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Datasets/TARGET-AML

Acute Myeloid Leukemia

MixedProject-curated
TARGETAvailable from GDC

2,492 children and young adults with acute myeloid leukaemia - the largest paediatric cancer genomics cohort in this corpus, with 3.5 years of median follow-up and therapeutic agents recorded for 35% of cases.

Can it answer your research question?

4 of 6 supported
  • Overall survival

    Supported

    Vital status and time are populated; median follow-up 3.5 years; 2,158 cases with follow-up; endpoint: overall survival.API

  • Progression or recurrence

    Not supported

    progression_or_recurrence is informative for no case in the harmonized records.API

  • Treatment response

    Supported

    treatment_outcome is populated for 85% of cases; criteria: complete response.API

  • Which drug was given

    Supported

    therapeutic_agents is recorded for 35% of cases.API

  • Stage-adjusted modelling

    Not supported

    ajcc_pathologic_stage is informative for no case in the harmonized records.API

  • Analysis by race or ethnicity

    Supported

    Ethnicity is informative for 83% of cases.API

Judged from the repository’s own field completeness (thresholds). “Not measured” means we have not probed that field for this record, not that the data lack it.

At a glance

Every percentage is the repository’s own record of what is filled in
CasesAPI
2,492
Measurement types
9
52,213 files, 96 TB
Follow-upAPI
3.5 years
for 2,158 of 2,492 cases; overall survival
Treatment responseAPI
Recorded
complete response

SubjectCALC

Myeloid

As filed by the repository

Myeloid LeukemiasNot ApplicableUnknownHematopoietic and reticuloendothelial systems

Who is in the cohortAPI

A usable value“Not reported” or “unknown”No value recorded
  • Sex1,136 male · 1,022 female · 31 unknown · 303 no value recorded
  • Race1,551 white · 251 black or african american · 97 asian · 44 3 other · 216 unknown · 30 not reported · 303 no value recorded
  • Ethnicity1,693 not hispanic or latino · 385 hispanic or latino · 81 unknown · 30 not reported · 303 no value recorded
  • Vital status1,416 alive · 742 dead · 30 not reported · 1 unknown · 303 no value recorded

Age at diagnosis

Median 10, middle half 4–15, range 0–30 years across 2,158 cases.

What was measured, and on how many

9 measurement types, none covering the whole cohort.

  • Bulk RNA sequencing2,281 of 2,492 · 92%
  • microRNA sequencing1,836 of 2,492 · 74%
  • Whole genome sequencing227 of 2,492 · 9%
  • Whole exome sequencing22 of 2,492 · <1%
  • Targeted DNA panel sequencing803 of 2,492 · 32%
  • SNP genotyping array (copy number, germline)190 of 2,492 · 8%
  • DNA methylation array319 of 2,492 · 13%
  • Clinical2,181 of 2,492 · 88%
  • Somatic Structural Variation30 of 2,492 · 1%
Platform, files and data levels
AssayPlatformFilesLevels
Bulk RNA sequencingAPIillumina29,043-
microRNA sequencingAPIillumina5,694-
Whole genome sequencingAPIillumina846-
Whole exome sequencingAPIillumina607-
Targeted DNA panel sequencingAPIillumina13,145-
SNP genotyping array (copy number, germline)APIillumina469-
DNA methylation arrayAPIillumina2,400-
ClinicalAPI-7-
Somatic Structural VariationAPI-116-
How complete all 29 clinical fields are - 10 informative for at least 80% of cases

A field counts as informative only when it holds a real value. “Not reported” blocks an analysis just as a missing field does. Fields marked 1:n can hold several records per case, so they show the share of cases with any record.

Informative - a usable valuePresent but uninformative - “not reported” or “unknown”Absent - no value recorded

Outcome

  • Vital statusAPI87%
  • Cause of deathAPI0%
  • Progression or recurrenceAPI0%
  • Last known disease statusAPI0%

Follow-up

  • Disease response at follow-upAPI0%
  • Progression at follow-upAPI0%
  • ECOG performance statusAPI0%

Treatment

  • Treatment type1:nAPI86%
  • Treatment given1:nAPI86%
  • Treatment outcome1:nAPI85%
  • Therapeutic agentsAPI35%
  • Prior treatmentAPI0%
  • Regimen or line of therapyAPI0%

Staging

  • AJCC pathologic stageAPI0%
  • AJCC clinical stageAPI0%

Diagnosis

  • Primary diagnosisAPI88%
  • Tissue or organ of originAPI88%
  • Prior malignancyAPI0%
  • Synchronous malignancyAPI0%

Pathology

  • Morphology (ICD-O)API88%
  • Classification of tumorAPI88%
  • Tumor gradeAPI0%

Demographics

  • Sex at birthAPI87%
  • EthnicityAPI83%
  • RaceAPI78%
  • Population groupAPI0%
  • Country of residenceAPI0%

Exposure

  • Tobacco smoking statusAPI0%
  • Alcohol historyAPI0%

Access

MixedSome components are open; others need approval.

