Treatment of overt extraocular retinoblastoma.

Background: Overt extraocular retinoblastoma is common in developing countries and little information about its treatment is available. The aim of this study is to report our experience in the treatment of these cases using a uniform approach.

Procedure: Patients with overt extraocular retinoblastoma including orbital extension, preauricular lymph node invasion and/or metastatic disease on diagnosis or after extraocular relapse admitted to the Hospital JP Garrahan from August 1987 to December 2000 were retrospectively reviewed. Patients were treated according to two different protocols (1987-1993 and 1994-2000). Treatment included: neoadjuvant combination chemotherapy followed by limited surgery in case of orbital extension (enucleation or resection of residual orbital mass) and adjuvant chemotherapy and radiotherapy. Chemotherapy included cyclophosphamide, vincristine, etoposide, doxorubicin (in protocol 87), idarubicin (in protocol 94), cisplatin (in protocol 87), and carboplatin (in protocol 94).

Results: Forty-one patients were included. Fifteen of them had orbital or preauricular disease and had a 5-year event-free survival (pEFS) of 84%. Twenty-six had distant metastatic disease and non survived 5-years. One patient died of toxicity and one died in complete remission. One patient had a secondary leukemia. The remaining adverse events included CNS and/or systemic relapse.

Conclusions: This treatment strategy was highly efficacious for patients with orbital and/or lymph node extension. Orbital exenteration is not necessary for these patients. Those patients with distant metastatic or CNS disease were not curable with this approach.