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Pediatric Oncology

Retinoblastoma: Review of Current Management

^aTexas Children's Cancer Center, ^bDepartment of Ophthalmology, ^cDepartment of Pediatrics, ^dSection of Hematology/Oncology, ^eDepartment of Molecular and Human Genetics, ^fCenter for Cell & Gene Therapy, and ^gDepartment of Molecular & Cellular Biology, Baylor College of Medicine, Houston, Texas, USA; ^hDepartment of Pathology, Weill Medical College of Cornell University at The Methodist Hospital, Houston, Texas, USA

Key Words. Retinoblastoma • Chemotherapy • Genetics of retinoblastoma • Second malignancies • Animal models

Correspondence: Murali Chintagumpala, M.D., 6701 Fannin Street, Clinical Care Center, 14th Floor, MC CC1410, Houston, Texas 77030, USA. Telephone: 832-822-1482; Fax: 832-825-1503; e-mail: mxchinta{at}txccc.org

Disclosure: No potential conflicts of interest were reported by the authors, planners, reviewers, or staff managers of this article.

The most common ocular cancer in children is retinoblastoma. It affects approximately 300 children in the U.S. every year. It can affect one or both eyes and the disease can be inherited. Altered discoloration of the pupil and strabismus are the usual symptoms that lead to medical attention. Subsequent appropriate diagnostic studies and care provided by a multidisciplinary team, including an ophthalmologist, a pediatric oncologist, a radiation oncologist, and a geneticist, among others, often result in optimal short-term and long-term care. The best initial and subsequent treatments are based on whether the child has unilateral or bilateral disease, the stage of the disease, and the age of the child. Enucleation, chemotherapy, and various forms of radiation therapy along with local ophthalmic therapies can be used in the treatment of retinoblastoma. Cure rates are high in children when the tumor is confined to the eye and has not spread systemically or into the orbit or brain. Children with the heritable form of retinoblastoma are at high risk for developing subsequent malignancies, most commonly sarcomas. This risk is greater for those children with the heritable form of the disease who were exposed to ionizing radiation at age <1 year. Exciting discoveries using animal models are providing new insights into the development of this disease and opening new avenues for targeted therapies that may lead to high cure rates with minimal toxicities.