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Treatment Option Overview for Retinoblastoma
Source: Retinoblastoma Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 3, 2025 · Captured 2026-09-09.
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Treatment planning by a multidisciplinary team of cancer specialists—including a pediatric oncologist, ophthalmologist, and radiation oncologist—with experience treating ocular tumors of childhood is required to optimize treatment outcomes.[1] Evaluation at specialized centers is highly recommended before the initiation of treatment to improve the likelihood of ocular salvage and vision preservation.
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The goals of therapy include the following:
Eradicate the disease to save the patient's life.
Preserve as much vision as possible.
Decrease risk of late sequelae from treatment, particularly subsequent neoplasms (SNs).
Many treatments considered to be standard of care have not been studied in a randomized fashion.
Treatment of retinoblastoma depends on the intraocular and extraocular disease burden, disease laterality, germline RB1 gene status, and the potential for preserving vision. For patients presenting with intraocular disease, particularly those with bilateral eye involvement, a conservative approach consisting of tumor reduction with intravenous or intra-arterial chemotherapy (ophthalmic artery chemotherapy), coupled with aggressive local therapy, may result in high ocular salvage rates.[2] Radiation therapy, one of the most effective treatments in retinoblastoma, is usually reserved for cases of intraocular or extraocular disease progression.
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A risk-adapted, judicious combination of the following therapeutic options should be considered:
Enucleation.
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Local treatment (cryotherapy, laser therapy, and brachytherapy).
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Systemic chemotherapy.
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Intra-arterial chemotherapy (ophthalmic artery infusion of chemotherapy).
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Intravitreal chemotherapy.
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Intracameral chemotherapy.
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Radiation therapy (external-beam radiation therapy [EBRT], brachytherapy).
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The treatment options for intraocular, extraocular, and recurrent retinoblastoma are described in Table 13.
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| Treatment Group | Treatment Options | |
|---|---|---|
| CNS = central nervous system; EBRT = external-beam radiation therapy. | ||
| Intraocular retinoblastoma: | ||
| Unilateral retinoblastoma | Enucleation for large intraocular tumors, with or without adjuvant chemotherapy | |
| Conservative ocular salvage approaches when the eye and vision can be saved: | ||
| —Chemoreduction with either systemic or intra-arterial chemotherapy with or without intravitreal chemotherapy | ||
| —Local treatments (cryotherapy, thermotherapy, and plaque radiation therapy) | ||
| Bilateral retinoblastoma | Enucleation for large intraocular tumors, followed by pathology-based, risk-adapted chemotherapy when the eye and vision cannot be saved | |
| Conservative ocular salvage approaches when the eye and vision can be saved: | ||
| —Chemoreduction with either systemic or intra-arterial chemotherapy with or without intravitreal chemotherapy | ||
| —Local treatments (cryotherapy, thermotherapy, and plaque radiation therapy) | ||
| —EBRT | ||
| Extraocular retinoblastoma: | ||
| Orbital and locoregional retinoblastoma | Chemotherapy | |
| Radiation therapy | ||
| Enucleation (for extraocular extension) | ||
| CNS disease | Systemic chemotherapy and CNS-directed therapy with radiation therapy | |
| Systemic chemotherapy followed by myeloablative chemotherapy and stem cell rescue with or without radiation therapy | ||
| Synchronous trilateral retinoblastoma | Systemic chemotherapy followed by surgery and myeloablative chemotherapy with stem cell rescue | |
| Systemic chemotherapy followed by surgery and radiation therapy | ||
| Extracranial metastatic retinoblastoma | Systemic chemotherapy followed by myeloablative chemotherapy with stem cell rescue and radiation therapy | |
| Progressive or recurrent intraocular retinoblastoma | Enucleation | |
| Radiation therapy (EBRT or plaque radiation therapy) | ||
| Local treatments (cryotherapy or thermotherapy) | ||
| Salvage chemotherapy (systemic or intra-arterial) | ||
| Intravitreal chemotherapy, especially for refractory or recurrent vitreous seeding | ||
| Progressive or recurrent extraocular retinoblastoma | Systemic chemotherapy and radiation therapy for orbital disease | |
| Systemic chemotherapy followed by myeloablative chemotherapy with stem cell rescue, and radiation therapy for extraorbital disease | ||
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- Enucleation for large intraocular tumors, with or without adjuvant chemotherapy
- Conservative ocular salvage approaches when the eye and vision can be saved:
- Enucleation for large intraocular tumors, followed by pathology-based, risk-adapted chemotherapy when the eye and vision cannot be saved
- Conservative ocular salvage approaches when the eye and vision can be saved:
- Chemotherapy
- Radiation therapy
- Enucleation
- Systemic chemotherapy and CNS-directed therapy with radiation therapy
- Systemic chemotherapy followed by myeloablative chemotherapy and stem cell rescue with or without radiation therapy
- Systemic chemotherapy followed by surgery and myeloablative chemotherapy with stem cell rescue
- Systemic chemotherapy followed by surgery and radiation therapy
- Systemic chemotherapy followed by myeloablative chemotherapy with stem cell rescue and radiation therapy
- Enucleation
- Radiation therapy (EBRT or plaque radiation therapy)
- Local treatments (cryotherapy or thermotherapy)
- Salvage chemotherapy (systemic or intra-arterial)
- Intravitreal chemotherapy, especially for refractory or recurrent vitreous seeding
- Systemic chemotherapy and radiation therapy for orbital disease
- Systemic chemotherapy followed by myeloablative chemotherapy with stem cell rescue, and radiation therapy for extraorbital disease
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