Long-Term Results for Children With High-Risk Neuroblastoma Treated on a Randomized Trial of Myeloablative Therapy Followed by 13-cis-Retinoic Acid: A Children's Oncology Group Study
Open accessAbstract
Purpose: We assessed the long-term outcome of patients enrolled on CCG-3891, a high-risk neuroblastoma study in which patients were randomly assigned to undergo autologous purged bone marrow transplantation (ABMT) or to receive chemotherapy, and subsequent treatment with 13-cis-retinoic acid (cis-RA). Patients and Methods: Patients received the same induction chemotherapy, with random assignment (N = 379) to consolidation with myeloablative chemotherapy, total-body irradiation, and ABMT versus three cycles of intensive chemotherapy. Patients who completed consolidation without disease progression were randomly assigned to receive no further therapy or cis-RA for 6 months. Results: The event-free survival (EFS) for patients randomly assigned to ABMT was significantly higher than those randomly assigned to chemotherapy; the 5-year EFS (mean ± SE) was 30% ± 4% versus 19% ± 3%, respectively (P = .04). The 5-year EFS (42% ± 5% v 31% ± 5%) from the time of second random assignment was higher for cis-RA than for no further therapy, though it was not significant. The 5-year overall survival (OS) from the second random assignment of patients who underwent both random assignments and who were assigned to ABMT/cis-RA was 59% ± 8%; for ABMT/no cis-RA, it was 41% ± 7%; for continuing chemotherapy/cis-RA, it was 38% ± 7%; and for chemotherapy/no cis-RA, it was 36% ± 7%. Conclusion: Myeloablative therapy and autologous hematopoietic cell rescue result in significantly better 5-year EFS than nonmyeloablative chemotherapy; neither myeloablative therapy with autologous hematopoietic cell rescue nor cis-RA given after consolidation therapy significantly improved OS.
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Results of several clinical trials indicate that 13-cis RA, alone or in combination, seems to be effective at different levels against malignant gliomas, and cancer occurring in the central and peripheral nervous system.
“the results of several clinical trials indicate that 13-cis RA, alone or in combination, seems to be effective at different levels against malignant gliomas115, and cancer occurring in the central and peripheral nervous system116.”
From Advances and challenges in retinoid delivery systems in regenerative and therapeutic medicine (2020) · cites this paper as [116]
The ATRA derivative 13-cis-retinoic acid (13-cis-RA) is successfully used in the treatment of children with high-risk neuroblastoma, reducing the risk of recurrence after high-dose chemotherapy and stem cell transplantation and increasing long-term survival.
“For example, the ATRA derivative 13-cis-retinoic acid (13-cis-RA) is successfully used in the treatment of children with high-risk neuroblastoma, reducing the risk of recurrence after high-dose chemotherapy and stem cell transplantation and increasing long-term survival (Matthay et al, 2009).”
From Unlocking the potential of retinoic acid in anticancer therapy (2014) · cites this paper as Matthay et al, 2009
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- Authors
- Matthay, K. K., Reynolds, C. P., Seeger, R. C., Shimada, H., Adkins, E. S., Haas-Kogan, D., Gerbing, R. B., London, W. B., & Villablanca, J. G.
- Affiliations
- University of Southern California (US)
- Corresponding author
- Matthay, Katherine K.
- ORCID
- Venue
- Journal of Clinical Oncology
- Published
- 2009-03-01
- Volume
- 27
- Issue
- 7
- Pages
- 1007-1013
- ISSN
- 0732-183X (ISSN-L)
- 1527-7755
- PMC
- PMC2738615
- OpenAlex
- W2147800896
- Cited by
- 858
- References
- 26
- Citations by year
- 2026: 32 · 2025: 35 · 2024: 40 · 2023: 30 · 2022: 41 · 2021: 70 · 2020: 51 · 2019: 55 · 2018: 56 · 2017: 57 · 2016: 46 · 2015: 67 · 2014: 41 · 2013: 64 · 2012: 57
- FWCI
- 36.57
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Journal of Clinical Oncology (publishedVersion)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- U.S. Department of Health and Human Services
- Frank A. Campini Foundation
- Children’s Oncology Group
- National Institutes of Health
- National Cancer Institute (CA‐98413, P01CA081403, U10CA098413, CA-81403, R01CA082830, U10CA013539, CA82830, CA 13539)
- Topics
- Health Sciences › Medicine › Neurology › Neuroblastoma Research and Treatments
- Health Sciences › Medicine › Neurology › Neurofibromatosis and Schwannoma Cases
- Health Sciences › Medicine › Pediatrics, Perinatology and Child Health › Childhood Cancer Survivors' Quality of Life
- Keywords
- high-risk neuroblastoma, autologous bone marrow transplantation, myeloablative chemotherapy, total body irradiation, isotretinoin, event-free survival, overall survival, long-term outcomes, consolidation therapy, childhood cancer
- MeSH terms
- Adolescent, Antineoplastic Combined Chemotherapy Protocols, Child, Child, Preschool, Combined Modality Therapy, Humans, Infant, Neuroblastoma, Whole-Body Irradiation, Isotretinoin, Survival Analysis, Bone Marrow Transplantation
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