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

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The delivery of chemotherapy via ophthalmic artery cannulation as initial treatment for advanced unilateral retinoblastoma appears to be more effective than does systemic chemotherapy for chemoreduction. In the setting of a multidisciplinary state-of-the-art center, intra-arterial chemotherapy may result in ocular salvage rates greater than 80% for patients with advanced intraocular unilateral retinoblastoma.[13]; [14,15][Level of evidence: 3iiiDii]; [16][Level of evidence: 3iiiDiv]

Because a proportion of children who present with unilateral retinoblastoma will eventually develop disease in the opposite eye, these children undergo genetic counseling and testing and periodic examinations of the unaffected eye, regardless of the treatment they received. Asynchronous bilateral disease occurs most frequently in patients with affected parents and in children diagnosed during the first months of life.

Standard Treatment Options for Bilateral Retinoblastoma

The management of bilateral disease depends on the extent of the disease in each eye. Systemic therapy is generally chosen based on the eye with more extensive disease. Treatment modality options described for unilateral disease may be applied to one or both affected eyes in patients with bilateral disease. Systemic or intra-arterial chemotherapy (chemoreduction) coupled with aggressive local treatments and very close monitoring is usually the treatment of choice; the goal is ocular and vision preservation and the delay or avoidance of EBRT and enucleation.

Standard treatment options for bilateral retinoblastoma include the following:

  1. Enucleation for large intraocular tumors, followed by risk-adapted chemotherapy when the eye and vision cannot be saved.
  2. Conservative ocular salvage approaches when the eye and vision can be saved.
    • Chemoreduction with either of the following:
      • Systemic chemotherapy with subtenon chemotherapy.
      • Ophthalmic artery infusion chemotherapy.
    • Local treatments including cryotherapy, thermotherapy, and plaque radiation therapy.
    • EBRT.

Intraocular tumor burden is usually asymmetric, and treatment is dictated by the most advanced eye. While up-front enucleation of an advanced eye and risk-adapted adjuvant chemotherapy may be required, a more conservative approach using primary chemoreduction with close follow-up for response and aggressive local treatment may be indicated. EBRT is now reserved for patients whose eyes do not respond adequately to primary systemic or intra-arterial chemotherapy and local consolidation.

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