Avelumab-based neoadjuvant therapy in patients with muscle-invasive bladder cancer (AURA Oncodistinct-004): a phase 2 multicenter clinical trial

Background Immunotherapy is becoming a standard of care for non-metastatic muscle-invasive bladder cancer (MIBC). The optimal chemotherapy partner for chemo-immunotherapy combinations remains unknown. We evaluated the efficacy and safety of neoadjuvant avelumab-based regimens in patients with MIBC.M...

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Main Authors: Emmanuel Seront, Ahmad Awada, Nieves Martinez Chanza, Philippe Barthélémy, Lieveke Ameye, Françoise Rothé, Aurélien Carnot, Nuria Kotecki, Sandrine Rorive, Thibault Tricard, Jérémy Blanc, Vinciane Casert, Brieuc Sautois, Jan Van den Brande, Vincent Vanhaudenarde, Lionel Staudacher, Veronique Debien, Jean-Christophe Fantoni, Thierry Roumeguère
Format: Article
Language:English
Published: BMJ Publishing Group 2025-05-01
Series:Journal for ImmunoTherapy of Cancer
Online Access:https://jitc.bmj.com/content/13/5/e012045.full
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author Emmanuel Seront
Ahmad Awada
Nieves Martinez Chanza
Philippe Barthélémy
Lieveke Ameye
Françoise Rothé
Aurélien Carnot
Nuria Kotecki
Sandrine Rorive
Thibault Tricard
Jérémy Blanc
Vinciane Casert
Brieuc Sautois
Jan Van den Brande
Vincent Vanhaudenarde
Lionel Staudacher
Veronique Debien
Jean-Christophe Fantoni
Thierry Roumeguère
author_facet Emmanuel Seront
Ahmad Awada
Nieves Martinez Chanza
Philippe Barthélémy
Lieveke Ameye
Françoise Rothé
Aurélien Carnot
Nuria Kotecki
Sandrine Rorive
Thibault Tricard
Jérémy Blanc
Vinciane Casert
Brieuc Sautois
Jan Van den Brande
Vincent Vanhaudenarde
Lionel Staudacher
Veronique Debien
Jean-Christophe Fantoni
Thierry Roumeguère
author_sort Emmanuel Seront
collection DOAJ
description Background Immunotherapy is becoming a standard of care for non-metastatic muscle-invasive bladder cancer (MIBC). The optimal chemotherapy partner for chemo-immunotherapy combinations remains unknown. We evaluated the efficacy and safety of neoadjuvant avelumab-based regimens in patients with MIBC.Methods The multicenter phase 2 AURA trial (NCT03674424) enrolled patients with non-metastatic MIBC undergoing radical cystectomy. Cisplatin-eligible patients were randomized to receive avelumab with either dose-dense methotrexate-vinblastine-doxorubicin-cisplatin (ddMVAC-A) or gemcitabine-cisplatin (GC-A). Cisplatin-ineligible patients received either avelumab alone (A) or combined with paclitaxel-gemcitabine (PG-A). The primary endpoint was pathological complete response (pCR). Secondary endpoints included safety, event-free survival, and overall survival (OS).Results Between July 2018 and September 2021, 137 eligible patients were enrolled in the trial. In the cisplatin-eligible cohort (n=79), pCR rates were 58% (95% CI: 42% to 72%) in the ddMVAC-A arm and 53% (95% CI: 37% to 68%) in the GC-A arm. The 36-month OS rates were 87% (95% CI: 76% to 98%) for ddMVAC-A and 67% (95% CI: 53% to 84%) for GC-A. In the cisplatin-ineligible cohort (n=58), pCR rates were 14% (95% CI: 6% to 31%) in the PG-A arm and 32% (95% CI: 18% to 51%) in the A arm. The 36-month OS rates were 48% (95% CI: 33% to 71%) for PG-A and 42% (95% CI: 27% to 65%) for A. Overall, 51 (38%) patients experienced grade 3–4 treatment-related adverse events.Conclusions Avelumab combined with cisplatin-based neoadjuvant chemotherapy showed promising efficacy in MIBC with a favorable safety profile, also with the ddMVAC regimen. Among cisplatin-ineligible patients, avelumab monotherapy showed encouraging activity, with no additional benefit observed from the PG-A regimen. These results support the use of the ddMVAC regimen as a potential chemotherapy partner for neoadjuvant chemo-immunotherapy combinations in future phase 3 trials, providing an alternative to the GC regimen currently under investigation.Trial registration number NCT03674424.
