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Cancer Health Center

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Retinoblastoma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment of Progressive or Recurrent Retinoblastoma

The prognosis for a patient with progressive or recurrent retinoblastoma depends on the site and extent of the progression or recurrence and previous treatment received. Intraocular and extraocular recurrence have very different prognoses and are treated in distinctly different ways.

Treatment options for progressive or recurrent intraocular retinoblastoma include the following:

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  1. Enucleation.
  2. Radiation therapy (external-beam or plaque radiation therapy).
  3. Local treatments (cryotherapy or thermotherapy).
  4. Salvage chemotherapy (systemic or intra-arterial).

Treatment options for progressive or recurrent extraocular retinoblastoma include the following:

  1. Systemic chemotherapy and radiation therapy for orbital disease.
  2. Systemic chemotherapy followed by myeloablative chemotherapy with stem cell rescue and radiation therapy for extraorbital disease.

New intraocular tumors can arise in patients with the heritable form of disease, whose eyes have been treated with local control measures only, because every cell in the retina carries the RB1 mutation; this should not be considered a recurrence. Even with previous treatment consisting of chemoreduction and local control measures in very young patients with heritable retinoblastoma, surveillance may detect new tumors at an early stage and additional local control therapy, including plaque brachytherapy, can be successful in eradicating tumor.[1,2,3,4,5]

When the recurrence or progression of retinoblastoma is confined to the eye and is small, the prognosis for sight and survival may be excellent with local therapy only.[6][Level of evidence: 3iiDiv] If the recurrence or progression is confined to the eye but is extensive, the prognosis for sight is poor; however, survival remains excellent. Intra-arterial chemotherapy into the ophthalmic artery has been effective in patients who relapse after systemic chemotherapy and radiation therapy.[7] Radiation therapy should be considered for patients that have not been previously irradiated. Finally, enucleation may be required in cases of progressive disease after all eye-salvaging treatments have failed.

Recurrence in the orbit after enucleation is treated with aggressive chemotherapy in addition to local radiation therapy because of the high risk of metastatic disease.[8][Level of evidence: 3iiA]

If the recurrence or progression is extraocular, the chance of survival is poor.[9] However, the use of intensive systemic chemotherapy and consolidation with high-dose chemotherapy and autologous hematopoietic stem cell rescue may improve the chance of cure, particularly for patients with extracranial recurrence (refer to the Treatment Options for Extraocular Retinoblastoma section of this summary for more information). For patients recurring after those intensive approaches, clinical trials may be considered.

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