Chemotherapy May Allow Preservation of the Eye and Avoidance of Radiation Therapy for Children with Early-Stage Retinoblastoma

Chemotherapy May Allow Preservation of the Eye and Avoidance of Radiation Therapy for Children with Early-Stage Retinoblastoma

Treatment for retinoblastoma, a cancer of the eye that occurs in children, is often successful with surgical removal of the eye and followup radiation therapy. However, this treatment involves the loss of an eye as well as the potential long-term side effects of radiation therapy, such as interference with a child’s growth and development and the occurrence of a second cancer later in life. In a recent study, researchers found that children with early-stage retinoblastoma may be able to keep the affected eye(s) and avoid radiation therapy by receiving 6 cycles of a chemotherapy combination.

Retinoblastoma, a disease most often occurring in children 5 years or younger, is characterized by the presence of cancer cells in the retina, a nerve tissue of the eye. Although this type of cancer may affect 1 or both eyes, it usually does not spread to other parts of the body. However, this type of cancer is associated with a high risk of the development of a second cancer later in life. Treatment for retinoblastoma depends on the stage of disease (extent of disease at diagnosis) and whether 1 or 2 eyes are affected. Treatment may consist of the surgical removal of the affected eye (called enucleation), external-beam radiation therapy (high-energy rays, delivered by a machine outside the body, to kill cancer cells). Other options that may be part of treatment or are under study include cryotherapy (use of cold temperatures to kill cancer cells), photocoagulation (use of lasers to destroy blood vessels that go to the cancer cells), thermal therapy (use of heat to kill cancer cells), plaque radiation therapy (a method of applying radioactive material directly into or near the cancer cells), or chemotherapy (use of drugs to kill cancer cells).

Researchers in Philadelphia evaluated a new treatment for retinoblastoma in an attempt to save the affected eye(s) and avoid external-beam radiation therapy and its potential long-term side effects. Forty-seven patients with retinoblastoma of all stages, having a total of 75 affected eyes, received 6 cycles of intravenous chemotherapy drugs called vincristine, etoposide, and carboplatin. In addition, 83% of the patients received local treatment, meaning treatment to the eye only, with therapies such as cryotherapy, photocoagulation, thermal therapy, or plaque radiation therapy.

The results showed that the chemotherapy, combined with local treatment when needed, was successful in 100% of children with early-stage retinoblastoma, allowing preservation of the eye(s) and avoidance of external-beam radiation. Overall, 26% of patients did require either removal of an eye or the use of external-beam radiation. The side effects from the chemotherapy were mild. No child had progression of cancer or development of a second cancer.

These researchers concluded that, in children with early-stage retinoblastoma, the use of chemotherapy combined with local treatment to the eye may eliminate the need for surgical removal of the eye(s) and use of external-beam radiation therapy. They noted that further study is needed to develop more effective therapies for children with advanced retinoblastoma. (

Journal of Clinical Oncology, Vol 18, No 1, pp 12-17, 2000)

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