131I-mIBG therapy in relapsed/refractory neuroblastoma: A weapon from the future past.

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

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i - mibg治疗复发/难治性神经母细胞瘤:来自未来的武器。
神经母细胞瘤(Neuroblastoma, NB)是儿童中最常见的颅外实体瘤,其结局多种多样,从自发缓解到高风险病例,往往导致复发或难治性疾病。大约50%的NB患者具有高风险特征,尽管进行了强化治疗,但仍经常复发或难治性疾病,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基胍(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方面显示出希望。自1985年以来,人们对I-mIBG进行了研究,以确定最大耐受剂量和副作用,最近的试验探索其在一线治疗中的应用。我们的系统综述基于MEDLINE、EMBASE和Cochrane CENTRAL数据库,截止到2023年12月,评估了1³1 I-mIBG治疗复发/难治性NB的有效性和毒性。它还讨论了它与新兴疗法如CAR-T细胞、单倍体干细胞移植和迪努妥昔单抗结合的潜在作用。
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