Journal of Clinical Oncology, Vol 16, 210-221, Copyright © 1998 by American Society of Clinical Oncology
Intensive chemotherapy and bone marrow rescue for young children with newly diagnosed malignant brain tumors
WP Mason, A Grovas, S Halpern, IJ Dunkel, J Garvin, G Heller, M Rosenblum, S Gardner, D Lyden, S Sands, D Puccetti, K Lindsley, TE Merchant, B O'Malley, L Bayer, MM Petriccione, J Allen and JL Finlay
Memorial Sloan-Kettering Cancer Center, New York, NY 10021, USA.
PURPOSE: To evaluate a strategy that avoids radiotherapy in children less
than 6 years of age with newly diagnosed malignant brain tumors, by
administering myeloablative consolidation chemotherapy with autologous bone
marrow reconstitution (ABMR) after maximal surgical resection and
conventional induction chemotherapy. PATIENTS AND METHODS: Between March
1991 and April 1995, 62 children (median age, 30 months) with newly
diagnosed malignant brain tumors were enrolled onto this trial. Children
received conventional induction chemotherapy with vincristine, cisplatin,
cyclophosphamide, and etoposide, repeated every 3 weeks for five cycles.
Children without disease progression on induction chemotherapy were offered
consolidation with myeloablative chemotherapy that incorporated
carboplatin, thiotepa, and etoposide followed by ABMR. Irradiation was used
only for residual tumor at consolidation or for progressive/recurrent
disease. RESULTS: Induction chemotherapy was well tolerated by most
patients; however, progression was noted in 17 children (27%) and four (6%)
died of treatment complications. Of 37 children who received consolidation
chemotherapy with ABMR, 15 are free of disease progression (median
post-ABMR without further treatment, >44 months). The remaining 22 all
progressed within 15 months of ABMR; three of 37 (8%) died of
treatment-related complications. The 3-year overall survival (OS) and
event-free survival (EFS) rates from diagnosis for all children are 40%
(95% confidence interval [CI], 28% to 52%) and 25% (95% CI, 13% to 37%),
respectively. Radiotherapy was administered to 19 of 62 children: 17 for
progressive disease (PD) and two for residual disease at the time of ABMR.
CONCLUSION: A significant proportion of children with malignant brain
tumors can avoid radiotherapy and prolonged maintenance chemotherapy yet
still achieve durable remission with this brief intensive chemotherapy
regimen.
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