title: High-dose chemotherapy with autologous hematopoietic stem-cell rescue for pediatric brain tumor patients: a single institution experience from UCLA.
abstract: Background. Dose-dependent response makes certain pediatric brain tumors appropriate targets for high-dose chemotherapy with autologous hematopoietic stem-cell rescue (HDCT-AHSCR). Methods. The clinical outcomes and toxicities were analyzed retrospectively for 18 consecutive patients ≤19 y/o treated with HDCT-AHSCR at UCLA (1999-2009). Results. Patients' median age was 2.3 years. Fourteen had primary and 4 recurrent tumors: 12 neural/embryonal (7 medulloblastomas, 4 primitive neuroectodermal tumors, and a pineoblastoma), 3 glial/mixed, and 3 germ cell tumors. Eight patients had initial gross-total and seven subtotal resections. HDCT mostly consisted of carboplatin and/or thiotepa ± etoposide (n = 16). Nine patients underwent a single AHSCR and nine ≥3 tandems. Three-year progression-free and overall survival probabilities were 60.5% ± 16 and 69.3% ± 11.5. Ten patients with pre-AHSCR complete remissions were alive/disease-free, whereas 5 of 8 with measurable disease were deceased (median followup: 2.3 yrs). Nine of 13 survivors avoided radiation. Single AHSCR regimens had greater toxicity than ≥3 AHSCR (P < .01). Conclusion. HDCT-AHSCR has a definitive, though limited role for selected pediatric brain tumors with poor prognosis and pretransplant complete/partial remissions.
authors.affiliation: UCLA Medical Center, Mattel Children's Hospital, Los Angeles, CA 90095, USA.
authors.forename: Eduard H
authors.initials: EH
authors.lastname: Panosyan
authors.forename: Alan K
authors.initials: AK
authors.lastname: Ikeda
authors.forename: Vivian Y
authors.initials: VY
authors.lastname: Chang
authors.forename: Dan R
authors.initials: DR
authors.lastname: Laks
authors.forename: Charles L
authors.initials: CL
authors.lastname: Reeb
authors.forename: La Vette
authors.initials: LV
authors.lastname: Bowles
authors.forename: Joseph L
authors.initials: JL
authors.lastname: Lasky
authors.forename: Theodore B
authors.initials: TB
authors.lastname: Moore