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

Warren P. Mason, Alfred Grovas, Steven Halpern, Ira J. Dunkel, James Garvin, Glenn Heller, Marc Rosenblum, Sharon Gardner, David Lyden, Stephen Sands, Diane Puccetti, Karen Lindsley, Thomas E. Merchant, Bernard O'Malley, Lisa Bayer, Mary Mc Elwain Petriccione, Jeffrey Allen, Jonathan L. Finlay

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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.

Original languageEnglish (US)
Pages (from-to)210-221
Number of pages12
JournalJournal of Clinical Oncology
Volume16
Issue number1
DOIs
StatePublished - Jan 1 1998
Externally publishedYes

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Brain Neoplasms
Bone Marrow
Drug Therapy
Induction Chemotherapy
Consolidation Chemotherapy
Radiotherapy
Etoposide
Disease Progression
Maintenance Chemotherapy
Confidence Intervals
Thiotepa
Carboplatin
Residual Neoplasm
Vincristine
Cyclophosphamide
Cisplatin
Disease-Free Survival
Therapeutics
Survival Rate
Survival

All Science Journal Classification (ASJC) codes

  • Oncology
  • Cancer Research

Cite this

Mason, Warren P. ; Grovas, Alfred ; Halpern, Steven ; Dunkel, Ira J. ; Garvin, James ; Heller, Glenn ; Rosenblum, Marc ; Gardner, Sharon ; Lyden, David ; Sands, Stephen ; Puccetti, Diane ; Lindsley, Karen ; Merchant, Thomas E. ; O'Malley, Bernard ; Bayer, Lisa ; Petriccione, Mary Mc Elwain ; Allen, Jeffrey ; Finlay, Jonathan L. / Intensive chemotherapy and bone marrow rescue for young children with newly diagnosed malignant brain tumors. In: Journal of Clinical Oncology. 1998 ; Vol. 16, No. 1. pp. 210-221.
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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.",
author = "Mason, {Warren P.} and Alfred Grovas and Steven Halpern and Dunkel, {Ira J.} and James Garvin and Glenn Heller and Marc Rosenblum and Sharon Gardner and David Lyden and Stephen Sands and Diane Puccetti and Karen Lindsley and Merchant, {Thomas E.} and Bernard O'Malley and Lisa Bayer and Petriccione, {Mary Mc Elwain} and Jeffrey Allen and Finlay, {Jonathan L.}",
year = "1998",
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Mason, WP, Grovas, A, Halpern, S, Dunkel, IJ, Garvin, J, Heller, G, Rosenblum, M, Gardner, S, Lyden, D, Sands, S, Puccetti, D, Lindsley, K, Merchant, TE, O'Malley, B, Bayer, L, Petriccione, MME, Allen, J & Finlay, JL 1998, 'Intensive chemotherapy and bone marrow rescue for young children with newly diagnosed malignant brain tumors', Journal of Clinical Oncology, vol. 16, no. 1, pp. 210-221. https://doi.org/10.1200/JCO.1998.16.1.210

Intensive chemotherapy and bone marrow rescue for young children with newly diagnosed malignant brain tumors. / Mason, Warren P.; Grovas, Alfred; Halpern, Steven; Dunkel, Ira J.; Garvin, James; Heller, Glenn; Rosenblum, Marc; Gardner, Sharon; Lyden, David; Sands, Stephen; Puccetti, Diane; Lindsley, Karen; Merchant, Thomas E.; O'Malley, Bernard; Bayer, Lisa; Petriccione, Mary Mc Elwain; Allen, Jeffrey; Finlay, Jonathan L.

In: Journal of Clinical Oncology, Vol. 16, No. 1, 01.01.1998, p. 210-221.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Intensive chemotherapy and bone marrow rescue for young children with newly diagnosed malignant brain tumors

AU - Mason, Warren P.

AU - Grovas, Alfred

AU - Halpern, Steven

AU - Dunkel, Ira J.

AU - Garvin, James

AU - Heller, Glenn

AU - Rosenblum, Marc

AU - Gardner, Sharon

AU - Lyden, David

AU - Sands, Stephen

AU - Puccetti, Diane

AU - Lindsley, Karen

AU - Merchant, Thomas E.

AU - O'Malley, Bernard

AU - Bayer, Lisa

AU - Petriccione, Mary Mc Elwain

AU - Allen, Jeffrey

AU - Finlay, Jonathan L.

PY - 1998/1/1

Y1 - 1998/1/1

N2 - 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.

AB - 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.

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