Purpose: The risk for death in patients with retinoblastoma is increased in those who present with metastatic disease, and the role of intensive chemotherapy and autologous hematopoietic stem cell rescue in these patients remains unclear.
Design: Nonrandomized interventional case series.
Participants: Four consecutive patients with metastatic retinoblastoma.
Methods: We treated four patients with retinoblastoma metastatic to the bone and bone marrow with intensive chemotherapy, consolidation with megatherapy, and autologous hematopoietic stem cell rescue. Chemotherapy included courses of carboplatin and etoposide alternating with cyclophosphamide, etoposide, and either carboplatin or cisplatin. Radiation therapy was delivered to areas of bone metastases.
Main outcome measures: Patient survival.
Results: All patients completed and responded to the scheduled therapy; complete response of the bone marrow disease was documented after two courses of chemotherapy in all cases. Two patients are long-term survivors.
Conclusions: The treatment described has been successful in obtaining disease-free survival in patients with metastatic retinoblastoma.