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Introduction <p>Enfortumab vedotin plus pembrolizumab (EVP) is the standard first-line treatment for metastatic or locally advanced urothelial carcinoma (m/laUC). Real-world patients frequently present with unfavorable clinical characteristics, raising uncertainty about whether outcomes mirror those of the pivotal EV-302/KEYNOTE-A39 trial. This study assessed effectiveness and safety of EVP in a real-world cohort of patients with m/laUC.</p> Materials and methods <p>Retrospective multicenter study including patients treated with EVP across 25 German hospitals (between 03/2022 and 08/2025). Treatment delivery and monitoring followed local clinical standards. Adverse events (AEs) were documented per CTCAE v5.0. Responses were assessed locally using RECIST v1.1. Progression-free survival (PFS) and overall survival (OS) were analyzed using Kaplan–Meier methodology.</p> Results <p>A total of 468 patients (median age = 69&#xa0;years) were included. The overall response rate (ORR) was 50.2% (partial response:37.4%, complete response:12.8%). Median PFS was 10.2&#xa0;months (95%CI,8.0–12.7). 12-month and 24-month OS rates were 71.9% and 60.6%, respectively. ECOG PS 0–1 and occurrence of skin toxicity were associated with improved outcomes. Any-grade AEs occurred in 81.8% and grade ≥ 3 AEs in 35.8%. Peripheral sensory neuropathy and skin toxicity were the most common AEs. Ten treatment-related fatal events were recorded.</p> Conclusions <p>EVP demonstrated robust effectiveness and manageable toxicity in a large and diverse real-world cohort including patients with divergent histology. Skin toxicity was associated with superior ORR, PFS, and OS. Our findings support EVP as an effective first-line option for patients with m/laUC treated outside clinical trials.</p>

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Effectiveness and safety of enfortumab vedotin and pembrolizumab in a real-world patient population with urothelial carcinoma: results from a multi-institutional cohort (GUARDIANS)

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  • 发表日期：

    2026年7月12日

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  • Stefanie Zschäbitz,
  • Irfan Bhatti,
  • Jozefina Casuscelli,
  • Ludwig Otto Pachmayr,
  • Thomas Büttner,
  • Thomas Elegeert,
  • Nina Holzwarth,
  • Severin Rodler,
  • Martin Hennig,
  • Emily Rinderknecht,
  • Muammar Dib,
  • Katrin Schlack,
  • Christopher Darr,
  • Anna-Lisa Volk,
  • Philipp Schmucker,
  • Friedemann Zengerling,
  • Pia Paffenholz,
  • Joschka Heil,
  • Eva Erne,
  • Subhajit Mandal,
  • Frederik Wessels,
  • Thea Maria Busse,
  • Daniel Seidl,
  • Susan Foller,
  • Alexander Höllein,
  • Tim Nestler,
  • Christian Gratzke,
  • Louis Rhode,
  • Jakob Kohler,
  • Günter Niegisch,
  • Anna Katharina Seitz,
  • Julia Heinzelbecker,
  • Niklas Klümper,
  • Margitta Retz,
  • Viktor Grünwald,
  • Can Aydogdu,
  • Marco Julius Schnabel

摘要

Introduction

Enfortumab vedotin plus pembrolizumab (EVP) is the standard first-line treatment for metastatic or locally advanced urothelial carcinoma (m/laUC). Real-world patients frequently present with unfavorable clinical characteristics, raising uncertainty about whether outcomes mirror those of the pivotal EV-302/KEYNOTE-A39 trial. This study assessed effectiveness and safety of EVP in a real-world cohort of patients with m/laUC.

Materials and methods

Retrospective multicenter study including patients treated with EVP across 25 German hospitals (between 03/2022 and 08/2025). Treatment delivery and monitoring followed local clinical standards. Adverse events (AEs) were documented per CTCAE v5.0. Responses were assessed locally using RECIST v1.1. Progression-free survival (PFS) and overall survival (OS) were analyzed using Kaplan–Meier methodology.

Results

A total of 468 patients (median age = 69 years) were included. The overall response rate (ORR) was 50.2% (partial response:37.4%, complete response:12.8%). Median PFS was 10.2 months (95%CI,8.0–12.7). 12-month and 24-month OS rates were 71.9% and 60.6%, respectively. ECOG PS 0–1 and occurrence of skin toxicity were associated with improved outcomes. Any-grade AEs occurred in 81.8% and grade ≥ 3 AEs in 35.8%. Peripheral sensory neuropathy and skin toxicity were the most common AEs. Ten treatment-related fatal events were recorded.

Conclusions

EVP demonstrated robust effectiveness and manageable toxicity in a large and diverse real-world cohort including patients with divergent histology. Skin toxicity was associated with superior ORR, PFS, and OS. Our findings support EVP as an effective first-line option for patients with m/laUC treated outside clinical trials.