High-dose chemotherapy with autologous stem cell rescue for children with recurrent malignant brain tumors

Chie Schin Shih, Gregory A. Hale, Lindsey Gronewold, Xin Tong, Fred H. Laningham, Elizabeth A. Gilger, Deo Kumar Srivastava, Larry E. Kun, Amar Gajjar, Maryam Fouladi

Research output: Contribution to journalArticle

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Abstract

BACKGROUND. High-dose chemotherapy (HDCT) with autologous stem cell rescue (ASCR) has been reported to be effective in treating children with recurrent central nervous system (CNS) malignancies. METHODS. To evaluate the efficacy and toxicities of HDCT and ASCR, the medical records of 27 children with recurrent CNS malignancies who received such therapy at St. Jude Children's Research Hospital between 1989 and 2004 were reviewed. RESULTS. The median age at diagnosis was 4.5 years (range, 0.4-16.6 years) and that at ASCR was 6.7 years (range, 1.1-18.5 years). Diagnoses included medulloblastoma (13 patients), primitive neuroectodermal tumor (3 patients), pineoblastoma (2 patients), atypical teratoid rhabdoid tumor (2 patients), ependymoma (3 patients), anaplastic astrocytoma (2 patients), and glioblastoma multiforme (2 patients). The 5-year overall and progression-free survival (PFS) rates were 28.2% and 18.5%, respectively. The 5-year PFS rate for patients aged <3 years at diagnosis (57.1%) was significantly better than older patients (5.0%) (P = .019). Among the 6 long-term survivors (5 with M0 disease and 1 with M3 disease at diagnosis), 5 received both radiotherapy and HDCT as part of their salvage regimen; 4 were aged <3 years at diagnosis and had received chemotherapy only as part of frontline therapy. Two patients died of transplant-related toxicities; 44% experienced grade 3 or 4 transplant-related toxicities (toxicities were graded according to the National Cancer Institute Common Toxicity Criteria). CONCLUSIONS. HDCT with ASCR is not an effective salvage strategy for older children with recurrent CNS malignancies. The significantly better outcome in the younger cohort was most likely related to the use of radiotherapy as part of the salvage strategy.

Original languageEnglish (US)
Pages (from-to)1345-1353
Number of pages9
JournalCancer
Volume112
Issue number6
DOIs
StatePublished - Mar 15 2008

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Brain Neoplasms
Stem Cells
Drug Therapy
Central Nervous System
Disease-Free Survival
Radiotherapy
Survival Rate
Pinealoma
Transplants
Primitive Neuroectodermal Tumors
Ependymoma
Neoplasms
Medulloblastoma
National Cancer Institute (U.S.)
Astrocytoma
Glioblastoma
Medical Records
Survivors
Therapeutics
Research

Keywords

  • Autologous stem cell transplantation
  • Central nervous system neoplasms
  • High-dose chemotherapy
  • Recurrent
  • Salvage therapy

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

Shih, C. S., Hale, G. A., Gronewold, L., Tong, X., Laningham, F. H., Gilger, E. A., ... Fouladi, M. (2008). High-dose chemotherapy with autologous stem cell rescue for children with recurrent malignant brain tumors. Cancer, 112(6), 1345-1353. https://doi.org/10.1002/cncr.23305

High-dose chemotherapy with autologous stem cell rescue for children with recurrent malignant brain tumors. / Shih, Chie Schin; Hale, Gregory A.; Gronewold, Lindsey; Tong, Xin; Laningham, Fred H.; Gilger, Elizabeth A.; Srivastava, Deo Kumar; Kun, Larry E.; Gajjar, Amar; Fouladi, Maryam.

In: Cancer, Vol. 112, No. 6, 15.03.2008, p. 1345-1353.

Research output: Contribution to journalArticle

Shih, CS, Hale, GA, Gronewold, L, Tong, X, Laningham, FH, Gilger, EA, Srivastava, DK, Kun, LE, Gajjar, A & Fouladi, M 2008, 'High-dose chemotherapy with autologous stem cell rescue for children with recurrent malignant brain tumors', Cancer, vol. 112, no. 6, pp. 1345-1353. https://doi.org/10.1002/cncr.23305
Shih CS, Hale GA, Gronewold L, Tong X, Laningham FH, Gilger EA et al. High-dose chemotherapy with autologous stem cell rescue for children with recurrent malignant brain tumors. Cancer. 2008 Mar 15;112(6):1345-1353. https://doi.org/10.1002/cncr.23305
Shih, Chie Schin ; Hale, Gregory A. ; Gronewold, Lindsey ; Tong, Xin ; Laningham, Fred H. ; Gilger, Elizabeth A. ; Srivastava, Deo Kumar ; Kun, Larry E. ; Gajjar, Amar ; Fouladi, Maryam. / High-dose chemotherapy with autologous stem cell rescue for children with recurrent malignant brain tumors. In: Cancer. 2008 ; Vol. 112, No. 6. pp. 1345-1353.
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abstract = "BACKGROUND. High-dose chemotherapy (HDCT) with autologous stem cell rescue (ASCR) has been reported to be effective in treating children with recurrent central nervous system (CNS) malignancies. METHODS. To evaluate the efficacy and toxicities of HDCT and ASCR, the medical records of 27 children with recurrent CNS malignancies who received such therapy at St. Jude Children's Research Hospital between 1989 and 2004 were reviewed. RESULTS. The median age at diagnosis was 4.5 years (range, 0.4-16.6 years) and that at ASCR was 6.7 years (range, 1.1-18.5 years). Diagnoses included medulloblastoma (13 patients), primitive neuroectodermal tumor (3 patients), pineoblastoma (2 patients), atypical teratoid rhabdoid tumor (2 patients), ependymoma (3 patients), anaplastic astrocytoma (2 patients), and glioblastoma multiforme (2 patients). The 5-year overall and progression-free survival (PFS) rates were 28.2{\%} and 18.5{\%}, respectively. The 5-year PFS rate for patients aged <3 years at diagnosis (57.1{\%}) was significantly better than older patients (5.0{\%}) (P = .019). Among the 6 long-term survivors (5 with M0 disease and 1 with M3 disease at diagnosis), 5 received both radiotherapy and HDCT as part of their salvage regimen; 4 were aged <3 years at diagnosis and had received chemotherapy only as part of frontline therapy. Two patients died of transplant-related toxicities; 44{\%} experienced grade 3 or 4 transplant-related toxicities (toxicities were graded according to the National Cancer Institute Common Toxicity Criteria). CONCLUSIONS. HDCT with ASCR is not an effective salvage strategy for older children with recurrent CNS malignancies. The significantly better outcome in the younger cohort was most likely related to the use of radiotherapy as part of the salvage strategy.",
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AU - Shih, Chie Schin

