Purpose: Management of intraocular retinoblastoma was initiated with 2-drug chemotherapy in an effort to improve the rate of vision preservation and ocular salvage and to avoid or delay the use of external beam radiation treatment.
Methods: Six patients with intraocular retinoblastoma (five bilateral; one unilateral, 1 month old) received 6 to 7 monthly cycles of intravenously administered carboplatin and etoposide (VP-16) as primary treatment. No eyes were enucleated primarily. Twelve of the 33 discrete tumors in the 11 study eyes received prophylactic supplemental treatment with cryotherapy or laser hyperthermia. Response was documented with frequent eye examinations with the patient under general anesthesia and with repeated fundus photography.
Results: All eight larger tumors (> 10-mm diameter) underwent dramatic regression after treatment with chemotherapy alone, and six of these tumors ultimately became fully calcific. One larger tumor and two smaller tumors showed post-treatment growth, each within 2 months after completion of chemotherapy. Six larger tumors were observed without growth or further treatment for 7 to 21 months after completion of chemotherapy. Subretinal fluid resorbed completely in four of four eyes with extensive retinal detachment, and vitreous seeding diminished considerably in four of four eyes. In five eyes, intraocular disease recurrence at a distance from any initially observed tumor eventually required treatment with external beam radiation (three eyes) or enucleation (three eyes). Eight of 11 involved eyes were salvaged, including 5 of 8 with larger tumors and 4 of 4 with vitreous seeding; 4 retained eyes received no radiation exposure, including 3 with larger tumors and 1 with vitreous seeding. Good vision was preserved in six eyes, two of which were markedly improved after occlusion therapy for amblyopia. There was no extraocular disease recurrence and no serious harm from treatment during observation ranging from 12 to 40 months after diagnosis.
Conclusion: Chemotherapy with carboplatin and etoposide shows promise as initial treatment for intraocular retinoblastoma. Further study is indicated to define its proper role in the management of this disease.