Chemoreduction and local ophthalmic therapy for intraocular retinoblastoma

Purpose: To study the effectiveness of combined systemic chemotherapy and l ocal ophthalmic therapy for retinoblastoma with the goal of avoiding enucle ation and external-beam radiation therapy (EBRT). Patients and Methods: This was a prospective, nonrandomized, single-arm cli nical trial. Seventy-five eyes were followed in 47 children. Patients were treated with a six-cycle protocol of vincristine, etoposide, and carboplati n. Most (83%) also received ophthalmic treatment (cryotherapy, laser photoc oagulation, thermotherapy, or plaque radiation therapy) during and/or after the chemotherapy, Results: With a median follow-vp of 13 months, event-free survival was 74%, with an event defined as enucleation and/or EBRT. Six children required EB RT in seven eyes (9%); five required enucleation of one eye (7%); five requ ired a combination of EBRT and enucleation in six eyes (8%). Reese-Ellswort h groups 1,2, and 3 eyes held excellent results, with avoidance of EBRT or enucleation in all 39, Treatment of groups 4 and 5 was less successful, wit h 33% of six eyes and 53% of 30 eyes, respectively, requiring EBRT and/or e nucleation. Toxicities from chemotherapy were mild and included cytopenias (89%), fever and neutropenia (28%), infection (9%), and gastrointestinal sy mptoms, dehydration, and vincristine neurotoxicity (40%). No patients devel oped a second malignancy, metastatic disease, renal disease, or ototoxicity . Conclusion: In retinoblastoma patients with Reese-Ellsworth eye groups 1,2, or 3, systemic chemotherapy used with local ophthalmic therapies can elimi nate the need for enucleation or EBRT without significant systemic toxicity . More effective therapy is required for Reese-Ellsworth eye groups 4 and 5 . (C) 2000 by American Society of Clinical Oncology.