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Lookup NU author(s): Professor Deborah Tweddle
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© 2024 by American Society of Clinical Oncology.PURPOSE Outcomes for children with relapsed and refractory high-risk neuroblastoma (RR-HRNB) remain dismal. The BEACON Neuroblastoma trial (EudraCT 2012-000072-42) evaluated three backbone chemotherapy regimens and the addition of the antiangiogenic agent bevacizumab (B). MATERIALS Patients age 1-21 years with RR-HRNB with adequate organ function and AND METHODS performance status were randomly assigned in a 3 3 2 factorial design to temozolomide (T), irinotecan-temozolomide (IT), or topotecan-temozolomide (TTo) with or without B. The primary end point was best overall response (complete or partial) rate (ORR) during the first six courses, by RECIST or International Neuroblastoma Response Criteria for patients with measurable or evaluable disease, respectively. Safety, progression-free survival (PFS), and overall survival (OS) time were secondary end points. RESULTS One hundred sixty patients with RR-HRNB were included. For B random assignment (n 5 160), the ORR was 26% (95% CI, 17 to 37) with B and 18% (95% CI, 10 to 28) without B (risk ratio [RR], 1.52 [95% CI, 0.83 to 2.77]; P 5 .17). Adjusted hazard ratio for PFS and OS were 0.89 (95% CI, 0.63 to 1.27) and 1.01 (95% CI, 0.70 to 1.45), respectively. For irinotecan ([I]; n 5 121) and topotecan (n 5 60) random assignments, RRs for ORR were 0.94 and 1.22, respectively. A potential interaction between I and B was identified. For patients in the bevacizumab-irinotecan-temozolomide (BIT) arm, the ORR was 23% (95% CI, 10 to 42), and the 1-year PFS estimate was 0.67 (95% CI, 0.47 to 0.80). CONCLUSION The addition of B met protocol-defined success criteria for ORR and appeared to improve PFS. Within this phase II trial, BIT showed signals of antitumor activity with acceptable tolerability. Future trials will confirm these results in the chemoimmunotherapy era.
Author(s): Moreno L, Weston R, Owens C, Valteau-Couanet D, Gambart M, Castel V, Zwaan CM, Nysom K, Gerber N, Castellano A, Laureys G, Ladenstein R, Rossler J, Makin G, Murphy D, Morland B, Vaidya S, Thebaud E, van Eijkelenburg N, Barone G, Tandonnet J, Corradini N, Chastagner P, Paillard C, Bautista FJ, Melcon SG, De Wilde B, Marshall L, Gray J, Burchill SA, Schleiermacher G, Chesler L, Peet A, Leach MO, McHugh K, Hayes R, Jerome N, Caron H, Laidler J, Fenwick N, Holt G, Moroz V, Kearns P, Gates S, Pearson ADJ, Wheatley K, Thebaut E, Hobin D, Elliott M, Tweddle D, Jannier S, Gallego S, Coze C, Auvrignon A, Defachelles AS, Almaraz RL, Brichard B, Luksch R, Beck-Popovic M, Willis C, Hettmer S, Schweigerer L, Kontny U, Christiansen H, Garaventa A, Ng A, Howell L, Yeomanson D
Publication type: Article
Publication status: Published
Journal: Journal of Clinical Oncology
Year: 2024
Volume: 42
Issue: 10
Pages: 1135-1145
Print publication date: 01/04/2024
Online publication date: 08/01/2024
Acceptance date: 05/10/2023
Date deposited: 03/03/2025
ISSN (print): 0732-183X
ISSN (electronic): 1527-7755
Publisher: Lippincott Williams and Wilkins
URL: https://doi.org/10.1200/jco.23.00458
DOI: 10.1200/JCO.23.00458
PubMed id: 38190578
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