Feasibility and effectiveness of treatment strategy of tandem high‐dose chemotherapy and autologous stem cell transplantation in combination with 131I‐MIBG therapy for high‐risk neuroblastoma
Abstract
This study was performed to evaluate the safety and effectiveness of tandem HDCT/ASCT combined with targeted radiotherapy using 131 I‐MIBG for high‐risk neuroblastoma. Patients with high‐risk neuroblastoma were treated with 8 to 10 cycles of induction chemotherapy before tandem HDCT/ASCT. Patients received 131 I‐MIBG treatment before the second HDCT/ASCT. Local radiotherapy and maintenance therapy were performed after tandem HDCT/ASCT. Between 2012 and 2016, 19 patients were diagnosed with high‐risk neuroblastoma in our institution and 18 of them received tandem HDCT/ASCT combined with 131 I‐MIBG therapy. For the first HDCT/ASCT regimen, 12 patients received busulfan/melphalan and six patients received melphalan/etoposide/carboplatin. The second HDCT included ThioCy. The median dose of 131 I‐MIBG was 17.2 mCi/kg for the first eight patients, while 12 patients in the latter period of the study received reduced dose of 10.7 mCi/kg. The 5‐year OS and EFS rates were 79% and 61%, respectively, for all 19 patients with high‐risk neuroblastoma, and 83% and 64%, respectively, for 18 patients who completed tandem HDCT/ASCT combined with 131 I‐MIBG therapy. Six patients experienced disease relapse and five patients died. Treatment‐related mortality was not observed. Among 15 evaluable patients, 11 patients (73%) developed hypothyroidism, six patients (40%) had CKD, and six patients (40%) had growth failure. Hypothyroidism and growth failure were less frequent in patients who received reduced doses of 131 I‐MIBG therapy. Tandem HDCT/ASCT combined with HD 131 I‐MIBG therapy could be feasible for patients with high‐risk neuroblastoma with acceptable toxicity profiles and favorable outcomes.
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131I-MIBG combined with chemotherapy and stem cell rescue improves event-free survival in children with high-risk neuroblastoma.
“When combined with chemotherapy and stem cell rescue, 131I-MIBG improved event-free survival and overall survival in children with high-risk neuroblastoma.”
From Radiopharmaceuticals: navigating the frontier of precision medicine and therapeutic innovation (2024) · cites this paper as [88]
In a phase II trial, 3-year overall survival for 131I-MIBG plus chemotherapy and stem cell rescue was 74% versus 59%.
“The 3-year event-free survival increased from 48 to 61%, and the 3-year overall survival improved from 59 to 74% [88].”
From Radiopharmaceuticals: navigating the frontier of precision medicine and therapeutic innovation (2024) · cites this paper as [88]
131I-MIBG combined with chemotherapy and stem cell rescue improves overall survival in children with high-risk neuroblastoma.
“When combined with chemotherapy and stem cell rescue, 131I-MIBG improved event-free survival and overall survival in children with high-risk neuroblastoma.”
From Radiopharmaceuticals: navigating the frontier of precision medicine and therapeutic innovation (2024) · cites this paper as [88]
In a phase II trial, 3-year event-free survival for 131I-MIBG plus chemotherapy and stem cell rescue was 61% versus 48%.
“The 3-year event-free survival increased from 48 to 61%, and the 3-year overall survival improved from 59 to 74% [88].”
From Radiopharmaceuticals: navigating the frontier of precision medicine and therapeutic innovation (2024) · cites this paper as [88]
Common adverse events of 131I-MIBG included hematologic issues, infections, and mucositis.
“Common adverse events included hematologic issues, infections, and mucositis.”
From Radiopharmaceuticals: navigating the frontier of precision medicine and therapeutic innovation (2024) · cites this paper as [88]
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Radiopharmaceuticals: navigating the frontier of precision medicine and therapeutic innovation
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- Authors
- Suh, J. K., Koh, K., Min, S. Y., Kim, Y. S., Kim, H., Im, H. J., Namgoong, J., Kim, D. Y., Ahn, S. D., Lee, J. J., & Seo, J. J.
- Affiliations
- Asan Medical Center (KR)
- Corresponding author
- Koh, Kyung‐Nam
- ORCID
- Venue
- Pediatric Transplantation
- Publisher
- Wiley
- Published
- 2020-01-20
- Volume
- 24
- Issue
- 2
- Pages
- e13658-e13658
- ISSN
- 1397-3142 (ISSN-L)
- 1399-3046
- OpenAlex
- W3001941751
- Cited by
- 22
- References
- 33
- Citations by year
- 2025: 1 · 2024: 2 · 2023: 6 · 2022: 2 · 2021: 4 · 2020: 7
- FWCI
- 2.06
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Pediatric Transplantation (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Asan Institute for Life Sciences, Asan Medical Center (2015‐640)
- Topics
- Health Sciences › Medicine › Neurology › Neuroblastoma Research and Treatments
- Health Sciences › Medicine › Neurology › Neurofibromatosis and Schwannoma Cases
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Sarcoma Diagnosis and Treatment
- Keywords
- high-risk neuroblastoma, tandem high-dose chemotherapy, autologous stem cell transplantation, 131I-MIBG therapy, targeted radiotherapy, thiotepa, hypothyroidism, growth failure, chronic kidney disease, treatment toxicity, overall survival, event-free survival
- MeSH terms
- Abdominal Neoplasms, Antineoplastic Agents, Antineoplastic Combined Chemotherapy Protocols, Child, Child, Preschool, Dose-Response Relationship, Drug, Feasibility Studies, Female, Follow-Up Studies, Humans, Infant, Iodine Radioisotopes, Male, Neuroblastoma, Retrospective Studies, Risk, Spinal Neoplasms, Transplantation, Autologous, Chemotherapy, Adjuvant, Hematopoietic Stem Cell Transplantation, Radiotherapy, Adjuvant, 3-Iodobenzylguanidine, Induction Chemotherapy
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