1. ¹Pediatric Oncology Division, National Cancer Center Hospital, Tokyo, Japan
2. ²Hematopoietic Stem Cell Transplantation Division, National Cancer Center Hospital, Tokyo, Japan
3. ³Radiation Oncology Division, National Cancer Center Hospital, Tokyo, Japan
4. ⁴Ophthalmology Division, National Cancer Center Hospital, Tokyo, Japan

Correspondence: Dr H Matsubara, Department of Pediatrics, Graduate School of Medicine, Kyoto University, 54 Kawahara-cho, Shogoin, Sakyo-ku 606-8507, Kyoto, Japan. E-mail: matsu-ncc@umin.ac.jp

Received 4 February 2004; Accepted 15 December 2004; Published online 7 March 2005.

Abstract

The prognosis of patients with metastatic retinoblastoma is poor with conventional chemotherapy and radiation. Since retinoblastoma is highly chemosensitive, dose-escalation of chemotherapeutic agents with stem cell support should be promising. We report our experience with high-dose chemotherapy (HDC) and autologous stem cell transplantation (SCT) in patients with metastatic retinoblastoma. Five patients with metastatic retinoblastoma underwent HDC with autologous SCT following conventional chemotherapy and local radiation therapy. Stem cells (bone marrow in four and peripheral blood stem cells in one) were collected after marrow involvement was cleared. Melphalan was a key drug in all patients, and was administered in combination with other agents such as cisplatin, cyclophosphamide, carboplatin or thiotepa. Three patients are currently alive disease-free at 113, 107 and 38 months, respectively, from the time of SCT. They had no central nervous system (CNS) involvement. The two patients who died of disease had CNS involvement. No long-term sequelae of HDC have been noted. Our treatment strategy using HDC appears to be effective for treating metastatic retinoblastoma without CNS involvement.