Feng Jianhua, Cheng Frankie Wt, Leung Alex Wk, Lee Vincent, Yeung Eva Wm, Ching Lam Hoi, Cheung Jeanny, Lam Grace Ks, Chow Terry Tw, Yan Carol Ls, Kong Li Chi
Department of Paediatrics The Chinese University of Hong Kong Hong Kong China.
Department of Paediatrics The First Affiliated Hospital of Wenzhou Medical University Wenzhou China.
Pediatr Investig. 2020 Sep 27;4(3):168-177. doi: 10.1002/ped4.12216. eCollection 2020 Sep.
I-metaiodobenzylguanidine (I-mIBG) has a significant targeted antitumor effect for neuroblastoma. However, currently there is a paucity of data for the use of I-mIBG as a "front-line" therapeutic agent in those patients with newly diagnosed high-risk neuroblastoma as part of the conditioning regimen for myeloablative chemotherapy (MAC).
To evaluate the feasibility of upfront consolidation treatment with I-mIBG plus MAC and hematopoietic stem cell transplantation (HSCT) in high-risk neuroblastoma patients.
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 I-mIBG infusion and MAC followed by HSCT.
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 I-mIBG treatment achieved either complete response (CR) ( 1) or very good partial response (VGPR) ( 2) after HSCT. With a median follow-up duration of 13.0 months after I-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 I-mIBG treatment.
Upfront consolidation treatment with I-mIBG plus MAC and HSCT is feasible and tolerable in high-risk neuroblastoma patients, however the survival benefit of this I-mIBG regimen is only observed in the patients who were in CR/VGPR at the time of I-mIBG treatment.
间碘苄胍（I - mIBG）对神经母细胞瘤具有显著的靶向抗肿瘤作用。然而，目前在新诊断的高危神经母细胞瘤患者中，将I - mIBG作为清髓性化疗（MAC）预处理方案的“一线”治疗药物的数据较少。
评估I - mIBG联合MAC及造血干细胞移植（HSCT）进行 upfront 巩固治疗在高危神经母细胞瘤患者中的可行性。
对2003年至2019年新诊断的高危神经母细胞瘤患者进行回顾性单中心研究，这些患者在诱导治疗完成后无疾病进展（PD）。他们接受了I - mIBG输注和MAC，随后进行HSCT。
共纳入24例高危神经母细胞瘤患者，诊断时的中位年龄为3.0岁。接受这种序贯巩固治疗后，13例在I - mIBG治疗前为部分缓解（PR）的患者中有3例在HSCT后达到完全缓解（CR）（1例）或非常好的部分缓解（VGPR）（2例）。I - mIBG治疗后的中位随访时间为13.0个月，整个队列的5年无事件生存率和总生存率估计分别为29%和38%，I - mIBG治疗时处于CR/VGPR的患者分别为53%和67%。
I - mIBG联合MAC及HSCT进行 upfront 巩固治疗在高危神经母细胞瘤患者中是可行且可耐受的，然而这种I - mIBG方案的生存获益仅在I - mIBG治疗时处于CR/VGPR的患者中观察到。
