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Matthew W Wilson, Rachel Brennan, Shenghua Mao, Ibrahim Qaddoumi, Carlos Rodriguez-Galindo; A Prospective Single Institution Trial Using Toptecan Based Chemotherapy for the Treatment of Bilateral Intraocular Retinoblastoma: 5 Year Results.. Invest. Ophthalmol. Vis. Sci. 2014;55(13):6019.
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Purpose: To evaluate efficacy of systemic chemo-reduction using topotecan for advanced intraocular retinoblastoma.
Methods: 27 newly diagnosed bilateral retinoblastoma patients (14 males, median age 7.9 months), worse eye Reese- Ellsworth (RE) Group IV-V, were treated with 11 cycles of chemotherapy: topotecan and vincristine (TV) x 2 followed by 3 alternating courses of carboplatin and vincristine x 2 and TV x 1. Intensive focal therapy was applied after the first 2 cycles. Event free survival (EFS) was defined as avoidance of external beam radiation (EBRT) and enucleation.
Results: Of 54 eyes, 42 were RE IV-V and 36 were International Classification C-E. 25 patients completed all prescribed chemotherapy; one was removed due to persistent viral infection and one had bilateral progressive disease requiring EBRT. 11 eyes were enucleated: 1 at diagnosis, 9 with progressive disease including 3 treated with EBRT, and 1 which developed neovascular glaucoma. At 8 years, cumulative incidence of EBRT was 2.4% (SE±2.4%), EFS for patients was 66.7% (SE± 38.5%), and ocular survival for RE IV-V eyes was 76.2% (SE±26.3). Hematologic toxicities included febrile neutropenia (29 episodes in 275 courses) and 41 episodes of grade 4 thrombocytopenia (1.6 platelet transfusions/patient). Other patient specific toxicities included infection (n=15, 40% viral), grade 3 diarrhea (n=9) and carboplatin reaction (n=1). All patients are alive with median follow up was 4.9 years.
Conclusions: Topotecan combined with vincristine and carboplatin and aggressive focal therapies is and effective regimen for treatment of retinoblastoma. Toxicities were anticipated and managed with appropriate supportive care.
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