title: A multidisciplinary treatment strategy that includes high-dose chemotherapy for metastatic retinoblastoma without CNS involvement.
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.
authors.affiliation: Pediatric Oncology Division, National Cancer Center Hospital, Tokyo, Japan. matsu-ncc@umin.ac.jp
authors.forename: H
authors.initials: H
authors.lastname: Matsubara
authors.forename: A
authors.initials: A
authors.lastname: Makimoto
authors.forename: T
authors.initials: T
authors.lastname: Higa
authors.forename: H
authors.initials: H
authors.lastname: Kawamoto
authors.forename: S
authors.initials: S
authors.lastname: Sakiyama
authors.forename: A
authors.initials: A
authors.lastname: Hosono
authors.forename: J
authors.initials: J
authors.lastname: Takayama
authors.forename: Y
authors.initials: Y
authors.lastname: Takaue
authors.forename: S
authors.initials: S
authors.lastname: Murayama
authors.forename: M
authors.initials: M
authors.lastname: Sumi
authors.forename: A
authors.initials: A
authors.lastname: Kaneko
authors.forename: M
authors.initials: M
authors.lastname: Ohira
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001854:Bone Marrow Cells / Q000166:cytology; D016190:Carboplatin / Q000008:administration & dosage; D002490:Central Nervous System / Q000473:pathology; D002675:Child, Preschool; D002945:Cisplatin / Q000008:administration & dosage; D003131:Combined Modality Therapy / Q000379:methods; D003520:Cyclophosphamide / Q000008:administration & dosage; D018572:Disease-Free Survival; D004305:Dose-Response Relationship, Drug; D005134:Eye Neoplasms / Q000628:therapy*; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D009362:Neoplasm Metastasis; D009423:Nervous System Neoplasms / Q000628:therapy; D011379:Prognosis; D012175:Retinoblastoma / Q000628:therapy*; D033581:Stem Cell Transplantation / Q000379:methods; D013234:Stem Cells / Q000166:cytology; D013852:Thiotepa / Q000008:administration & dosage; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D003520:Cyclophosphamide; D013852:Thiotepa; D016190:Carboplatin; D002945:Cisplatin; D008558:Melphalan