Intensive multimodality therapy for extraocular retinoblastoma: A Children's Oncology Group trial (ARET0321) Journal Article


Authors: Dunkel, I. J.; Piao, J.; Chantada, G. L.; Banerjee, A.; Abouelnaga, S.; Buchsbaum, J. C.; Merchant, T. E.; Granger, M. M.; Jubran, R. F.; Weinstein, J. L.; Saguilig, L.; Abramson, D. H.; Krailo, M. D.; Rodriguez-Galindo, C.; Chintagumpala, M. M.
Article Title: Intensive multimodality therapy for extraocular retinoblastoma: A Children's Oncology Group trial (ARET0321)
Abstract: PURPOSE: Metastatic retinoblastoma has a poor prognosis when treated with conventional chemotherapy and radiation therapy (RT). Intensified therapy may improve the outcome. METHODS: A prospective, international trial enrolled patients with extraocular retinoblastoma. Patients with stage II or III (locoregional) retinoblastoma received four cycles of chemotherapy, followed by involved field RT (45 Gy). Patients with stage IVa or IVb (metastatic or trilateral) retinoblastoma also received four cycles of chemotherapy and those with ≥ partial response then received one cycle of high-dose carboplatin, thiotepa, and etoposide with autologous hematopoietic stem-cell support. Patients with stage IVa or IVb with residual tumor postchemotherapy received RT. The proportion of patients who achieved event-free survival would be reported and compared with historical controls separately for each of the three groups of patients. RESULTS: Fifty-seven eligible patients were included in the analyses. Event-free survival at 1 year was 88.1% (90% CI, 66.6 to 96.2) for stage II-III, 82.6% (90% CI, 61.0 to 92.9) for stage IVa, and 28.3% (90% CI, 12.7 to 46.2) for stage IVb/trilateral. Toxicity was significant as expected and included two therapy-related deaths. CONCLUSION: Intensive multimodality therapy is highly effective for patients with regional extraocular retinoblastoma and stage IVa metastatic retinoblastoma. Although the study met its aim for stage IVb, more effective therapy is still required for patients with CNS involvement (ClinicalTrials.gov identifier: NCT00554788).
Keywords: child; multimodality cancer therapy; combined modality therapy; antineoplastic agent; prospective study; prospective studies; antineoplastic combined chemotherapy protocols; retinoblastoma; pathology; retina tumor; retinal neoplasms; humans; human
Journal Title: Journal of Clinical Oncology
Volume: 40
Issue: 33
ISSN: 0732-183X
Publisher: American Society of Clinical Oncology  
Date Published: 2022-11-20
Start Page: 3839
End Page: 3847
Language: English
DOI: 10.1200/jco.21.02337
PUBMED: 35820112
PROVIDER: scopus
PMCID: PMC9671757
DOI/URL:
Notes: Article -- Export Date: 1 December 2022 -- Source: Scopus
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  1. Ira J Dunkel
    373 Dunkel
  2. David H Abramson
    394 Abramson