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Introduction <p>The efficacy of combined neoadjuvant and adjuvant therapy (CNAT) in upper tract urothelial carcinoma (UTUC) remains unclear despite its demonstrated potential in bladder urothelial carcinoma. High-risk features– clinical stage ≥ T3, node-positive disease, multifocality, high-grade pathology, hydronephrosis, and large tumor size – are associated with poor prognosis in UTUC. We investigated the oncological outcomes of CNAT versus adjuvant therapy (AT) alone in high-risk UTUC patients.</p> Materials and Methods <p>We analyzed perioperative data from 2433 patients with UTUC (2015–2023) across 17 centers in the US, Europe, and Asia. Propensity score matching was performed using preoperative clinical T and N stages. Kaplan–Meier curves and Cox proportional hazards models were used to evaluate overall survival (OS), cancer-specific survival (CSS), recurrence-free survival (RFS), and metastasis-free survival (MFS).</p> Results <p>Among 285 high-risk UTUC patients, 76 matched patients (38 CNAT, 38 AT) were analyzed after matching, with a median follow-up of 15 months. CNAT and AT groups had comparable oncological outcomes: 2-year OS (72.9% vs. 71.8%; p = 0.89), CSS (76.7% vs. 75.3%; p = 0.92), RFS (30.1% vs. 39%; p = 0.97), or MFS (45.5% vs. 44.7%; p = 0.91), respectively. Cox regression showed no significant survival benefit of CNAT over AT after adjusting for clinical and pathological factors (HR for OS: 1.06; p = 0.9).</p> Conclusion <p>In this large multicenter international cohort, our findings suggest that CNAT does not provide a clear advantage over AT alone in patients with high-risk UTUC. Prospective randomized trials are needed to clarify the role of multimodal therapy in UTUC management.</p>

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Combined neoadjuvant and adjuvant therapy versus adjuvant therapy in high-risk upper tract urothelial carcinoma: a propensity matched multicenter analysis (ROBUUST 2.0 International Collaborative Group)

  • 期刊论文

  • 发表日期：

    2025年4月18日

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  • Ahmed Eraky,
  • Reuben Ben-David,
  • Gabriele Bignante,
  • Zhenjie Wu,
  • Linhui Wang,
  • Randall Lee,
  • Andres F. Correa,
  • Daniel D. Eun,
  • Alessandro Antonelli,
  • Alessandro Veccia,
  • Francesco Ditonno,
  • Firas Abdollah,
  • Alex Stephens,
  • Shane Tinsley,
  • Daniel Sidhom,
  • Chandru P. Sundaram,
  • Sol C. Moon,
  • Soroush Rais-Bahrami,
  • Mark L. Gonzalgo,
  • Omri Falik Nativ,
  • Francesco Porpiglia,
  • Daniele Amparore,
  • Enrico Checcucci,
  • Antonio Tufano,
  • Sisto Perdonà,
  • Stephan Brönimann,
  • Nirmish Singla,
  • Ottavio De Cobelli,
  • Matteo Ferro,
  • Giuseppe Simone,
  • Gabriele Tuderti,
  • Margaret F. Meagher,
  • Ithaar H. Derweesh,
  • Takashi Yoshida,
  • Hidefumi Kinoshita,
  • Raj Bhanvadia,
  • Ali H. Zahalka,
  • Vitaly Margulis,
  • Farshad S. Moghaddam,
  • Hooman Djaladat,
  • Riccardo Autorino,
  • Reza Mehrazin

摘要

Introduction

The efficacy of combined neoadjuvant and adjuvant therapy (CNAT) in upper tract urothelial carcinoma (UTUC) remains unclear despite its demonstrated potential in bladder urothelial carcinoma. High-risk features– clinical stage ≥ T3, node-positive disease, multifocality, high-grade pathology, hydronephrosis, and large tumor size – are associated with poor prognosis in UTUC. We investigated the oncological outcomes of CNAT versus adjuvant therapy (AT) alone in high-risk UTUC patients.

Materials and Methods

We analyzed perioperative data from 2433 patients with UTUC (2015–2023) across 17 centers in the US, Europe, and Asia. Propensity score matching was performed using preoperative clinical T and N stages. Kaplan–Meier curves and Cox proportional hazards models were used to evaluate overall survival (OS), cancer-specific survival (CSS), recurrence-free survival (RFS), and metastasis-free survival (MFS).

Results

Among 285 high-risk UTUC patients, 76 matched patients (38 CNAT, 38 AT) were analyzed after matching, with a median follow-up of 15 months. CNAT and AT groups had comparable oncological outcomes: 2-year OS (72.9% vs. 71.8%; p = 0.89), CSS (76.7% vs. 75.3%; p = 0.92), RFS (30.1% vs. 39%; p = 0.97), or MFS (45.5% vs. 44.7%; p = 0.91), respectively. Cox regression showed no significant survival benefit of CNAT over AT after adjusting for clinical and pathological factors (HR for OS: 1.06; p = 0.9).

Conclusion

In this large multicenter international cohort, our findings suggest that CNAT does not provide a clear advantage over AT alone in patients with high-risk UTUC. Prospective randomized trials are needed to clarify the role of multimodal therapy in UTUC management.