Pathologic downstaging following neoadjuvant chemotherapy (NAC) predicts better survival in high-risk upper tract urothelial carcinoma (UTUC) patients, researchers reported in The Journal of Urology.

Using real-world, international data, Beat Foerster, MD, of the Medical University of Vienna in Austria, and colleagues studied 267 patients with nonmetastatic high-risk UTUC who received chemotherapy prior to radical nephroureterectomy (RNU) during 2005 to 2017. Of these, 31% received methotrexate, vinblastine, doxorubicin, and cisplatin (MVAC); 46% received gemcitabine and cisplatin (GC); 9% received gemcitabine and carboplatin (GCb); and 12% received other regimens.

A total of 10.1% of patients experienced pathologic complete response and 34.8% had a partial response. Overall, 44.9% had pathologic downstaging (pT1 or less). Smaller proportions of patients with clinical lymph node involvement experienced complete response and downstaging.

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On multivariable analysis, the use of GC and GCb did not differ from MVAC in achieving complete response and downstaging, respectively. Only on univariable analyses was the use of GCb associated with lower odds of downstaging. The number of chemotherapy cycles had no influence on results.

Pathologic downstaging strongly and significantly predicted recurrence-free survival, cancer-specific survival, and overall survival, Dr Foerster’s team reported.

“Approximately half of the patients experienced measurable response to chemotherapy at the time of surgery and had a major survival benefit during follow-up. Overall, our results confirm the beneficial role for the use of cisplatin-based preoperative chemotherapy in patients with high-risk UTUC and can be used in patients counselling and treatment planning,” they concluded. “However, results from prospective clinical trials are urgently needed to change the treatment paradigm of UTUC management.”

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Foerster B, Abufaraj M, Petros F, et al; UTUC collaboration. Efficacy of preoperative chemotherapy in high-risk upper tract urothelial carcinoma (UTUC) [published online January 3, 2020]. J Urol. doi: 10.1097/JU.0000000000000737