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Clinical Trial
. 2022 Nov 20;40(33):3839-3847.
doi: 10.1200/JCO.21.02337. Epub 2022 Jul 12.

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

Ira J Dunkel  1 , Jin Piao  2 , Guillermo L Chantada  3 , Anuradha Banerjee  4 , Sherif Abouelnaga  5 , Jeffrey C Buchsbaum  6 , Thomas E Merchant  7 , Meaghan M Granger  8 , Rima F Jubran  9 , Joanna L Weinstein  10 , Lauren Saguilig  11 , David H Abramson  1 , Mark D Krailo  2 , Carlos Rodriguez-Galindo  7 , Murali M Chintagumpala  12
Affiliations
Clinical Trial

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

Ira J Dunkel et al. J Clin Oncol. .

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).

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Conflict of interest statement

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

The following represents disclosure information provided by authors of this manuscript. All relationships are considered compensated unless otherwise noted. Relationships are self-held unless noted. I = Immediate Family Member, Inst = My Institution. Relationships may not relate to the subject matter of this manuscript. For more information about ASCO's conflict of interest policy, please refer to www.asco.org/rwc or ascopubs.org/jco/authors/author-center.

Open Payments is a public database containing information reported by companies about payments made to US-licensed physicians (Open Payments).

Figures

FIG 1.
FIG 1.
COG ARET0321 treatment schema. AUC, area under the curve; IV, intravenous; SC, subcutaneous.
FIG 2.
FIG 2.
CONSORT diagram.
FIG 3.
FIG 3.
Kaplan-Meier curves and at-risk table of EFS and OS for (A) stage II-III patients, (B) stage IVa patients, and (C) stage IVb patients. EFS, event-free survival; OS, overall survival.
See this image and copyright information in PMC

References

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