Clinicaltrials.gov identifier:
Enrollment:
345
Study Design:
Patients with UTUC ≤90 days post NU were randomized (1:1) to 4 cycles of gemcitabine-cisplatin (gemcitabine-carboplatin if GFR 30-49ml/min) or surveillance with subsequent chemotherapy if required. Pts had 6 monthly cross sectional imaging and cystoscopy for the first 2 years, then annually to 5 years. Toxicity was assessed by CTCAE v4.
Rationale:
The role of post nephro-ureterectomy (NU) treatment for UTUC is unclear. POUT (CRUK/11/027) addresses whether adjuvant chemotherapy improves disease free survival (DFS) for pts with histologically confirmed pT2-T4 N0-3 M0 UTUC.
Endpoints:
DFS
Results:
The results were presented at ASCO GU, J Clin Oncol; abstract 407 in February 2018. Between May 2012 & Sept 2017, 248 pts were recruited (123 surveillance; 125 chemotherapy) at 57 UK centers. In Oct 2017, the independent trial oversight committees recommended POUT close to recruitment as data collected thus far (as of 05/09/2017) met the early stopping rule for efficacy. At the time of interim analysis, median follow-up was 17.6 months (IQR 7.5-33.6). Patients had median age 69 years (range 36-88), 30% pT2, 65% pT3; 91% pN0; Grade ≥3 toxicities were reported in 60% chemotherapy pts & 24% surveillance pts. 47/123 (surveillance) & 29/125 (chemotherapy) DFS events were reported; unadjusted HR = 0.47 (95% CI: 0.29, 0.74) in favor of chemotherapy (log-rank p = 0.0009). Two year DFS was 51% for surveillance (95% CI: 39, 61) and 70% for chemotherapy (95% CI: 58, 79). PFS favored chemotherapy: HR = 0.49 (95% CI: 0.30, 0.79, p = 0.003).
