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Journal of Clinical Oncology, Vol 18, Issue 1 (January), 2000: 12
© 2000 American Society for Clinical Oncology

Chemoreduction and Local Ophthalmic Therapy for Intraocular Retinoblastoma

By Debra L. Friedman, Bruce Himelstein, Carol L. Shields, Jerry A. Shields, Michael Needle, David Miller, Greta R. Bunin, Anna T. Meadows

From the Department of Pediatrics, Division of Oncology, The Children’s Hospital of Philadelphia; University of Pennsylvania, Philadelphia PA; and Ocular Oncology Service, Wills Eye Hospital, Thomas Jefferson University, Philadelphia PA.

Address reprint requests to Debra L. Friedman, Division of Hematology/Oncology, CH-29, Children’s Hospital and Regional Medical Center, 4800 Sand Point Way NE, Seattle, WA 98105; email dfried{at}chmc.org

PURPOSE: To study the effectiveness of combined systemic chemotherapy and local ophthalmic therapy for retinoblastoma with the goal of avoiding enucleation and external-beam radiation therapy (EBRT).

PATIENTS AND METHODS: This was a prospective, nonrandomized, single-arm clinical trial. Seventy-five eyes were followed in 47 children. Patients were treated with a six-cycle protocol of vincristine, etoposide, and carboplatin. Most (83%) also received ophthalmic treatment (cryotherapy, laser photocoagulation, thermotherapy, or plaque radiation therapy) during and/or after the chemotherapy.

RESULTS: With a median follow-up of 13 months, event-free survival was 74%, with an event defined as enucleation and/or EBRT. Six children required EBRT in seven eyes (9%); five required enucleation of one eye (7%); five required a combination of EBRT and enucleation in six eyes (8%). Reese-Ellsworth groups 1, 2, and 3 eyes had excellent results, with avoidance of EBRT or enucleation in all 39. Treatment of groups 4 and 5 was less successful, with 33% of six eyes and 53% of 30 eyes, respectively, requiring EBRT and/or enucleation. Toxicities from chemotherapy were mild and included cytopenias (89%), fever and neutropenia (28%), infection (9%), and gastrointestinal symptoms, dehydration, and vincristine neurotoxicity (40%). No patients developed 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 eliminate the need for enucleation or EBRT without significant systemic toxicity. More effective therapy is required for Reese-Ellsworth eye groups 4 and 5.

Presented in part at the Thirty-Fifth Annual Meeting of the American Society of Clinical Oncology, Atlanta, GA, and at the 1999 Joint Annual Meeting of the International Society of Paediatric Oncology and the American Society of Paediatric Hematology/Oncology, Montreal, Canada.


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