Intensive Multimodality Therapy for Extraocular Retinoblastoma: A Children's Oncology Group Trial (ARET0321)

Ira J. Dunkel*, Jin Piao, Guillermo L. Chantada, Anuradha Banerjee, Sherif Abouelnaga, Jeffrey C. Buchsbaum, Thomas E. Merchant, Meaghan M. Granger, Rima F. Jubran, Joanna L. Weinstein, Lauren Saguilig, David H. Abramson, Mark D. Krailo, Carlos Rodriguez-Galindo, Murali M. Chintagumpala

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

14 Scopus citations

Abstract

PURPOSEMetastatic retinoblastoma has a poor prognosis when treated with conventional chemotherapy and radiation therapy (RT). Intensified therapy may improve the outcome.METHODSA 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.RESULTSFifty-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.CONCLUSIONIntensive 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).

Original languageEnglish (US)
Pages (from-to)3839-3847
Number of pages9
JournalJournal of Clinical Oncology
Volume40
Issue number33
DOIs
StatePublished - Nov 20 2022

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

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