AU - Hale, Gregory A.

AU - Gronewold, Lindsey

AU - Tong, Xin

AU - Laningham, Fred H.

AU - Gilger, Elizabeth A.

AU - Srivastava, Deo Kumar

AU - Kun, Larry E.

AU - Gajjar, Amar

AU - Fouladi, Maryam

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N2 - BACKGROUND. High-dose chemotherapy (HDCT) with autologous stem cell rescue (ASCR) has been reported to be effective in treating children with recurrent central nervous system (CNS) malignancies. METHODS. To evaluate the efficacy and toxicities of HDCT and ASCR, the medical records of 27 children with recurrent CNS malignancies who received such therapy at St. Jude Children's Research Hospital between 1989 and 2004 were reviewed. RESULTS. The median age at diagnosis was 4.5 years (range, 0.4-16.6 years) and that at ASCR was 6.7 years (range, 1.1-18.5 years). Diagnoses included medulloblastoma (13 patients), primitive neuroectodermal tumor (3 patients), pineoblastoma (2 patients), atypical teratoid rhabdoid tumor (2 patients), ependymoma (3 patients), anaplastic astrocytoma (2 patients), and glioblastoma multiforme (2 patients). The 5-year overall and progression-free survival (PFS) rates were 28.2% and 18.5%, respectively. The 5-year PFS rate for patients aged <3 years at diagnosis (57.1%) was significantly better than older patients (5.0%) (P = .019). Among the 6 long-term survivors (5 with M0 disease and 1 with M3 disease at diagnosis), 5 received both radiotherapy and HDCT as part of their salvage regimen; 4 were aged <3 years at diagnosis and had received chemotherapy only as part of frontline therapy. Two patients died of transplant-related toxicities; 44% experienced grade 3 or 4 transplant-related toxicities (toxicities were graded according to the National Cancer Institute Common Toxicity Criteria). CONCLUSIONS. HDCT with ASCR is not an effective salvage strategy for older children with recurrent CNS malignancies. The significantly better outcome in the younger cohort was most likely related to the use of radiotherapy as part of the salvage strategy.

AB - BACKGROUND. High-dose chemotherapy (HDCT) with autologous stem cell rescue (ASCR) has been reported to be effective in treating children with recurrent central nervous system (CNS) malignancies. METHODS. To evaluate the efficacy and toxicities of HDCT and ASCR, the medical records of 27 children with recurrent CNS malignancies who received such therapy at St. Jude Children's Research Hospital between 1989 and 2004 were reviewed. RESULTS. The median age at diagnosis was 4.5 years (range, 0.4-16.6 years) and that at ASCR was 6.7 years (range, 1.1-18.5 years). Diagnoses included medulloblastoma (13 patients), primitive neuroectodermal tumor (3 patients), pineoblastoma (2 patients), atypical teratoid rhabdoid tumor (2 patients), ependymoma (3 patients), anaplastic astrocytoma (2 patients), and glioblastoma multiforme (2 patients). The 5-year overall and progression-free survival (PFS) rates were 28.2% and 18.5%, respectively. The 5-year PFS rate for patients aged <3 years at diagnosis (57.1%) was significantly better than older patients (5.0%) (P = .019). Among the 6 long-term survivors (5 with M0 disease and 1 with M3 disease at diagnosis), 5 received both radiotherapy and HDCT as part of their salvage regimen; 4 were aged <3 years at diagnosis and had received chemotherapy only as part of frontline therapy. Two patients died of transplant-related toxicities; 44% experienced grade 3 or 4 transplant-related toxicities (toxicities were graded according to the National Cancer Institute Common Toxicity Criteria). CONCLUSIONS. HDCT with ASCR is not an effective salvage strategy for older children with recurrent CNS malignancies. The significantly better outcome in the younger cohort was most likely related to the use of radiotherapy as part of the salvage strategy.

KW - Autologous stem cell transplantation

KW - Central nervous system neoplasms

KW - High-dose chemotherapy

KW - Recurrent

KW - Salvage therapy

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