1. Identification of prognostic factors in childhood T-cell acute lymphoblastic leukemia: Results from DFCI ALL Consortium Protocols 05-001 and 11-001
- Author
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Justine M. Kahn, Jean-Marie Leclerc, Melissa A. Burns, Barbara L. Asselin, Sarah K. Hunt, Marshall A. Schorin, Donna Neuberg, Lisa M. Gennarini, Jennifer J.G. Welch, Alejandro Gutierrez, Andrew E. Place, Suzanne J. Forrest, Caroline Laverdière, Kara M. Kelly, Lynda M. Vrooman, Uma H. Athale, Bruno Michon, Jane E. O'Brien, Lewis B. Silverman, Marian H. Harris, Stephen E. Sallan, Peter D. Cole, Luis A. Clavell, Kristen E. Stevenson, Maria Luisa Sulis, and Yana Pikman
- Subjects
Oncology ,Adult ,Male ,medicine.medical_specialty ,Neoplasm, Residual ,Adolescent ,medicine.disease_cause ,Precursor T-Cell Lymphoblastic Leukemia-Lymphoma ,Article ,03 medical and health sciences ,Young Adult ,0302 clinical medicine ,Internal medicine ,Antineoplastic Combined Chemotherapy Protocols ,Biomarkers, Tumor ,Medicine ,Humans ,Child ,Randomized Controlled Trials as Topic ,Retrospective Studies ,Mutation ,business.industry ,Remission Induction ,Cancer ,High-Throughput Nucleotide Sequencing ,Infant ,Hematology ,medicine.disease ,Prognosis ,Childhood T-Cell Acute Lymphoblastic Leukemia ,Minimal residual disease ,Phenotype ,Clinical trial ,Reverse transcription polymerase chain reaction ,Survival Rate ,Clinical Trials, Phase III as Topic ,030220 oncology & carcinogenesis ,Child, Preschool ,Pediatrics, Perinatology and Child Health ,Female ,business ,Risk classification ,030215 immunology ,Follow-Up Studies - Abstract
BACKGROUND/OBJECTIVES While outcomes for pediatric T-cell acute lymphoblastic leukemia (T-ALL) are favorable, there are few widely accepted prognostic factors, limiting the ability to risk stratify therapy. DESIGN/METHODS Dana-Farber Cancer Institute (DFCI) Protocols 05-001 and 11-001 enrolled pediatric patients with newly diagnosed B- or T-ALL from 2005 to 2011 and from 2012 to 2015, respectively. Protocol therapy was nearly identical for patients with T-ALL (N = 123), who were all initially assigned to the high-risk arm. End-induction minimal residual disease (MRD) was assessed by reverse transcription polymerase chain reaction (RT-PCR) or next-generation sequencing (NGS), but was not used to modify postinduction therapy. Early T-cell precursor (ETP) status was determined by flow cytometry. Cases with sufficient diagnostic DNA were retrospectively evaluated by targeted NGS of known genetic drivers of T-ALL, including Notch, PI3K, and Ras pathway genes. RESULTS The 5-year event-free survival (EFS) and overall survival (OS) for patients with T-ALL was 81% (95% CI, 73-87%) and 90% (95% CI, 83-94%), respectively. ETP phenotype was associated with failure to achieve complete remission, but not with inferior OS. Low end-induction MRD (
- Published
- 2020