Phase I/II clinical trial of high-dose [131I] meta-iodobenzylguanidine therapy for high-risk neuroblastoma preceding single myeloablative chemotherapy and haematopoietic stem cell transplantation

Purpose Paediatric high-risk neuroblastoma has poor prognosis despite modern multimodality therapy. This phase I/II study aimed to determine the safety, dose-limiting toxicity (DLT), and efficacy of high-dose 131 I-meta-iodobenzylguanidine ( 131 I-mIBG) therapy combined with single high-dose chemoth...

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Bibliographic Details
Published in:European journal of nuclear medicine and molecular imaging 2022-04, Vol.49 (5), p.1574-1583
Main Authors: Kuroda, Rie, Wakabayashi, Hiroshi, Araki, Raita, Inaki, Anri, Nishimura, Ryosei, Ikawa, Yasuhiro, Yoshimura, Kenichi, Murayama, Toshinori, Imai, Yasuhito, Funasaka, Tatsuyoshi, Wada, Taizo, Kinuya, Seigo
Format: Article
Language:English
Subjects:
3-Iodobenzylguanidine - administration & dosage
3-Iodobenzylguanidine - adverse effects
Adverse events
Autografts
Cardiology
Chemotherapy
Child
Child, Preschool
Female
Hematopoietic Stem Cell Transplantation
Hematopoietic stem cells
Humans
Imaging
Infant
Iodine
Iodine 131
Iodine isotopes
Iodine Radioisotopes
Male
Medicine
Medicine & Public Health
Myelosuppression
Neuroblastoma
Neuroblastoma - radiotherapy
Nuclear Medicine
Oncology
Original Article
Orthopedics
Patients
Pediatric
Radiology
Risk
Scintigraphy
Solid tumors
Stem cell transplantation
Stem cells
Toxicity
Transplantation
Transplantation, Autologous
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Summary:Purpose Paediatric high-risk neuroblastoma has poor prognosis despite modern multimodality therapy. This phase I/II study aimed to determine the safety, dose-limiting toxicity (DLT), and efficacy of high-dose 131 I-meta-iodobenzylguanidine ( 131 I-mIBG) therapy combined with single high-dose chemotherapy (HDC) and haematopoietic stem cell transplantation (HSCT) in high-risk neuroblastoma in Japan. Methods Patients received 666 MBq/kg of 131 I-mIBG and single HDC and HSCT from autologous or allogeneic stem cell sources. The primary endpoint was DLT defined as adverse events associated with 131 I-mIBG treatment posing a significant obstacle to subsequent HDC. The secondary endpoints were adverse events/reactions, haematopoietic stem cell engraftment and responses according to the Response Evaluation Criteria in Solid Tumours version 1.1 (RECIST 1.1) and 123 I-mIBG scintigraphy. Response was evaluated after engraftment. Results We enrolled eight patients with high-risk neuroblastoma (six females; six newly diagnosed and two relapsed high-risk neuroblastoma; median age, 4 years; range, 1–10 years). Although all patients had adverse events/reactions after high-dose 131 I-mIBG therapy, we found no DLT. Adverse events and reactions were observed in 100% and 25% patients during single HDC and 100% and 12.5% patients during HSCT, respectively. No Grade 4 complications except myelosuppression occurred during single HDC and HSCT. The response rate according to RECIST 1.1 was observed in 87.5% (7/8) in stable disease and 12.5% (1/8) were not evaluated. Scintigraphic response occurred in 62.5% (5/8) and 37.5% (3/8) patients in complete response and stable disease, respectively. Conclusion 131 I-mIBG therapy with 666 MBq/kg followed by single HDC and autologous or allogeneic SCT is safe and efficacious in patients with high-risk neuroblastoma and has no DLT. Trial registration number jRCTs041180030. Name of registry Feasibility of high-dose iodine-131-meta-iodobenzylguanidine therapy for high-risk neuroblastoma preceding myeloablative chemotherapy and haematopoietic stem cell transplantation (High-dose iodine-131-meta-iodobenzylguanidine therapy for high-risk neuroblastoma). URL of registry https://jrct.niph.go.jp/en-latest-detail/jRCTs041180030 . Date of enrolment of the first participant to the trial 12/01/2018.
ISSN:1619-7070
1619-7089
DOI:10.1007/s00259-021-05630-7