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Treatment of Orbital and Locoregional Retinoblastoma
Source: Retinoblastoma Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 3, 2025 · Captured 2026-09-09.
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Context: Treatment of Extraocular Retinoblastoma
Orbital retinoblastoma occurs as a result of tumor progression through the emissary vessels and sclera. For this reason, transscleral disease is considered to be extraocular and should be treated as such. Orbital retinoblastoma is isolated in 60% to 70% of cases.
Treatment options for extraocular retinoblastoma (orbital and locoregional) include the following:
Chemotherapy.
Radiation therapy.
Enucleation (for extraocular extension).
Treatment includes systemic chemotherapy and radiation therapy. With this treatment approach, 60% to 85% of patients can be cured. Because most recurrences occur in the central nervous system (CNS), regimens that include drugs with well-documented CNS penetration are used.
The Children’s Oncology Group (COG) performed a prospective international trial (ARET0321 [NCT00554788]) that included patients with extraocular retinoblastoma. The study showed that intensified therapy improved the outcomes of patients with stage II, III or IVa disease, compared with historical controls. However, stage IVb patients need more effective therapy.[1][Level of evidence B4]
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Patients with stage II or III retinoblastoma (n = 19) received four cycles of chemotherapy (vincristine, cisplatin, cyclophosphamide, etoposide) followed by involved-field radiation therapy (45 Gy).
Patients with stage II or III retinoblastoma (n = 19) received four cycles of chemotherapy (vincristine, cisplatin, cyclophosphamide, etoposide) followed by involved-field radiation therapy (45 Gy).
The median follow-up was 7.3 years.
Patients with stage II or III retinoblastoma (n = 19) received four cycles of chemotherapy (vincristine, cisplatin, cyclophosphamide, etoposide) followed by involved-field radiation therapy (45 Gy).
The 3-year event-free survival (EFS) and overall survival (OS) rates were 88.1%.
Patients with stage IVa retinoblastoma (n = 18) received four cycles of induction therapy (noted above). Patients with more than a partial response underwent autologous hematopoietic stem cell support. Patients with residual tumor after chemotherapy received radiation therapy.
Patients with stage IVa retinoblastoma (n = 18) received four cycles of induction therapy (noted above). Patients with more than a partial response underwent autologous hematopoietic stem cell support. Patients with residual tumor after chemotherapy received radiation therapy.
The 3-year EFS and OS rates were 76.7%.
Patients with stage IVb retinoblastoma (n = 20) received the same treatment as patients with stage IVa disease.
Patients with stage IVb retinoblastoma (n = 20) received the same treatment as patients with stage IVa disease.
The 1-year EFS rate was 28.3%, and the OS rate was 42.1%.
Patients with stage IVb retinoblastoma (n = 20) received the same treatment as patients with stage IVa disease.
The 3-year EFS rate was 14.2%, and the OS rate was 12.3%.
Six patients developed secondary malignancies. There was one case each of osteosarcoma (8 years after treatment), thyroid papillary carcinoma (9 years after treatment), glioblastoma multiforme (8 years after treatment), large B-cell Epstein-Barr virus–associated lymphoproliferative disorder (8 months after treatment), acute myeloid leukemia (2 years after treatment), and alveolar soft part sarcoma of the bladder (5 years after treatment). Five patients had unilateral retinoblastoma, two of whom received radiation therapy (one each with unilateral and bilateral).
For patients with macroscopic orbital disease, delay of surgery until response to chemotherapy is achieved (usually after receiving two or three courses of treatment) has been effective. Patients then undergo enucleation and receive an additional four to six courses of chemotherapy. During consolidation, the patient receives local control therapies with orbital irradiation (40–45 Gy). Using this approach, orbital exenteration is not indicated.[2]
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Patients with isolated involvement of the optic nerve at the transsection level are considered to have extraocular disease and are treated using systemic therapy, similar to that used for macroscopic orbital disease, and irradiation of the entire orbit (36 Gy) with a 10 Gy boost to the chiasm (total of 46 Gy).[3]
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