I-mIBG therapy in relapsed/refractory neuroblastoma: A weapon from the future past.

Elizabeth Katherine Anna Triumbari, Francesco Fabozzi, Francesca Del Bufalo, Sabina Vennarini, Maria Felicia Villani, Maria Antonietta De Ioris, Angela Di Giannatale, Angela Mastronuzzi, Claudio Altini, Vittorio Cannatà, Ciucci Davide, Milena Pizzoferro, Margherita Drago, Federica D'Antonio, Maria Carmen Garganese

Journal: Critical reviews in oncology/hematology 2025;207():104606

PMID: 39732302

Abstract

Neuroblastoma (NB) is the most common extracranial solid tumor in children, with variable outcomes ranging from spontaneous remission to high-risk cases often leading to relapse or refractory disease. Approximately 50 % of patients with NB have high-risk features, often experiencing relapse or refractory disease despite intensive treatments and the prognosis remains poor, with long-term event-free survival (EFS) rates below 10 %,Radioactive iodine-labeled meta-iodobenzylguanidine (¹³¹I-mIBG) therapy, leveraging NB cells' radiosensitivity and expression of the norepinephrine transporter (NET), has shown promise in treating relapsed or refractory NB. Since 1985, ¹³¹I-mIBG has been studied to determine the maximum tolerated dose and side effects, with recent trials exploring its use in front-line treatment. Our systematic review, based on MEDLINE, EMBASE, and Cochrane CENTRAL databases up to December 2023, evaluates the effectiveness and toxicity of ¹³¹I-mIBG therapy in relapsed/refractory NB. It also discusses its potential role in conjunction with emerging therapies like CAR-T cells, haploidentical stem cell transplantation, and dinutuximab beta.

Copyright © 2025 Elsevier B.V. All rights reserved.

Address: Paediatric Haematology and Oncology, Cell and Gene Therapy, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.; Nuclear Medicine Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.; Medical Physics Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.; Pediatric Radiotherapy Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy.; Paediatric Haematology and Oncology, Cell and Gene Therapy, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy. Electronic address: [email protected].
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