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Program Description

The TARGET Acute Myeloid Leukemia projects employed comprehensive molecular characterization to determine the genetic changes that drive the initiation and progression of high-risk or hard-to-treat childhood cancers. Acute myeloid leukemia (AML) is a cancer that originates in the bone marrow from immature white blood cells known as myeloblasts. About 25% of all children with leukemia have AML. Although survival rates have increased since the 1970s, approximately half of all childhood AML cases relapse despite intensive treatment. Additional therapies following relapse are often unsuccessful and can be especially difficult and damaging for children. These patients would clearly benefit from targeted therapeutic approaches.

Through comprehensive genome-wide characterization, TARGET researchers are identifying the genetic and epigenetic alterations of relapsed disease. The ultimate goal is to translate their discoveries into novel treatments that will improve outcomes for children with AML. To learn more about pediatric AML and current treatment strategies, visit the NCI pediatric AML website.

TARGET AML molecular characterization analyses include gene expression array, copy number array, DNA methylation, Whole Genome Sequencing, Whole Exome Sequencing, RNA-seq, miRNA-seq and Targeted Capture Sequencing.

Publications

• McNeer NA, Philip J, Geiger H, et al. (2019) Genetic mechanisms of primary chemotherapy resistance in pediatric acute myeloid leukemia . Leukemia 33(8):1934-1943. (PMID: 30760869) View PubMed abstract View Publication Information and Supplementary Files
• Bolouri H, Farrar JE, Triche T Jr, et al. (2018) The molecular landscape of pediatric acute myeloid leukemia reveals recurrent structural alterations and age-specific mutational interactions. Nature Medicine 24(1):103-112. (PMID: 29227476) View PubMed abstract View Publication Information and Supplementary Files
• Lim EL, Trinh DL, Ries RE, et al. (2017) MicroRNA Expression-Based Model Indicates Event-Free Survival in Pediatric Acute Myeloid Leukemia. Journal of Clinical Oncology 35(35):3964-3977. (PMID: 29068783) View PubMed abstract View Publication Information and Supplementary Files
• Farrar JE, Schuback HL, Ries RE, et al. (2016) Genomic Profiling of Pediatric Acute Myeloid Leukemia Reveals a Changing Mutational Landscape from Disease Diagnosis to Relapse. Cancer Research 76(8):2197-205. (PMID: 26941285) View PubMed abstract View Publication Information and Supplementary Files
• Maxson JE, Ries RE, Wang YC, et al. (2016) CSF3R mutations have a high degree of overlap with CEBPA mutations in pediatric AML. Blood 127(24):3094-8. (PMID: 27143256) View PubMed abstract View Publication Information and Supplementary Files