title: Autologous hematopoietic stem cell transplantation for high-risk brain tumors in children.
abstract: Autologous hematopoietic stem cell transplant (AHSCT) has been advocated as a form of salvage therapy for children with high-risk or relapsed brain tumors but only limited data are available currently. We report the outcomes of pediatric brain tumors treated with AHSCT in a quaternary referral center in Hong Kong over 10 years (June 1996-May 2006). Thirteen patients with medulloblastoma (n = 9), cerebral primitive neuroectodermal tumor (n = 1), ependymoma (n = 1), germ cell tumor (n = 1) and cerebellar rhabdoid (n = 1) were transplanted because of tumor residual (n = 1) or recurrence (n = 12). Uniform upfront treatment protocols were adopted according to specific tumor types. Prior to AHSCT, 8 patients (61.5%) achieved complete remission and 5 (38.5%) were in partial remission. Conditioning employed thiotepa 300 mg/m2, etoposide 250 mg/m2)and carboplatin 500 mg/m2 daily for 3 days. Toxicity included mucositis and neutropenic fever in all patients, grade 4 hepatic toxicity in 4 patients (including hepatic veno-occlusive disease in 2 patients) and grade 4 renal toxicity in 1 patient. The 5-year event-free survival was 53.9%. Five patients died of disease recurrence or progression 8-21 months after transplant with a median disease-free period of 8 months post-transplant. One died of transplant-related complications in the early post-transplant period. Seven survived for a median of 5.4 years (maximum follow-up of 9.8 years), with six having Lansky-Karnofsky performance score above 80. All survivors had complete remission before transplant though 2 had leptomeningeal spread. We conclude that AHSCT can achieve long-term survival in children with recurrent brain tumor. However, those with macroscopic residual tumor before transplant cannot be salvaged.
authors: Department of Pediatrics and Adolescent Medicine, Queen Mary Hospital, The University of Hong Kong, 121 Pokfulam Road, Hong Kong SAR, China. cheukkld@hkucc.hku.hk
authors: Daniel Ka Leung
authors: DK
authors: Cheuk
authors: Tsz Leung
authors: TL
authors: Lee
authors: Alan Kwok Shing
authors: AK
authors: Chiang
authors: Shau Yin
authors: SY
authors: Ha
authors: Godfrey Chi Fung
authors: GC
authors: Chan
mesh_terms: D000293:Adolescent; D001932:Brain Neoplasms / Q000145:classification / Q000401:mortality / Q000601:surgery*; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008297:Male; D012189:Retrospective Studies; D016019:Survival Analysis; D014182:Transplantation, Autologous / Q000379:methods*; D016896:Treatment Outcome