Feasibility of high-dose iodine-131-metaiodobenzylguanidine therapy for high-risk neuroblastoma preceding myeloablative chemotherapy and hematopoietic stem cell transplantation: a study protocol

Objective(s): High-risk neuroblastoma is a childhood cancer with poorprognosis despite modern multimodality therapy. Internal radiotherapy using131I-metaiodobenzylguanidine (MIBG) is effective for treating the disease even if it isresistant to chemotherapy. The aim of this study is to evaluate the safety and efficacyof 131I-MIBG radiotherapy combined with myeloablative high-dose chemotherapyand hematopoietic stem cell transplantation.Methods: Patients with high-risk neuroblastoma will be enrolled in this study. A totalof 8 patients will be registered. Patients will receive 666 MBq/kg of 131I-MIBG andafter safety evaluation will undergo high-dose chemotherapy and hematopoietic stemcell transplantation. Autologous and allogeneic stem cell sources will be accepted.After engraftment or 28 days after hematopoietic stem cell transplantation, the safetyand response will be evaluated.Conclusion: This is the first prospective study of 131I-MIBG with high-dosechemotherapy and hematopoietic stem cell transplantation in Japan. The results willbe the basis of a future nationwide clinical trial.