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L. Mutapcic, T. G. Murray, M. A. Aziz-Sultan, A. C. Schefler, S. Quintero Wolfe, D. J. Hess, C. E. Fernandes, S. R. Dubovy; Supraselective Intra-Ophthalmic Artery Chemotherapy: Evaluation of Treatment Related Complications in Advanced Retinoblastoma. Invest. Ophthalmol. Vis. Sci. 2010;51(13):1579.
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To report the complication profile and safety evaluation of supraselective intra-arterial melphalan chemotherapy in children undergoing treatment with advanced retinoblastoma.
Twelve eyes of 10 children with advanced retinoblastoma (Reese-Ellsworth Vb or ICRB Group D) were treated with supraselective intra-ophthalmic artery infusion of melphalan, a protocol approved by our IRB, as an alternative to enucleation. Children were placed under general anesthesia and anticoagulated for cannulation of the ophthalmic artery with microcatheters via femoral artery approach. Melphalan was infused over a 30-minute period. Serial ophthalmic examinations, retinal photography, and ultrasonographic imaging were used to evaluate treatment regime.
Ophthalmic artery cannulation was successfully performed in 12 eyes of 10 patients (total 16 times). Striking regression of tumor, subretinal and vitreous seeds were seen early in each case. No severe systemic side effects occurred. Mild neutropenia was seen in 4 patients. No transfusions were required. Three patients developed a self-limited vitreous hemorrhage obscuring tumor visualization. One patient developed periocular edema associated with inferior rectus muscle inflammation per orbital MRI. This same patient had scattered intraretinal hemorrhages and peripapillary cotton wool spots consistent with a Purtscher’s-like retinopathy that resolved spontaneously. At 6 month follow-up examination, 9 eyes had no evidence of tumor progression while 3 eyes were enucleated for tumor progression. In each enucleated case, viable tumor was identified on histopathologic examination.
Ophthalmic intra-arterial infusion with melphalan is an excellent globe-conserving treatment option in advanced retinoblastoma cases with minimal systemic side effects. Local toxicities include microemboli to the retina and choroid (1/12, 8%), vitreous hemorrhage (3/12, 25%), and myositis (1/12, 8%). Enucleation remained a definitive treatment for tumor progression in 3 of 12 patients in this small case series with limited follow up. Further studies are necessary to establish the role of supraselective intra-arterial melphalan chemotherapy for children with retinoblastoma.
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