Chemotherapy in the Management of Retinoblastoma

Abstract

Treatment of retinoblastoma aims to save life and preserve useful vision, and thus needs to be individualized. Factors that need to be considered include unilaterality or bilaterality of the disease, potential for vision, and intraocular and extraocular staging. A multidisciplinary approach is pivotal, and it is in this context that the role of chemotherapy must be discussed. The first clinical experience with chemotherapy in the treatment of retinoblastoma using nitrogen mustard was reported in 1953 (Kupfer 1953). Institutional experiences in the management of extraocular retinoblastoma were reported subsequently, usually applying regimens modeled after the treatment regimens for metastatic neuroblastoma (Lonsdale et al. 1968; Pratt et al. 1985). Since then, the role of chemotherapy has been expanding, and now it is a main component in the management of intraocular disease. Retinoblastoma is a very chemosensitive disease; in general, chemotherapy is indicated in patients with extraocular disease, in the subgroup of patients with intraocular disease with high-risk histologic features, and in patients with bilateral disease in conjunction with aggressive focal therapies. Agents with documented efficacy include microtubule inhibitors (vincristine and paclitaxel), platinum compounds (cisplatin and carboplatin), topoisomerase II inhibitors (teniposide and etoposide), alkylating agents (cyclophosphamide and ifosfamide), anthracyclines (doxorubicin and idarubicin), and topoisomerase I inhibitors (topotecan) (Schouten-van Meeteren et al. 2002; Rodriguez-Galindo et al. 2007).