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Study Description

There are 200+ fully characterized patient cases with AML (all tumor/normal pairs, 100 with relapse sample as well) that make up the TARGET AML dataset, each with gene expression, tumor and paired normal copy number analyses, methylation and comprehensive next-generation sequencing to include whole genome sequencing, mRNA-seq and miRNA-seq. A subset of these cases will also have whole exome sequencing data available as well. There are additionally a large number of cases with partial molecular characterization making this a large and informative genomic dataset.

Additionally, the TARGET AML cohort includes some highly aggressive subtypes including AML "induction failures." Upon diagnosis, AML patients without high-risk genetic markers undergo standard chemotherapy, called the primary induction phase. This phase lasts about 30 days and is designed to eliminate cancer cells. Success of treatment is measured by the percentage of myeloblasts, i.e. immature white blood cells, detected in the patient following primary induction. Patients with greater than 15% myeloblasts did not sufficiently respond to standard therapy and are "induction failures." Currently, these patients have very poor prognoses and few clinical options. There are 30 patient cases that make up the TARGET AML Induction failure dataset, each with a primary tumor sample, a "normal" fibroblast sample grown from the patient's bone marrow, and a sample obtained at the end of induction. The trios for each case are characterized with next generation sequencing platforms to include whole genome, miRNA-Seq and mRNA-Seq. Gene expression and copy number analyses were also determined from mRNA-seq and whole genome sequencing respectively.

All cases can be sorted according to data type via the Sample Matrix on the TARGET Data Matrix (https://ocg.cancer.gov/programs/target/data-matrix). Please visit the TARGET website listed above for additional information on this and other TARGET genomics projects. Please see the TARGET Publication Guidelines at the OCG website (http://ocg.cancer.gov/programs/target/target-publication-guidelines) for updated details on sharing of any TARGET substudy data.

  • Study Weblink: TARGET AML
  • Study Type: Cohort
  • Number of study subjects that have individual level data available through Authorized Access: 2095

Authorized Access
Publicly Available Data (Public ftp)

Note: Access to publicly available data is available on the public ftp site for study phs000218.v21.p7.

Study Inclusion/Exclusion Criteria

AML patient samples were obtained from the Children's Oncology Group (most from study AAML0531) and chosen for inclusion in the TARGET project based on the following criteria: bone marrow and peripheral blood blast counts of > 50% and availability of DNA and RNA for comprehensive molecular characterization of the genome, transcriptome and epigenome. The majority of cases in the project had 3 or fewer cytogenetic abnormalities and the patients achieved a remission following the standard two rounds of induction therapy, which allowed the post-induction "normal" tissue to be used as tumor comparator. However, there does exist subsets of cases within the AML dataset that express a greater number of cytogenetic abnormalities and/or failed induction.

Study History

Pediatric ALL and neuroblastoma were the first diseases to be piloted for the TARGET initiative, and ARRA funding allowed for its expansion to include pediatric AML, as well as osteosarcoma and kidney cancers. TARGET is jointly managed by the NCI Office of Cancer Genomics (OCG) and Cancer Therapy Evaluation Program (CTEP). The AML study is run as a cooperative collaboration between investigators and management staff at NCI, Children's Oncology Group, Fred Hutchinson Cancer Research Center and the Johns Hopkins University.

May, 2005 NCI and the American Cancer Society co-sponsor "Childhood Cancer Targeted Therapeutics Workshop" that ultimately served as a basis for the conception of TARGET.
October, 2006 TARGET project officially initiated.
January, 2009 First publication of characterization and analysis of 221 pediatric ALL patients from the pilot phase of TARGET.
September, 2009 ARRA funding allows for expansion of TARGET pilot project to include additional cancers of study and next generation sequencing.

Selected publications
Diseases/Traits Related to Study (MESH terms)
Links to Related Resources
Authorized Data Access Requests
Study Attribution