First-line chemotherapy with local treatment can prevent external-beam irradiation and enucleation in low-stage intraocular retinoblastoma

Purpose: To evaluate the efficacy of first-line chemotherapy (CT) in preven ting external-beam radiotherapy (EBR) and/or enucleation in patients with r etinoblastoma (Rbl). Patients and Methods: Twenty-four patients with newly diagnosed unilateral or bilateral Rbl received CT associated with local treatment (LT). Two to f ive courses of etoposide and carboplatin were administered at 3- to 4-week intervals, depending on tumor response, and were completed each time by LT, Results: Tumor response was observed in all eyes, Twenty-one of 24 patients showed ct complete response (CR) that persisted at ct median follow-up (FU ) of 31 months (range, 4 to 41 months). Among the three patients who relaps ed, two were lost to FU and one died of progressive disease. CR was achieve d by CT and LT alone in 15 (71.4%) of 21 patients with less advanced diseas e (groups I to III), Six other patients with advanced disease (groups IV an d V) experienced treatment failure and needed salvage treatment by EBR and/ or enucleation. The difference between the two patient groups with regard t o disease stage wets statistically significant (P < .0001), EBR could be av oided in 13 (68.4%) of 19 patients, who presented with groups I to III (15 eyes) and group V (one eye) disease, whereas enucleation could be avoided i n only two (40%) of five. Conclusion: CT combined with intensive LT is effective in patients with gro ups I to III Rbl, permitting the avoidance of EBR in the majority of these young children and, thus, reducing the risk of long-term sequelae. This is in contrast with the disappointing results for patients with groups IV and V Rbl, in whom EBR and/or enucleation was needed. J Clin Oncol 18:2881-2887 . (C) 2000 by American Society of Clinical Oncology.