title: High-dose chemotherapy for high-risk retinoblastoma: clinical course and outcome of 14 cases in the National Cancer Center, Japan.
abstract: The prognosis of high-risk retinoblastoma (RB) with extraocular disease, relapse, or invasion of the cut end of the optic nerve is extremely poor. Following the discontinuation of thiotepa production in Japan, BU- and melphalan (Mel)-based regimens have been used, followed by the standard treatment for neuroblastoma. This study retrospectively analyzed 14 high-risk RB patients who underwent high-dose chemotherapy (HDC) and hematopoietic SCT; 8 received a BU/Mel conditioning regimen and 6 received other regimens. The disease status at HDC was relapse in 8 patients and extraocular involvement in 5. All patients received peripheral blood stem cell infusion >1.5 × 10(6)/kg. Engraftment occurred within a median of 11 days (BU/Mel: 10-13, others: 9-13). Primary toxicities included mucositis (⩾grade 3) in 9 patients (4 with BU/Mel, 5 with others). Veno-occlusive disease (VOD) occurred in two 1-year-old patients in the BU/Mel group. There were no treatment-related deaths. Of 4 (2 with BU/Mel, 2 with others) patients with central nervous system (CNS) relapse after HDC, 3 died. In conclusion, the BU/Mel regimen may be feasible for high-risk RB under careful monitoring for VOD, particularly in younger patients. CNS relapse associated with a lethal prognosis occurred after all regimens; therefore, further evaluation of HDC efficacy for high-risk RB is required.
authors.affiliation: Division of Pediatric Oncology, National Cancer Center, Tokyo, Japan.
authors.forename: N
authors.initials: N
authors.lastname: Yasui
authors.affiliation: Division of Pediatric Oncology, National Cancer Center, Tokyo, Japan.
authors.forename: H
authors.initials: H
authors.lastname: Kawamoto
authors.affiliation: Department of Ophthalmic Oncology, National Cancer Center Hospital, Tokyo, Japan.
authors.forename: M
authors.initials: M
authors.lastname: Fujiwara
authors.affiliation: Department of Ophthalmic Oncology, National Cancer Center Hospital, Tokyo, Japan.
authors.forename: Y
authors.initials: Y
authors.lastname: Aihara
authors.affiliation: Division of Pediatric Oncology, National Cancer Center, Tokyo, Japan.
authors.forename: C
authors.initials: C
authors.lastname: Ogawa
authors.affiliation: Division of Pediatric Oncology, National Cancer Center Hospital East, Tokya, Japan.
authors.forename: A
authors.initials: A
authors.lastname: Hosono
authors.affiliation: Department of Ophthalmic Oncology, National Cancer Center Hospital, Tokyo, Japan.
authors.forename: S
authors.initials: S
authors.lastname: Suzuki
mesh_terms: D064591:Allografts; D002066:Busulfan / Q000008:administration & dosage*; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D007564:Japan; D008297:Male; D008558:Melphalan / Q000008:administration & dosage*; D019653:Myeloablative Agonists / Q000008:administration & dosage*; D036102:Peripheral Blood Stem Cell Transplantation; D019572:Retinal Neoplasms / Q000175:diagnosis / Q000628:therapy*; D012175:Retinoblastoma / Q000175:diagnosis / Q000628:therapy*; D012189:Retrospective Studies; D019172:Transplantation Conditioning
chemical_list: D019653:Myeloablative Agonists; D002066:Busulfan; D008558:Melphalan