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.
Highlights
Certain pediatric brain tumors such as those of primitive neuroectodermal origin have demonstrated dose-dependent chemotherapy responses [1,2,3] and the principles of highdose chemotherapy with stem-cell rescue have been applied to these pediatric brain tumors to those of other solid tumors [4,5,6]
A total of 18 patients were identified in our comprehensive Pediatric Blood and Marrow Transplant database, who had HDCT-AHSCR from 1999– 2009 for brain tumor treatment
Chang staging principles applied to all tumors and revealed 4 patients with M2, four with M3 and the remaining with M0 status as a highest stage at any time prior to HDCT-AHSCR
Summary
Certain pediatric brain tumors such as those of primitive neuroectodermal origin have demonstrated dose-dependent chemotherapy responses [1,2,3] and the principles of highdose chemotherapy with stem-cell rescue have been applied to these pediatric brain tumors to those of other solid tumors [4,5,6]. High-dose chemotherapy with autologous hematopoietic stem-cell rescue (HDCT-AHSCR) has been used successfully in children with recurrent/refractory or poor-prognosis medulloblastomas, primitive neuroectodermal tumors (PNETs), atypical teratoid/rhabdoid tumors, and central nervous system (CNS) germ cell tumors (GCTs) [7,8,9,10,11,12,13,14,15,16,17]. Variable intensity of regimens and numbers of myeloablations/AHSCRs may potentially result in different outcomes and toxicity profiles. Dose-dependent response makes certain pediatric brain tumors appropriate targets for high-dose chemotherapy with autologous hematopoietic stem-cell rescue (HDCT-AHSCR). 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). HDCT-AHSCR has a definitive, though limited role for selected pediatric brain tumors with poor prognosis and pretransplant complete/partial remissions
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