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spelling doaj-art-2f5c846d69714cdd9f67e5403ab3c0612025-08-20T01:52:22ZengBMJ Publishing GroupJournal for ImmunoTherapy of Cancer2051-14262025-05-0113510.1136/jitc-2025-012045Avelumab-based neoadjuvant therapy in patients with muscle-invasive bladder cancer (AURA Oncodistinct-004): a phase 2 multicenter clinical trialEmmanuel Seront0Ahmad Awada1Nieves Martinez Chanza2Philippe Barthélémy3Lieveke Ameye4Françoise Rothé5Aurélien Carnot6Nuria Kotecki7Sandrine Rorive8Thibault Tricard9Jérémy Blanc10Vinciane Casert11Brieuc Sautois12Jan Van den Brande13Vincent Vanhaudenarde14Lionel Staudacher15Veronique Debien16Jean-Christophe Fantoni17Thierry Roumeguère189 Medical Oncology Department, Cliniques universitaires Saint-Luc, Brussels, Belgium1 Medical Oncology Department, Institut Jules Bordet, Hôpital Universitaire de Bruxelles, Brussels, Belgium1 Medical Oncology Department, Institut Jules Bordet, Hôpital Universitaire de Bruxelles, Brussels, Belgium3 Medical Oncology Department, ICANS, Strasbourg, France10 Clinical Trials Center, Institut Jules Bordet, Hôpital Universitaire de Bruxelles, Brussels, Belgium11 Breast Cancer Translational research Laboratory, Institut Jules Bordet, Hôpital Universitaire de Bruxelles, Brussels, Belgium2 Medical Oncology Department, Centre Oscar Lambret, Lille, France1 Medical Oncology Department, Institut Jules Bordet, Hôpital Universitaire de Bruxelles, Brussels, Belgium12 Pathology Department, CurePath, Jumet, Belgium14 Urology Department, Hôpitaux Universitaires de Strasbourg, Strasbourg, France1 Medical Oncology Department, Institut Jules Bordet, Hôpital Universitaire de Bruxelles, Brussels, Belgium4 Medical Oncology Department, Centre Hospitalier Universitaire Ambroise Paré, Mons, Belgium5 Medical Oncology Department, CHU de Liège Hôpital du Sart Tilman, Liège, Belgium6 Medical Oncology Department, University Hospital Antwerp, Edegem, Belgium7 Medical Oncology Department, CHU UCL Namur, Namur, Belgium8 Medical Oncology Department, Hôpital Paris Saint-Joseph, Paris, France1 Medical Oncology Department, Institut Jules Bordet, Hôpital Universitaire de Bruxelles, Brussels, Belgium13 Urology Department, Centre Hospitalier Universitaire de Lille, Lille, France15 Urology Department, Institut Jules Bordet, Hôpital Universitaire de Bruxelles, Brussels, BelgiumBackground Immunotherapy is becoming a standard of care for non-metastatic muscle-invasive bladder cancer (MIBC). The optimal chemotherapy partner for chemo-immunotherapy combinations remains unknown. We evaluated the efficacy and safety of neoadjuvant avelumab-based regimens in patients with MIBC.Methods The multicenter phase 2 AURA trial (NCT03674424) enrolled patients with non-metastatic MIBC undergoing radical cystectomy. Cisplatin-eligible patients were randomized to receive avelumab with either dose-dense methotrexate-vinblastine-doxorubicin-cisplatin (ddMVAC-A) or gemcitabine-cisplatin (GC-A). Cisplatin-ineligible patients received either avelumab alone (A) or combined with paclitaxel-gemcitabine (PG-A). The primary endpoint was pathological complete response (pCR). Secondary endpoints included safety, event-free survival, and overall survival (OS).Results Between July 2018 and September 2021, 137 eligible patients were enrolled in the trial. In the cisplatin-eligible cohort (n=79), pCR rates were 58% (95% CI: 42% to 72%) in the ddMVAC-A arm and 53% (95% CI: 37% to 68%) in the GC-A arm. The 36-month OS rates were 87% (95% CI: 76% to 98%) for ddMVAC-A and 67% (95% CI: 53% to 84%) for GC-A. In the cisplatin-ineligible cohort (n=58), pCR rates were 14% (95% CI: 6% to 31%) in the PG-A arm and 32% (95% CI: 18% to 51%) in the A arm. The 36-month OS rates were 48% (95% CI: 33% to 71%) for PG-A and 42% (95% CI: 27% to 65%) for A. Overall, 51 (38%) patients experienced grade 3–4 treatment-related adverse events.Conclusions Avelumab combined with cisplatin-based neoadjuvant chemotherapy showed promising efficacy in MIBC with a favorable safety profile, also with the ddMVAC regimen. Among cisplatin-ineligible patients, avelumab monotherapy showed encouraging activity, with no additional benefit observed from the PG-A regimen. These results support the use of the ddMVAC regimen as a potential chemotherapy partner for neoadjuvant chemo-immunotherapy combinations in future phase 3 trials, providing an alternative to the GC regimen currently under investigation.Trial registration number NCT03674424.https://jitc.bmj.com/content/13/5/e012045.full
spellingShingle Emmanuel Seront
Ahmad Awada
Nieves Martinez Chanza
Philippe Barthélémy
Lieveke Ameye
Françoise Rothé
Aurélien Carnot
Nuria Kotecki
Sandrine Rorive
Thibault Tricard
Jérémy Blanc
Vinciane Casert
Brieuc Sautois
Jan Van den Brande
Vincent Vanhaudenarde
Lionel Staudacher
Veronique Debien
Jean-Christophe Fantoni
Thierry Roumeguère
Avelumab-based neoadjuvant therapy in patients with muscle-invasive bladder cancer (AURA Oncodistinct-004): a phase 2 multicenter clinical trial
Journal for ImmunoTherapy of Cancer
title Avelumab-based neoadjuvant therapy in patients with muscle-invasive bladder cancer (AURA Oncodistinct-004): a phase 2 multicenter clinical trial
title_full Avelumab-based neoadjuvant therapy in patients with muscle-invasive bladder cancer (AURA Oncodistinct-004): a phase 2 multicenter clinical trial
title_fullStr Avelumab-based neoadjuvant therapy in patients with muscle-invasive bladder cancer (AURA Oncodistinct-004): a phase 2 multicenter clinical trial
title_full_unstemmed Avelumab-based neoadjuvant therapy in patients with muscle-invasive bladder cancer (AURA Oncodistinct-004): a phase 2 multicenter clinical trial
title_short Avelumab-based neoadjuvant therapy in patients with muscle-invasive bladder cancer (AURA Oncodistinct-004): a phase 2 multicenter clinical trial
title_sort avelumab based neoadjuvant therapy in patients with muscle invasive bladder cancer aura oncodistinct 004 a phase 2 multicenter clinical trial
url https://jitc.bmj.com/content/13/5/e012045.full
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