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Retinoblastoma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment Options for Unilateral and Bilateral Retinoblastoma


A number of large centers have published trial results that used systemic chemotherapy in conjunction with aggressive local consolidation for patients with bilateral disease.[10,17,18,19,20,21,22,23,24,25] The backbone of the chemoreduction has generally been carboplatin, etoposide, and vincristine. Chemotherapy shrinks the tumors (chemoreduction), allowing greater efficacy of subsequent local therapy.[10] Treatment strategies often differ in terms of chemotherapy regimens and local control measures. Using this approach, the International Classification of Retinoblastoma grouping system has been proven to predict ocular salvage.[26,27]; [28][Level of evidence: 3iiDiv]

  • Groups A and B: Systemic chemotherapy in conjunction with local control have resulted in ocular salvage rates greater than 90% for early intraocular disease.[27,28,29]
  • Groups C and D: Ocular salvage rates using this approach are 70% to 90% for Group C eyes and 40% to 50% for Group D eyes.[27,28,29] However, for patients with more advanced intraocular disease (typically Group D eyes), EBRT is frequently required for ocular salvage.[28]; [29][Level of evidence: 3iiDiii]

    For patients with large intraocular tumor burden with subretinal or vitreous seeds (Groups D eyes), the administration of higher doses of carboplatin, coupled with subtenon carboplatin, and the addition of lower doses of EBRT (36 Gy) for patients with persistent disease has been explored. Using this intensive approach, eye survival may approach a rate of 70% at 60 months.[30][Level of evidence: 2Div]

  • Group E: The treatment for Group E eyes is up-front enucleation. The use of prolonged systemic chemotherapy for Group E eyes to avoid or delay enucleation has been associated with lower disease-specific survival.[12][Level of evidence: 3iiiB]

Delivery of chemotherapy via ophthalmic artery cannulation has also been shown to be feasible and effective in patients with newly diagnosed bilateral disease as tandem administration and in the salvage setting.[14,15,31,32][Level of evidence: 3iiDii] Bilateral administrations increase the risk of systemic toxicity caused by melphalan exposure.[33] In these circumstances, intra-arterial chemotherapy with single-agent carboplatin may be used to treat the less-advanced eye during the tandem procedure.[34] These treatments should only be performed in an experienced center with a state-of-the-art treatment infrastructure and a dedicated multidisciplinary team.

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