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


Cavitary Retinoblastoma

In patients with cavitary retinoblastoma, minimal visual response is seen after intravenous chemotherapy and/or intra-arterial chemotherapy. Despite the blunted clinical response, cavitary retinoblastoma has a favorable long-term outcome with stable tumor regression and globe salvage. Aggressive or prolonged chemotherapy or adjunctive therapies are generally not necessary. In a retrospective series of 26 cavitary retinoblastomas that were treated with intravenous chemoreduction and/or intra-arterial chemotherapy, the mean reduction in tumor base was 22%, and the mean reduction in tumor thickness was 29%. Despite minimal reduction, tumor recurrence was noted in only one eye, globe salvage was achieved in 22 eyes, and there were no cases of metastasis or death during 49 months (range, 6-189 months) of follow-up.[35]

Treatment Options Under Clinical Evaluation for Intraocular Retinoblastoma

Studies are planned for a variety of patient groups. The International Classification of Retinoblastoma is being utilized for these trials.

The following is an example of a national and/or institutional clinical trial that is currently being conducted. Information about ongoing clinical trials is available from the NCI Web site.

  • ARET12P1 (NCT02097134) (Intra-arterial Melphalan in Treating Younger Patients With Unilateral Retinoblastoma): This pilot clinical trial is studying whether unilateral Group D retinoblastoma, or retinoblastoma affecting one eye that has spread to the vitreous fluid, can be treated with intra-arterial injection. This may provide children with unilateral retinoblastoma a lower chance of needing surgery to remove the eye and reduce the severity of treatment side effects.

Current Clinical Trials

Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with intraocular retinoblastoma. The list of clinical trials can be further narrowed by location, drug, intervention, and other criteria.

General information about clinical trials is also available from the NCI Web site.


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  34. Francis JH, Gobin YP, Brodie SE, et al.: Experience of intra-arterial chemosurgery with single agent carboplatin for retinoblastoma. Br J Ophthalmol 96 (9): 1270-1, 2012.
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This information is produced and provided by the National Cancer Institute (NCI). The information in this topic may have changed since it was written. For the most current information, contact the National Cancer Institute via the Internet web site at http:// cancer .gov or call 1-800-4-CANCER.

WebMD Public Information from the National Cancer Institute

Last Updated: May 28, 2015
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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