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Treatment of CNS Disease
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
Intracranial dissemination occurs by direct extension through the optic nerve. The prognosis for these patients is dismal. Treatment includes platinum-based, intensive systemic chemotherapy and CNS-directed therapy. Although intrathecal chemotherapy has been used traditionally, there is no preclinical or clinical evidence to support its use.
Treatment options for extraocular retinoblastoma (CNS disease) include the following:
Systemic chemotherapy and CNS-directed therapy with radiation therapy.
Systemic chemotherapy followed by myeloablative chemotherapy and stem cell rescue with or without radiation therapy.
The administration of radiation therapy to these patients is controversial. Responses have been observed with craniospinal radiation using 25 Gy to 35 Gy to the entire craniospinal axis and a boost (10 Gy) to sites of measurable disease.[1]
Source links and citations
The COG conducted a prospective study (ARET0321 [NCT00554788]) of patients with extraocular retinoblastoma, which included patients with stage IVb disease who were treated with four cycles of induction therapy (vincristine, cisplatin, cyclophosphamide, and etoposide).[1][Level of evidence B4]
Source links and citations
Patients who had more than a partial response were then treated with high-dose carboplatin, thiotepa, and etoposide with autologous hematopoietic stem cell support.
Patients with residual disease after chemotherapy received radiation therapy.
In the 20 patients with stage IVb disease, the 1-year EFS rate was 28.3%, and the OS rate was 42.1%. The 3-year EFS rate was 14.2%, and the OS rate was 12.3%.
Studies cited in this source section
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