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Superselective intra-arterial melphalan therapy for newly diagnosed and refractory retinoblastoma: results from a single institution

Authors Thampi S, Hetts, Cooke D, Stewart P, Robbins E, Banerjee A, Dubois, Char DH, Van Halbach, Matthay KK

Received 29 January 2013

Accepted for publication 11 March 2013

Published 27 May 2013 Volume 2013:7 Pages 981—989


Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 4

Sheila Thampi,1 Steven W Hetts,3 Daniel L Cooke,3 Paul J Stewart,2 Elizabeth Robbins,1 Anuradha Banerjee,1 Steven G DuBois,1 Devron Char,2 Van Halbach,3 Katherine Matthay1

1Department of Pediatrics, 2Department of Ophthalmology, 3Department of Radiology and Biomedical Imaging, Division of Neurointerventional Radiology, University of California, San Francisco School of Medicine, San Francisco, CA, USA

Background: Intra-arterial administration of melphalan chemotherapy has shown promise in the treatment of retinoblastoma. This report describes our results using superselective intra-arterial melphalan in patients with newly diagnosed retinoblastoma and those who were treated for progression after systemic chemotherapy.
Methods: This is a retrospective review of all retinoblastoma patients treated with intra-arterial melphalan at the University of California, San Francisco from March 2010 to August 2012. Twenty eyes (16 patients) underwent 40 intra-arterial melphalan infusions, and dose was determined by age. Patients were treated at monthly intervals and received a range of 1–5 treatments. Response to therapy, toxicity, and procedural radiation exposure was assessed.
Results: All patients are alive without metastatic disease at a median follow-up of 14.5 (1–29) months. Treatment with enucleation or external beam radiation was avoided in 11/20 eyes (55%) overall [6/12 (50%) in newly diagnosed eyes and 5/8 (63%) in refractory/relapsed eyes]. Response rates (per the International Classification of Retinoblastoma) were as follows: 6/7 (86%) in groups A–C and 5/13 (38%) in groups D and E. Nonhematologic and hematologic toxicities were minimal and comparable with those in previous reports. The mean procedural radiation dose was 20.2 ± 11.9 mGy per eye per procedure.
Conclusion: Superselective intra-arterial melphalan therapy is effective for less advanced eyes but further modifications to therapy are required to improve results in eyes with advanced retinoblastoma.

Keywords: retinoblastoma, intra-arterial, melphalan

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