Intensive chemotherapy and bone marrow rescue for young children with newly diagnosed malignant brain tumors Journal Article


Authors: Mason, W. P.; Grovas, A.; Halpern, S.; Dunkel, I. J.; Garvin, J.; Heller, G.; Rosenblum, M.; Gardner, S.; Lyden, D.; Sands, S.; Puccetti, D.; Lindsley, K.; Merchant, T. E.; O'Malley, B.; Bayer, L.; Petriccione, M. M.; Allen, J.; Finlay, J. L.
Article Title: Intensive chemotherapy and bone marrow rescue for young children with newly diagnosed malignant brain tumors
Abstract: 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 far 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.
Keywords: cancer chemotherapy; cancer survival; preschool child; treatment outcome; child, preschool; major clinical study; drug tolerability; neutropenia; cisplatin; cancer radiotherapy; combined modality therapy; brain tumor; brain neoplasms; infection; neoplasm recurrence, local; bone marrow suppression; etoposide; mucosa inflammation; nausea; thrombocytopenia; vomiting; antineoplastic combined chemotherapy protocols; cyclophosphamide; vincristine; thiotepa; infant; disease progression; hyperpigmentation; remission; remission induction; sepsis; bone marrow transplantation; transplantation, autologous; bone marrow rescue; hearing loss; electrolyte disturbance; autologous bone marrow transplantation; humans; human; male; female; priority journal; article
Journal Title: Journal of Clinical Oncology
Volume: 16
Issue: 1
ISSN: 0732-183X
Publisher: American Society of Clinical Oncology  
Date Published: 1998-01-01
Start Page: 210
End Page: 221
Language: English
PUBMED: 9440745
PROVIDER: scopus
DOI: 10.1200/JCO.1998.16.1.210
DOI/URL:
Notes: Article -- Export Date: 12 December 2016 -- Source: Scopus
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MSK Authors
  1. Glenn Heller
    303 Heller
  2. Ira J Dunkel
    255 Dunkel
  3. David C Lyden
    84 Lyden
  4. Marc Rosenblum
    255 Rosenblum
  5. Jonathan Finlay
    62 Finlay
  6. Stephen Alan Sands
    5 Sands