Abstract 958: Improving survival of extraocular, metastatic and trilateral retinoblastoma patients with combined intensive therapy.

Abstract PURPOSE: Most extraocular, metastatic and trilateral retinoblastoma (RB) patients manifest multidrug resistance due to P-glycoprotein and few survive beyond a year when treated with conventional chemotherapy and radiation. We report results of cyclosporine circumvention of multidrug resistance to carboplatin, etoposide and vincristine (CEV), combined with consolidation with a myeloablative conditioning regimen (high-dose carboplatin, etoposide and cyclophosphamide), with autologous stem cell transplantation (ASCT) for stem cell rescue. METHOD: Since 1998, we treated 9 such young children: three (ages 6.3, 1.3 and 2.3 years) with non-germline RB with massive optic nerve/optic chiasm involvement (resection and orbital radiation), one (age 4.8 years) with germline RB and mandibular metastasis (totally resection after CEV-response avoiding radiation), one (age 5 months) with germline RB and leptomeningeal disease (intraventricular cytarabine/topotecan-response avoiding craniospinal radiation), one (age 2.6 years) with non-germline RB with orbital/intracranial/leptomeningeal recurrence (intraventricular cytarabine/topotecan and orbital radiation), and three (ages 3, 5 and 27 months) with germline trilateral RB with leptomeningeal disease (intraventricular cytarabine/topotecan-response avoiding craniospinal radiation). RESULTS: Eight children have completed therapy, and one child with trilateral suprasellar RB and leptomeningeal disease is still on treatment prior to ASCT. Two patients died: one child with orbital/intracranial/leptomeningeal recurrence died of disease 1.8 years post-metastasis (1.4 years post ASCT); one child with trilateral suprasellar RB with leptomeningeal disease died of disease 2.7 years post-diagnosis (2 years post ASCT), resulting from a local recurrence along the needle track site of the original suprasellar biopsy. Six children of the 8 children who completed therapy are alive at a median of 12.5 years from diagnosis (range 5.3-13.3 years): 3 children with massive optic nerve involvement are relapse-free 12.7, 13.3 and 12.6 years post-diagnosis (12.3, 12.8, 12.3 years post ASCT); one child with mandibular recurrence is relapse-free 12.4 years post-metastasis (11.7 years post ASCT); one child with leptomeningeal RB is relapse-free 12.1 years post-diagnosis (11.6 years post ASCT); and one child with trilateral suprasellar RB with leptomeningeal disease is relapse-free 5.3 years post-diagnosis (4.8 years post ASCT). CONCLUSION: Six of eight children who have completed therapy for extraocular, metastatic and trilateral retinoblastoma, who in the past would have died, may be cured by intensive multimodality therapy. Citation Format: Helen SL Chan, Elise Héon, Tal Schechter, Helen Dimaras, Brenda L. Gallie, John J. Doyle. Improving survival of extraocular, metastatic and trilateral retinoblastoma patients with combined intensive therapy. [abstract]. In: Proceedings of the 104th Annual Meeting of the American Association for Cancer Research; 2013 Apr 6-10; Washington, DC. Philadelphia (PA): AACR; Cancer Res 2013;73(8 Suppl):Abstract nr 958. doi:10.1158/1538-7445.AM2013-958

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