Atypical teratoid/rhabdoid tumors (ATRT)

Improved survival in children 3 years of age and older with radiation therapy and high-dose alkylator-based chemotherapy

Tanya M. Tekautz, Christine E. Fuller, Susan Blaney, Maryam Fouladi, Alberto Broniscer, Thomas E. Merchant, Matthew Krasin, James Dalton, Gregory Hale, Larry E. Kun, Dana Wallace, Richard J. Gilbertson, Amar Gajjar

Research output: Contribution to journalReview article

240 Citations (Scopus)

Abstract

Purpose: To describe clinical features, therapeutic approaches, and prognostic factors in pediatric patients with atypical teratoid/rhabdoid tumors (ATRT) treated at St Jude Children's Research Hospital (SJCRH). Patients and Methods: Primary tumor samples from patients diagnosed with ATRT at SJCRH between July 1984 and June 2003 were identified. Pathology review included histologic, immunohistochemical analysis, and fluorescence in situ hybridization for SMARCB1 (also known as hSNF5/INI1) deletion. Clinical records of patients with pathologic confirmation of ATRT were reviewed. Results: Thirty-seven patients were diagnosed with ATRT at SJCRH during the 19-year study interval. Six patients were excluded from this clinical review based on pathologic or clinical criteria. Of the remaining 31 patients, 22 were younger than 3 years. Posterior fossa primary lesions and metastatic disease at diagnosis were more common in younger patients with ATRT. All patients underwent surgical resection; 30 received subsequent chemotherapy. The majority of patients aged 3 years or older received postoperative craniospinal radiation. Two-year event-free (EFS) and overall survival (OS) of children aged 3 years or older (EFS, 78% + 14%; OS, 89% ± 11%) were significantly better than those for younger patients (EFS, 11% ± 6%; OS, 17% ± 8%); EFS, P = .009 and OS, P = .0001. No other clinical characteristics were predictive of survival. Three of four patients 3 years or older with progressive disease were successfully rescued with ifosfamide, carboplatin, and etoposide therapy. Conclusion: Children presenting with ATRT before the age of 3 years have a dismal prognosis. ATRT presenting in older patients can be cured using a combination of radiation and high-dose alkylating therapy. Older patients with relapsed ATRT can have salvage treatment using ICE chemotherapy.

Original languageEnglish (US)
Pages (from-to)1491-1499
Number of pages9
JournalJournal of Clinical Oncology
Volume23
Issue number7
DOIs
StatePublished - Dec 1 2005

Fingerprint

Alkylating Agents
Radiotherapy
Drug Therapy
Survival
Atypical Teratoid Tumor
Typical Teratoid Rhabdoid Tumor
Research
Radiation
Salvage Therapy
Ifosfamide
Carboplatin
Etoposide
Fluorescence In Situ Hybridization

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

Atypical teratoid/rhabdoid tumors (ATRT) : Improved survival in children 3 years of age and older with radiation therapy and high-dose alkylator-based chemotherapy. / Tekautz, Tanya M.; Fuller, Christine E.; Blaney, Susan; Fouladi, Maryam; Broniscer, Alberto; Merchant, Thomas E.; Krasin, Matthew; Dalton, James; Hale, Gregory; Kun, Larry E.; Wallace, Dana; Gilbertson, Richard J.; Gajjar, Amar.

In: Journal of Clinical Oncology, Vol. 23, No. 7, 01.12.2005, p. 1491-1499.

Research output: Contribution to journalReview article

Tekautz, TM, Fuller, CE, Blaney, S, Fouladi, M, Broniscer, A, Merchant, TE, Krasin, M, Dalton, J, Hale, G, Kun, LE, Wallace, D, Gilbertson, RJ & Gajjar, A 2005, 'Atypical teratoid/rhabdoid tumors (ATRT): Improved survival in children 3 years of age and older with radiation therapy and high-dose alkylator-based chemotherapy', Journal of Clinical Oncology, vol. 23, no. 7, pp. 1491-1499. https://doi.org/10.1200/JCO.2005.05.187
Tekautz, Tanya M. ; Fuller, Christine E. ; Blaney, Susan ; Fouladi, Maryam ; Broniscer, Alberto ; Merchant, Thomas E. ; Krasin, Matthew ; Dalton, James ; Hale, Gregory ; Kun, Larry E. ; Wallace, Dana ; Gilbertson, Richard J. ; Gajjar, Amar. / Atypical teratoid/rhabdoid tumors (ATRT) : Improved survival in children 3 years of age and older with radiation therapy and high-dose alkylator-based chemotherapy. In: Journal of Clinical Oncology. 2005 ; Vol. 23, No. 7. pp. 1491-1499.
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abstract = "Purpose: To describe clinical features, therapeutic approaches, and prognostic factors in pediatric patients with atypical teratoid/rhabdoid tumors (ATRT) treated at St Jude Children's Research Hospital (SJCRH). Patients and Methods: Primary tumor samples from patients diagnosed with ATRT at SJCRH between July 1984 and June 2003 were identified. Pathology review included histologic, immunohistochemical analysis, and fluorescence in situ hybridization for SMARCB1 (also known as hSNF5/INI1) deletion. Clinical records of patients with pathologic confirmation of ATRT were reviewed. Results: Thirty-seven patients were diagnosed with ATRT at SJCRH during the 19-year study interval. Six patients were excluded from this clinical review based on pathologic or clinical criteria. Of the remaining 31 patients, 22 were younger than 3 years. Posterior fossa primary lesions and metastatic disease at diagnosis were more common in younger patients with ATRT. All patients underwent surgical resection; 30 received subsequent chemotherapy. The majority of patients aged 3 years or older received postoperative craniospinal radiation. Two-year event-free (EFS) and overall survival (OS) of children aged 3 years or older (EFS, 78{\%} + 14{\%}; OS, 89{\%} ± 11{\%}) were significantly better than those for younger patients (EFS, 11{\%} ± 6{\%}; OS, 17{\%} ± 8{\%}); EFS, P = .009 and OS, P = .0001. No other clinical characteristics were predictive of survival. Three of four patients 3 years or older with progressive disease were successfully rescued with ifosfamide, carboplatin, and etoposide therapy. Conclusion: Children presenting with ATRT before the age of 3 years have a dismal prognosis. ATRT presenting in older patients can be cured using a combination of radiation and high-dose alkylating therapy. Older patients with relapsed ATRT can have salvage treatment using ICE chemotherapy.",
author = "Tekautz, {Tanya M.} and Fuller, {Christine E.} and Susan Blaney and Maryam Fouladi and Alberto Broniscer and Merchant, {Thomas E.} and Matthew Krasin and James Dalton and Gregory Hale and Kun, {Larry E.} and Dana Wallace and Gilbertson, {Richard J.} and Amar Gajjar",
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T1 - Atypical teratoid/rhabdoid tumors (ATRT)

