Multiagent chemotherapy as neoadjuvant treatment for multifocal intraocular retinoblastoma

Purpose. To evaluate the efficacy of multiagent chemotherapy in the neoadju vant treatment of retinoblastoma. Design: Noncomparative, prospective, case series. Participants. Twenty consecutive patients with multifocal intraocular retin oblastoma (4 unilateral, 16 bilateral [36 eyes]). Intervention: Eight cycles of chemotherapy with carboplatin and vincristine were administered at 3-week intervals over a 6-month period. Supplemental therapy was withheld until disease progression was documented. Main Outcome Measures:. Disease progression (defined as tumor growth, vitre ous or subretinal seed progression, and new tumor formation), delay of exte rnal beam radiotherapy, and ocular survival. Results: Thirty-six eyes were treated. Eighteen eyes had Reese-Ellsworth gr oup I-III tumors, and 16 eyes had Reese-Ellsworth group IV-V tumors, at dia gnosis. Two patients, who had unilateral disease at diagnosis, subsequently had tumors develop in the contralateral eye. Nineteen of 20 patients (95%) completed eight cycles of chemotherapy without disease progression. Three eyes of three different patients were successfully treated with chemotherap y alone. Thirty-three of 36 eyes (92%) progressed after completion of chemo therapy: 15 of the 18 eyes (83.3%) with Reese-Ellsworth group I-III and 16 of 16 eyes (100%) with Reese-Ellsworth group IV-V tumors. Seventeen eyes (5 2%) had growth of a tumor, whereas 14 eyes (42%) had progressive vitreous s eeding, and 2 eyes (6%) had new tumors develop. Fifteen eyes (42%) required external beam radiotherapy. Twenty-nine of 36 (80.5%) eyes were salvaged. The median follow-up after chemotherapy was 19 months (range, 3-42 months). Conclusions. Multiagent chemotherapy alone does not ensure a cure for multi focal intraocular retinoblastoma. Supplemental focal therapy is needed to c ontrol disease progression. Ophthalmology 2001;108: 2106-2115 (C) 2001 by t he American Academy of Ophthalmology.