (mean age=73 years) were followed for a median of 13.5 months (3.7–103.3 mos). Eight patients had an initial CR, while three initially had NR. Two of the NR patients had negative biopsy results but persistently positive cytology results; both of these patients underwent a second induction course and achieved a CR. The third NR patient had persistence of lesions after induction and was offered a nephroureterctomy. Total kidney preservation rate was 91% (10/11). There were no treatment-related adverse events.

Conclusion: This study demonstrates the safety and efficacy of intrarenal BCG/IFN maintenance therapy for patients with UT-CIS. Unlike other mechanisms of delivery, including percutaneous administration or reflux via double pigtail stents, this office-based technique spares the morbidity of a chronically indwelling nephrostomy tube or ureteral stent.