Upfront consolidation treatment with 131I-mIBG followed by myeloablative chemotherapy and hematopoietic stem cell transplantation in high-risk neuroblastoma

作     者：Jianhua Feng Frankie WT Cheng Alex WK Leung Vincent Lee Eva WM Yeung Hoi Ching Lam Jeanny Cheung Grace KS Lam Terry TW Chow Carol LS Yan Chi Kong Li Feng Jianhua;Cheng Frankie WT;Leung Alex WK;Lee Vincent;Yeung Eva WM;Lam Hoi Ching;Cheung Jeanny;Lam Grace KS;Chow Terry TW;Yan Carol LS;Li Chi Kong

作者机构：Department of PaediatricsThe Chinese University of Hong KongHong KongChina Department of PaediatricsThe First Affiliated Hospital of Wenzhou Medical UniversityWenzhouChina Department of Paediatrics and Adolescent MedicineHong Kong Children’s HospitalHong KongChina Department of Clinical OncologyPrince of Wales HospitalThe Chinese University of Hong KongHong KongChina 

出 版 物：《Pediatric Investigation》 (儿科学研究（英文）)

年 卷 期：2020年第4卷第3期

页      面：168-177页

学科分类：1004[医学-公共卫生与预防医学(可授医学、理学学位)] 1002[医学-临床医学] 100214[医学-肿瘤学] 10[医学] 

主　　题：Neuroblastoma 131I-mIBG Transplantation 

摘      要：Importance:131I-metaiodobenzylguanidine(131I-mIBG)has a significant targeted antitumor effect for neuroblastoma.However,currently there is a paucity of data for the use of 131I-mIBG as afront-linetherapeutic agent in those patients with newly diagnosed high-risk neuroblastoma as part of the conditioning regimen for myeloablative chemotherapy(MAC).Objective:To evaluate the feasibility of upfront consolidation treatment with 131I-mIBG plus MAC and hematopoietic stem cell transplantation(HSCT)in high-risk neuroblastoma patients.Methods:A retrospective,single-center study was conducted from 2003-2019 on newly diagnosed high-risk neuroblastoma patients without progressive disease(PD)after the completion of induction therapy.They received 131I-mIBG infusion and MAC followed by HSCT.Results:A total of 24 high-risk neuroblastoma patients were enrolled with a median age of 3.0 years at diagnosis.After receiving this sequential consolidation treatment,3 of 13 patients who were in partial response(PR)before 131I-mIBG treatment achieved either complete response(CR)(n=1)or very good partial response(VGPR)(n=2)after HSCT.With a median follow-up duration of 13.0 months after 131I-mIBG therapy,the 5-year event-free survival and overall survival rates estimated were 29%and 38%for the entire cohort,and 53%and 67%for the patients who were in CR/VGPR at the time of 131I-mIBG treatment.Interpretation:Upfront consolidation treatment with 131I-mIBG plus MAC and HSCT is feasible and tolerable in high-risk neuroblastoma patients,however the survival benefit of this 131I-mIBG regimen is only observed in the patients who were in CR/VGPR at the time of 131I-mIBG treatment.