Fabozzi Francesco, Villani Maria Felicia, Del Bufalo Francesca, Altini Claudio, Cannatà Vittorio, Davide Ciucci, Pizzoferro Milena, Drago Margherita, D'Antonio Federica, Triumbari Elizabeth Katherine Anna, Di Giannatale Angela, Vennarini Sabina, Mastronuzzi Angela, De Ioris Maria Antonietta, Garganese Maria Carmen
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.
Crit Rev Oncol Hematol. 2025 Mar;207:104606. doi: 10.1016/j.critrevonc.2024.104606. Epub 2024 Dec 26.
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.
神经母细胞瘤(NB)是儿童最常见的颅外实体瘤,其预后差异很大,从自发缓解到高危病例,常导致复发或难治性疾病。约50%的NB患者具有高危特征,尽管接受了强化治疗,仍常出现复发或难治性疾病,预后仍然很差,长期无事件生存率(EFS)低于10%。放射性碘标记的间碘苄胍(¹³¹I-mIBG)治疗利用NB细胞的放射敏感性和去甲肾上腺素转运体(NET)的表达,在治疗复发或难治性NB方面显示出前景。自1985年以来,人们一直在研究¹³¹I-mIBG以确定其最大耐受剂量和副作用,最近的试验探索了其在一线治疗中的应用。我们基于截至2023年12月的MEDLINE、EMBASE和Cochrane CENTRAL数据库进行的系统评价,评估了¹³¹I-mIBG治疗复发/难治性NB的有效性和毒性。它还讨论了其与CAR-T细胞、单倍体相合干细胞移植和地努图希单抗β等新兴疗法联合使用的潜在作用。