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

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 Matthay<sup>1

1</sup>Department 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