Successful treatment of metastatic retinoblastoma

BACKGROUND. In the past, patients with metastatic retinoblastoma have had a poor prognosis when treated with conventional modalities. In the current s tudy, the authors evaluated the use of combined intensive conventional chem otherapy, high dose chemotherapy with autologous stem cell rescue (ASCR), a nd radiation therapy. METHODS. Four patients with metastatic retinoblastoma were treated. All had orbital and bone marrow metastases. In addition, three patients had bone m etastases and two patients had liver metastases. None had central nervous s ystem disease. Patients received intensive conventional chemotherapy that i ncluded vincristine, cyclophosphamide, etoposide, and either cisplatin or c arboplatin. Stem cells were harvested after bone marrow disease was no long er detectable. High dose chemotherapy with carboplatin (500 m/m(2)/day x 3 days or area under the curve = 7 via the Calvert formula) and thiotepa (300 mg/m(2)/day x 3 days) with (n = 3 patients) or without (n = 1 patient) eto poside (250 mg/m(2)/day x 3 days) was administered with ASCR. Sites that or iginally harbored bulky disease were irradiated after recovery from the hig h dose chemotherapy. RESULTS. The therapy was associated with substantial acute hematopoietic an d mucosal toxicities. At last follow-up, all four patients had survived eve nt free from 46-80 months after the diagnosis of metastatic disease. CONCLUSIONS. The treatment strategy described in the current study is effec tive for patients with metastatic retinoblastoma that does not involve the central nervous system. However, a multicenter trial should be considered t o evaluate it in a larger group of patients. (C) 2000 American Cancer Socie ty.