Altini Claudio, Villani Maria F, Di Giannatale Angela, Cassano Bartolomeo, Pizzoferro Milena, Serra Annalisa, Castellano Aurora, Cannatà Vittorio, Garganese Maria C
Imaging Department, Nuclear Medicine Unit, IRCCS Bambino Gesù Children's Hospital.
Paediatric Haematology/Oncology Department, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
Nucl Med Commun. 2022 Feb 1;43(2):129-144. doi: 10.1097/MNM.0000000000001496.
131I-meta-iodobenzylguanidine (131I-MIBG) combined with myeloablative chemotherapy represents an effective treatment in children affected by relapsed/refractory neuroblastoma (NBL) for disease palliation and in improving progression-free survival. The aim of our study is to evaluate the feasibility, safety and efficacy of tandem 131I-MIBG followed by high-dose chemotherapy with Melphalan.
Thirteen patients (age range: 3-17 years) affected by relapsed/refractory NB, previously treated according to standard procedures, were included in the study. Each treatment cycle included two administrations of 131I-MIBG (with a dosimetric approach) followed by a single dose of Melphalan with peripheral blood stem cell rescue.
At the end of the treatment, ten patients experienced grade 4 neutropenia, two grade 3 and one patient grade 2, three patients presented febrile neutropenia and all needed RBC and platelets transfusions; one patient presented grade 4 mucositis, four grade 3 and one patient grade 2 mucositis. One patient showed progressive disease, eight patients showed stable disease and four patients showed partial response.
High-dose 131I-MIBG therapy combined with chemotherapy represent a well-tolerated and effective modality of treatment in heavily pretreated patients affected by relapsed/refractory NBL. However, further studies, including a wider cohort of patients, are needed.
131I-间碘苄胍（131I-MIBG）联合清髓性化疗是治疗复发/难治性神经母细胞瘤（NBL）患儿以缓解疾病及提高无进展生存期的有效方法。本研究旨在评估序贯131I-MIBG后给予美法仑大剂量化疗的可行性、安全性及疗效。
本研究纳入13例复发/难治性NB患儿（年龄范围：3 - 17岁），这些患儿此前已按照标准程序接受过治疗。每个治疗周期包括两次131I-MIBG给药（采用剂量测定法），随后给予单剂量美法仑并进行外周血干细胞救援。
治疗结束时，10例患者出现4级中性粒细胞减少，2例为3级，1例为2级；3例患者出现发热性中性粒细胞减少，所有患者均需要输注红细胞和血小板；1例患者出现4级黏膜炎，4例为3级，1例为2级黏膜炎。1例患者疾病进展，8例患者疾病稳定，4例患者部分缓解。
131I-MIBG大剂量疗法联合化疗对于复发/难治性NBL的 heavily pretreated患者是一种耐受性良好且有效的治疗方式。然而，需要进一步开展研究，包括纳入更广泛的患者队列。 （注：“heavily pretreated”直译为“大量预处理过的”，结合语境这里可能是指之前经过多种治疗的意思，但表述不太准确，可根据具体医学背景进一步优化）
