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Retinoblastoma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment Option Overview

Treatment planning by a multidisciplinary team of cancer specialists, including a pediatric oncologist, ophthalmologist, and radiation oncologist, who have experience treating ocular tumors of childhood is required to optimize treatment planning.[1]

The goals of therapy are threefold:

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  1. Eradicate the disease to save the patient's life.
  2. Preserve as much vision as possible.
  3. Decrease risk of late sequelae from treatment, particularly subsequent neoplasms.

The type of treatment required depends on both the extent of the disease within the eye and whether the disease has spread beyond the eye, either to the brain or to the rest of the body.[2] Eyes with glaucoma and those in which glaucoma resulted in buphthalmia are significantly associated with high-risk pathology risk factors and the occurrence of microscopically residual tumor.[3] Enucleation is reserved for patients with advanced unilateral intraocular disease with no hope for useful vision in the affected eye. Subsequent risk of extraocular recurrence may be increased in the presence of high-risk histopathologic features such as massive choroid invasion, scleral invasion, and optic nerve invasion.[4,5]; [6][Level of evidence: 3iiDi] Clinical features predictive of these histological findings include eyes with glaucoma, especially those that have become buphthalmic. Routine bone marrow biopsy and lumbar puncture are not indicated, except when there is a high level of suspicion that the tumor has spread beyond the globe.[7,8] Examples include patients with an abnormal complete blood count or those whose tumors show massive choroidal involvement and which extend beyond the lamina cribrosa on pathologic examination of the enucleated specimen.

It is not uncommon for patients with retinoblastoma to have extensive disease within one eye at diagnosis, with either massive tumors involving more than one-half of the retina, multiple tumors diffusely involving the retina, or obvious seeding of the vitreous. For those with bilateral disease, systemic therapy may be used to treat the more severe eye.[9,10] There are data suggesting that the use of systemic chemotherapy may decrease the risk of development of trilateral retinoblastoma.[11]


  1. Chintagumpala M, Chevez-Barrios P, Paysse EA, et al.: Retinoblastoma: review of current management. Oncologist 12 (10): 1237-46, 2007.
  2. Kopelman JE, McLean IW, Rosenberg SH: Multivariate analysis of risk factors for metastasis in retinoblastoma treated by enucleation. Ophthalmology 94 (4): 371-7, 1987.
  3. Chantada GL, Gonzalez A, Fandino A, et al.: Some clinical findings at presentation can predict high-risk pathology features in unilateral retinoblastoma. J Pediatr Hematol Oncol 31 (5): 325-9, 2009.
  4. Cuenca A, Giron F, Castro D, et al.: Microscopic scleral invasion in retinoblastoma: clinicopathological features and outcome. Arch Ophthalmol 127 (8): 1006-10, 2009.
  5. Gupta R, Vemuganti GK, Reddy VA, et al.: Histopathologic risk factors in retinoblastoma in India. Arch Pathol Lab Med 133 (8): 1210-4, 2009.
  6. Chantada GL, Dunkel IJ, Antoneli CB, et al.: Risk factors for extraocular relapse following enucleation after failure of chemoreduction in retinoblastoma. Pediatr Blood Cancer 49 (3): 256-60, 2007.
  7. Moscinski LC, Pendergrass TW, Weiss A, et al.: Recommendations for the use of routine bone marrow aspiration and lumbar punctures in the follow-up of patients with retinoblastoma. J Pediatr Hematol Oncol 18 (2): 130-4, 1996.
  8. Pratt CB, Meyer D, Chenaille P, et al.: The use of bone marrow aspirations and lumbar punctures at the time of diagnosis of retinoblastoma. J Clin Oncol 7 (1): 140-3, 1989.
  9. Abramson DH, Beaverson K, Sangani P, et al.: Screening for retinoblastoma: presenting signs as prognosticators of patient and ocular survival. Pediatrics 112 (6 Pt 1): 1248-55, 2003.
  10. Shields CL, Mashayekhi A, Demirci H, et al.: Practical approach to management of retinoblastoma. Arch Ophthalmol 122 (5): 729-35, 2004.
  11. Shields CL, Meadows AT, Shields JA, et al.: Chemoreduction for retinoblastoma may prevent intracranial neuroblastic malignancy (trilateral retinoblastoma). Arch Ophthalmol 119 (9): 1269-72, 2001.

WebMD Public Information from the National Cancer Institute

Last Updated: February 25, 2014
This information is not intended to replace the advice of a doctor. Healthwise disclaims any liability for the decisions you make based on this information.
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