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Three-drug intra-arterial chemotherapy using simultaneous carboplatin, topotecan and melphalan for intraocular retinoblastoma: preliminary results
  1. Brian P Marr1,
  2. Scott E Brodie1,2,
  3. Ira J Dunkel3,
  4. Y Pierre Gobin4,
  5. David H Abramson1
  1. 1Ophthalmic Oncology Service, Memorial Sloan-Kettering Cancer Center, New York, New York, USA
  2. 2Department of Ophthalmology, Mt Sinai School of Medicine, New York, New York, USA
  3. 3Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, New York, USA
  4. 4Departments of Neurosurgery, Radiology and Neurology, Weill Cornell Medical College, New York, New York, USA
  1. Correspondence to Dr Brian P Marr, Ophthalmic Oncology Service, Memorial Sloan-Kettering Cancer Center, 70 East 66th Street, New York, NY 10021, USA; marrb{at}


Aims To report outcomes with selective intra-arterial chemotherapy (SIAC) using simultaneous carboplatin, topotecan, and melphalan for advanced intraocular retinoblastoma.

Methods A retrospective chart review was conducted of patients who received three-drug (melphalan, topotecan, and carboplatin) SIAC during 2006–2011.

Results Twenty-six eyes of 25 patients received the three-drug chemotherapy for treatment of advanced retinoblastoma. Reese-Ellsworth group was 5b in 21 eyes, 5a in 2, 4a in 2, and 3a in 1. Seventeen patients (68%) had recurrence after prior intravenous chemotherapy with or without radiotherapy. In the three-drug therapy, dose ranges were 2.5–7.5 mg for melphalan, 0.3–0.6 mg for topotecan, and 25–50 mg for carboplatin, and median infusions per eye was 2 (range 1–4). At a mean follow-up of 14 months (range 1–43 months), all patients are alive and no patient developed metastatic disease. Twenty-three of 26 eyes (88%) survived. Eleven of the 26 eyes (35%) developed recurrent disease and were treated with enucleation (n=3) or with focal therapy (n=8) with or without plaque brachytherapy (n=3). The Kaplan-Meier estimate of ocular survival at 24 months was 75% (95% CI). Electroretinogram showed improvement greater than 25 µV in 4 eyes (15%), loss greater than 25 µV in 12 eyes (46%), and no change greater than 25 µV in 10 eyes (39%).

Conclusions Three-drug SIAC has been used successfully to rescue eyes after treatment failure of intravenous chemotherapy and/or single- or double-agent SIAC. Twenty-three of 26 eyes avoided both enucleation and external beam radiotherapy and retained electroretinogram function.

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