T2 - Improved survival in children 3 years of age and older with radiation therapy and high-dose alkylator-based chemotherapy

AU - Tekautz, Tanya M.

AU - Fuller, Christine E.

AU - Blaney, Susan

AU - Fouladi, Maryam

AU - Broniscer, Alberto

AU - Merchant, Thomas E.

AU - Krasin, Matthew

AU - Dalton, James

AU - Hale, Gregory

AU - Kun, Larry E.

AU - Wallace, Dana

AU - Gilbertson, Richard J.

AU - Gajjar, Amar

PY - 2005/12/1

Y1 - 2005/12/1

N2 - Purpose: To describe clinical features, therapeutic approaches, and prognostic factors in pediatric patients with atypical teratoid/rhabdoid tumors (ATRT) treated at St Jude Children's Research Hospital (SJCRH). Patients and Methods: Primary tumor samples from patients diagnosed with ATRT at SJCRH between July 1984 and June 2003 were identified. Pathology review included histologic, immunohistochemical analysis, and fluorescence in situ hybridization for SMARCB1 (also known as hSNF5/INI1) deletion. Clinical records of patients with pathologic confirmation of ATRT were reviewed. Results: Thirty-seven patients were diagnosed with ATRT at SJCRH during the 19-year study interval. Six patients were excluded from this clinical review based on pathologic or clinical criteria. Of the remaining 31 patients, 22 were younger than 3 years. Posterior fossa primary lesions and metastatic disease at diagnosis were more common in younger patients with ATRT. All patients underwent surgical resection; 30 received subsequent chemotherapy. The majority of patients aged 3 years or older received postoperative craniospinal radiation. Two-year event-free (EFS) and overall survival (OS) of children aged 3 years or older (EFS, 78% + 14%; OS, 89% ± 11%) were significantly better than those for younger patients (EFS, 11% ± 6%; OS, 17% ± 8%); EFS, P = .009 and OS, P = .0001. No other clinical characteristics were predictive of survival. Three of four patients 3 years or older with progressive disease were successfully rescued with ifosfamide, carboplatin, and etoposide therapy. Conclusion: Children presenting with ATRT before the age of 3 years have a dismal prognosis. ATRT presenting in older patients can be cured using a combination of radiation and high-dose alkylating therapy. Older patients with relapsed ATRT can have salvage treatment using ICE chemotherapy.

AB - Purpose: To describe clinical features, therapeutic approaches, and prognostic factors in pediatric patients with atypical teratoid/rhabdoid tumors (ATRT) treated at St Jude Children's Research Hospital (SJCRH). Patients and Methods: Primary tumor samples from patients diagnosed with ATRT at SJCRH between July 1984 and June 2003 were identified. Pathology review included histologic, immunohistochemical analysis, and fluorescence in situ hybridization for SMARCB1 (also known as hSNF5/INI1) deletion. Clinical records of patients with pathologic confirmation of ATRT were reviewed. Results: Thirty-seven patients were diagnosed with ATRT at SJCRH during the 19-year study interval. Six patients were excluded from this clinical review based on pathologic or clinical criteria. Of the remaining 31 patients, 22 were younger than 3 years. Posterior fossa primary lesions and metastatic disease at diagnosis were more common in younger patients with ATRT. All patients underwent surgical resection; 30 received subsequent chemotherapy. The majority of patients aged 3 years or older received postoperative craniospinal radiation. Two-year event-free (EFS) and overall survival (OS) of children aged 3 years or older (EFS, 78% + 14%; OS, 89% ± 11%) were significantly better than those for younger patients (EFS, 11% ± 6%; OS, 17% ± 8%); EFS, P = .009 and OS, P = .0001. No other clinical characteristics were predictive of survival. Three of four patients 3 years or older with progressive disease were successfully rescued with ifosfamide, carboplatin, and etoposide therapy. Conclusion: Children presenting with ATRT before the age of 3 years have a dismal prognosis. ATRT presenting in older patients can be cured using a combination of radiation and high-dose alkylating therapy. Older patients with relapsed ATRT can have salvage treatment using ICE chemotherapy.

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