How is upper tract transitional cell carcinoma (TCC) treated?

Updated: Aug 07, 2020
  • Author: Kyle A Richards, MD, FACS; Chief Editor: Bradley Fields Schwartz, DO, FACS  more...
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Medical therapy for upper tract urothelial cell carcinoma (UTUC) can be topical or systemic. Topical chemotherapeutic agents are delivered by instillation and consist of bacillus Calmette-Guérin (BCG), which is the preferred agent, or mitomycin C. These agents can be administered either percutaneously or via a retrograde approach through a ureteral catheter. For high-grade disease, topical instillation therapy is most appropriate in patients for whom radical surgery is absolutely or relatively contraindicated—those with bilateral disease and/or limited renal function.

In April 2020 the US Food and Drug Administration approved mitomycin pyelocalyceal (Jelmyto) for treatment of low-grade upper tract urothelial cancer (LG-UTUC) in adults. Mitomycin pyelocalyceal is instilled via ureteral catheter or a nephrostomy tube. Approval was based on the OLYMPUS study, in which 41 of 71 patients (58%) achieved a complete response (CR) at 3 months, after receiving 6 weekly instillations of mitomycin pyelocalyceal. Patients with a CR at 3 months then received monthly instillations for a maximum of 11 additional instillations, and 46% of those patients remained in a CR at the 12-month visit.25

BCG is an attenuated form of Mycobacterium tuberculosis, and its use carries a small but significant risk of BCG sepsis. To prevent adverse systemic effects, BCG should not be used in patients with hematuria.

Because of an ongoing shortage of BCG in the United States, the National Comprehensive Cancer Network [23]  and several urologic societies have recommended strategies on prioritizing use of intravesical BCG and alternative treatment approaches for some patients.

The safety of BCG and mitomycin C as adjuvant therapy has been well studied in bladder cancer; however, their efficacy in decreasing recurrence rates, delaying tumor progression, and improving survival rates in upper urinary tract cancer has not been firmly established. [26]  Furthermore, the administration of these agents often requires hospitalization and skilled nursing to prevent hyperperfusion and systemic absorption.

Systemic chemotherapy is often reserved for patients with metastatic disease.

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