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Enucleation with or without adjuvant chemotherapy
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 Intraocular Retinoblastoma / Treatment of Unilateral Intraocular Retinoblastoma
Because unilateral disease is usually massive and there is often no expectation that useful vision can be preserved, up-front surgery (enucleation) is commonly performed. Careful examination of the enucleated specimen by an experienced pathologist is necessary to determine whether high-risk features for metastatic disease are present. These high-risk features include the following:[1-5]; [6][Level of evidence C1 and C2]
Anterior chamber seeding.
Massive choroidal involvement.
Tumor beyond the lamina cribrosa.
Scleral and extrascleral extension.
Pre-enucleation magnetic resonance imaging has low sensitivity and specificity for the detection of high-risk pathology.[7]
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High-risk pathology has been associated with the presence of minimal dissemination in bone marrow and cerebrospinal fluid using quantitative polymerase chain reaction for detection of CRX or GD2 synthase. In a group of 96 children with nonmetastatic retinoblastoma and high-risk pathology, the 3-year disease-free survival rate was 78% for patients with detectable minimal dissemination, compared with 98% for those without detectable disease (P = .004).[8]
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Systemic adjuvant therapy with vincristine, doxorubicin, and cyclophosphamide or with vincristine, carboplatin, and etoposide has been used to prevent the development of metastatic disease in patients with certain high-risk features assessed by pathological review after enucleation.[3]
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The Children’s Oncology Group ARET0332 (NCT00335738) trial prospectively studied the role of adjuvant chemotherapy in 321 eligible children with newly diagnosed enucleated unilateral retinoblastoma. Central histopathological review was performed for all patients' pathology slides. Defined indications for adjuvant chemotherapy included massive choroid replacement defined as posterior uveal invasion grades IIC and IID, any posterior uveal involvement less than 3 mm with concomitant optic nerve involvement, and optic nerve involvement posterior to the lamina cribrosa. Treatment consisted of six cycles of carboplatin, etoposide, and vincristine administered every 4 weeks.[6][Level of evidence C1 and C2]
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Central review of enucleated eyes by ophthalmic pathologists identified a group of children (19% of all eligible patients) who were misclassified by the institutional pathology report.
The importance of universal guidelines for handling and interpreting retinoblastoma specimens is instrumental in the recognition and categorization of high-risk features.
For all patients on the study, the overall 2-year event-free survival (EFS) rate was 98%, and the overall survival (OS) rate was 99%.
For patients with high-risk features requiring adjuvant chemotherapy, the 2-year EFS rate was 96%.
For patients with low-risk features for whom observation was indicated, the 2-year EFS rate was 99%.
The combination of extensive involvement of both the choroid and the optic nerve posterior to the lamina cribrosa represents the highest risk of recurrence and supports the need for more intensive chemotherapy for this group.
The average time to first recurrence was less than 12 months, and the time to death after recurrence was approximately 8 months.
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Preserved source evidence · Not medical advice