Treatment outcomes of extra-ocular retinoblastoma after multimodal therapy: a prospective study.

INTRODUCTION: This prospective study was undertaken to evaluate the outcomes of multimodal therapy in extra-ocular retinoblastoma (EORB) and to identify clinical and treatment-related factors influencing survival. METHODS: Children presenting with EORB [International Retinoblastoma Staging System (IRSS) stage II, III, or IV] were enrolled. Stage II were treated with adjuvant chemotherapy and external beam radiotherapy (EBRT), following enucleation. Stage III received neoadjuvant chemotherapy, secondary enucleation, adjuvant chemotherapy, and EBRT. Stage IVA was treated with induction chemotherapy and autologous stem cell transplantation, while Stage IVB received palliative care. RESULTS: Thirty-five patients (median age, 36.9 months) were included. The overall Kaplan-Meier survival was 71.4% at 24 months (95% CI, 0.59-0.84). The Kaplan-Meier survival for Stage II, III and IVA patients was 85.7% at 24 months (95% CI, 0.68-0.94). Central nervous system progression was the leading cause of death. Optic nerve invasion beyond the orbital apex and omission of EBRT was significantly associated with lower survival (p < 0.01 and p = 0.03 respectively). CONCLUSIONS: Early intervention with protocol-based multimodal therapy can significantly improve survival outcomes in Stage II, III, and IVA EORB. The extent of optic nerve invasion and administration of EBRT are critical factors for survival.