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Efficacy of High-Dose Chemotherapy and Three-Dimensional Conformal Radiation for Atypical Teratoid/Rhabdoid Tumor: A Report From the Children's Oncology Group Trial ACNS0333

Reddy, Alyssa T ; Strother, Douglas R ; Judkins, Alexander R ; Burger, Peter C ; Pollack, Ian F ; Krailo, Mark D ; Buxton, Allen B ; Williams-Hughes, Chris ; Fouladi, Maryam ; Mahajan, Anita ; Merchant, Thomas E ; Ho, Ben ; Mazewski, Claire M ; Lewis, Victor A ; Gajjar, Amar ; Vezina, Louis-Gilbert ; Booth, Timothy N ; Parsons, Kerry W ; Poss, Vicky L ; Zhou, Tianni ; Biegel, Jaclyn A ; Huang, Annie

Journal of clinical oncology, 2020-04, Vol.38 (11), p.1175-1185 [Periódico revisado por pares]

United States: American Society of Clinical Oncology

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Título:
Efficacy of High-Dose Chemotherapy and Three-Dimensional Conformal Radiation for Atypical Teratoid/Rhabdoid Tumor: A Report From the Children's Oncology Group Trial ACNS0333
Autor: Reddy, Alyssa T ; Strother, Douglas R ; Judkins, Alexander R ; Burger, Peter C ; Pollack, Ian F ; Krailo, Mark D ; Buxton, Allen B ; Williams-Hughes, Chris ; Fouladi, Maryam ; Mahajan, Anita ; Merchant, Thomas E ; Ho, Ben ; Mazewski, Claire M ; Lewis, Victor A ; Gajjar, Amar ; Vezina, Louis-Gilbert ; Booth, Timothy N ; Parsons, Kerry W ; Poss, Vicky L ; Zhou, Tianni ; Biegel, Jaclyn A ; Huang, Annie
Assuntos: Adolescent ; Antineoplastic Combined Chemotherapy Protocols - adverse effects ; Antineoplastic Combined Chemotherapy Protocols - therapeutic use ; Child ; Child, Preschool ; Combined Modality Therapy ; Female ; Humans ; Infant ; Infant, Newborn ; Male ; Original Reports ; Prognosis ; Radiotherapy, Conformal - methods ; Rhabdoid Tumor - genetics ; Rhabdoid Tumor - mortality ; Rhabdoid Tumor - therapy ; SMARCB1 Protein - genetics ; Teratoma - genetics ; Teratoma - mortality ; Teratoma - therapy ; Young Adult
É parte de: Journal of clinical oncology, 2020-04, Vol.38 (11), p.1175-1185
Notas: J.A.B. and A.H. are co-senior authors.
Descrição: Atypical teratoid/rhabdoid tumor (AT/RT) is an aggressive, early-childhood brain tumor without standard effective treatment. To our knowledge, we conducted the first AT/RT-specific cooperative group trial, ACNS0333, to examine the efficacy and safety of intensive postoperative chemotherapy and focal radiation to treat AT/RT. Patients from birth to 22 years of age with AT/RT were eligible. After surgery, they received 2 courses of multiagent chemotherapy, followed by 3 courses of high-dose chemotherapy with peripheral blood stem cell rescue and involved-field radiation therapy. Timing of radiation was based on patient age and disease location and extent. Central testing of tumor and blood for status was mandated. Tumor molecular subclassification was performed retrospectively. The primary analysis was event-free survival (EFS) for patients < 36 months of age compared with a cooperative groups' historical cohort. Although accrual was based on the therapeutic question, potential prognostic factors, including age, tumor location, M stage, surgical resection, order of therapy, germline status, and molecular subtype, were explored. Of 65 evaluable patients, 54 were < 36 months of age. ACNS0333 therapy significantly reduced the risk of EFS events in patients < 36 months of age compared with the historical cohort ( < .0005; hazard rate, 0.43; 95% CI, 0.28 to 0.66). Four-year EFS and overall survival for the entire cohort were 37% (95% CI, 25% to 49%) and 43% (95% CI, 31% to 55%), respectively. Timing of radiation did not affect survival, and 91% of relapses occurred by 2 years from enrollment. Treatment-related deaths occurred in 4 patients. The ACNS0333 regimen dramatically improved survival compared with historical therapies for patients with AT/RT. Clinical characteristics and molecular subgrouping suggest prognostic differences. ACNS0333 results lay a foundation on which to build future studies and incorporate testing of new therapeutic agents.
Editor: United States: American Society of Clinical Oncology
Idioma: Inglês

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Efficacy of High-Dose Chemotherapy and Three-Dimensional Conformal Radiation for Atypical Teratoid/Rhabdoid Tumor: A Report From the Children's Oncology Group Trial ACNS0333

United States: American Society of Clinical Oncology

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