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

Abstract

Abstract 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 a “front‐line” therapeutic 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.

Keywords

• Neuroblastoma
• 131I‐mIBG
• Transplantation