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Qi Cui, Vasiliki Aivaliotis, Joan O'Brien, Paul Stewart; Topotecan and Cyclophosphamide as Salvage Therapy in the Treatment of Retinoblastoma. Invest. Ophthalmol. Vis. Sci. 2013;54(15):3977.
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Retinoblastoma is the leading primary ocular malignancy of childhood. Currently, first-line therapy for intraocular retinoblastoma consists of systemic chemoreduction in combination with focal consolidative therapy. More recently, intra-arterial chemotherapy has gained momentum as a new option for the management of unilateral intraocular retinoblastoma. In this study, we examined the effectiveness of a novel two-agent chemotherapy regimen as salvage therapy for children who failed initial treatment.
We conducted a cross-sectional retrospective chart review looking at cases of retinoblastoma that received treatment at the University of California, San Francisco between 1994 and 2010. Fifty-two patients were identified. Of these, eight patients and 14 globes failed primary chemotherapy and subsequently received salvage chemotherapy consisting of Topotecan and Cyclophosphamide. Outcome evaluated include the ICRB disease stage, tumor recurrence, the need for enucleation, side effects, and mortality.
Three globes were stage B, five were stage C, four were stage D, and two were stage E at the time of diagnosis. The duration of follow-up was 19.6 ±10.2 (mean ± SD) months, and the age at last follow up was 54.3 ± 12.7 months. Treatment with Topotecan and Cyclophosphamide was associated with globe salvage in 10 out of 14 eyes (71%) overall, and 10 out of 12 eyes (83%) in bilateral cases. In those with OU involvement, two globes were enucleated and one patient experienced recurrence following treatment. Both subjects with unilateral involvement were enucleated. Systemic side effects included anemia (1 subject), neurosensory hearing loss (1 subject), neutropenia (2 subjects), and an allergic reaction to carboplatin (1 subject).
Administration of novel two-agent systemic chemotherapy as salvage treatment for retinoblastoma was associated with 71% globe preservation overall. This is comparable to the effect of intra-arterial (ophthalmic artery) chemotherapy using melphalan, carboplatin, and/or topotecan as reported by Abramson et al. 2008 (1) In addition, systemic chemotherapy is not limited by laterality and demonstrated 83% globe preservation in bilateral cases. 1). Abramson DH, Dunke IJ, Brodie SE et al. A phase I/II study of direct intraarterial (ophthalmic artery) chemotherapy with melphalan for intraocular retinoblastoma initial results. Ophthalmoloy 2008; 115: 1398-404.
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