Data Access Request through dbGaP using an eRA Commons account; institutional signing official approval required

Open components
10,479 open-access files
Controlled components
41,734 controlled-access files
Typical turnaround
days to a few weeks after signing official approval
License
NIH Genomic Data Sharing Policy terms
Citation requirement
Cite the dataset accession and the program's publication guidelines; see the GDC publication policy.

What it is good for

Written and checked by a reviewer, not generated from metadata.

  1. 1. How does the genomic landscape of paediatric AML differ from adult AML?

    Needs approved accessn ≈ 2,281HUMAN

    2,281 RNA-sequenced paediatric cases can be contrasted directly with adult AML cohorts, and the marker paper established that the differences are substantial and age-dependent. This remains the definitive paediatric comparison set.

    Statistical note: Adult comparison cohorts differ in platform and processing; model batch explicitly.

    NeedsBulk RNA sequencingmicroRNA

    A published example: PMID 29227476

  2. 2. Which molecular features predict survival in paediatric AML?

    Needs approved accessn ≈ 2,159HUMAN

    Vital status informative for 86.6% of 2,492 cases with median follow-up of 41.7 months gives a well-powered survival cohort, and therapeutic agents are recorded for 35% so treatment can be partially controlled.

    Statistical note: Cause of death is not recorded, so competing risks from treatment-related mortality cannot be separated from disease death.

    NeedsBulk RNA sequencingClinical dataOutcome datademographic.vital_statusdiagnoses.days_to_last_follow_up
  3. 3. Are there racial differences in paediatric AML biology or outcome?

    Needs approved accessn ≈ 1,802HUMAN

    251 Black or African American patients alongside 1,551 white patients is one of the better-powered settings in this corpus for a race-stratified comparison in a paediatric cancer.

    Statistical note: Race is self-reported; genetic ancestry from germline data is the more defensible variable for biological claims. Treatment differences may confound outcome comparisons.

    NeedsBulk RNA sequencingClinical datademographic.racedemographic.vital_status

What it cannot tell you

1 blocking

Written by a reviewer against the source, not inferred from what is missing.

BlockingMissing dataHUMAN

Progression or recurrence is populated for 0.3% of cases and informative for none, and cause of death is absent entirely. Relapse - the central clinical event in paediatric AML - cannot be modeled from these records.

Affects: Relapse-free survival; Competing-risk analysis; Relapse biology

Mitigation: Restrict to overall survival, or obtain relapse data from the trial group.

MajorTreatment contextHUMAN

Treatment type is populated for 86% of cases but therapeutic agents only for 35%, and treatment outcome detail is sparse. Protocol era spans many years with evolving therapy.

Affects: Treatment-stratified analysis; Agent-specific response

Mitigation: Adjust for enrolment era; treat agent-level claims cautiously.

MajorStatistical powerHUMAN

Deeper molecular layers cover far fewer cases than RNA sequencing - whole genome 227, exome 22, methylation 319 - so multi-omic analyses run on a small fraction of the cohort.

Affects: Multi-omic integration

Mitigation: Build the intersection explicitly; do not assume cohort-wide coverage.

MajorAccess constraintHUMAN

Most of the 52,213 files are controlled access.

Affects: Sequence-level analysis

Mitigation: Submit a dbGaP request early.

Not appropriate for

  • Relapse or event-free survival analysis. - Progression and recurrence fields are effectively empty.HUMAN
  • Assuming molecular coverage is uniform across the cohort. - Whole genome sequencing covers 227 of 2,492 cases; an analysis specified as "TARGET-AML whole genomes" is working with under a tenth of the cohort.HUMAN

Who has used it

Articles that analyzed these data, counted separately from articles that only cited the paper. Only the first counts as reuse.

Analyzed the dataCALC2
137
accession in the methods; 76% of a 29-article sample really cited it
Declared using it
25
named in a data availability statement
Cited the paper
695
attention to the publication, not reuse of the data
Against expectation
Well below
about 570 articles expected; index -2.05

What articles did with it

Only the last row counts as reuse. Where the accession appears in an article tells us what the authors did with the data.

  • Cited the paper695
  • Mentioned the dataset130
  • Accession found in text32
  • Declared using it25
  • Analyzed the data137

Against comparable datasets

137 articles analyzed these data. About 570 would be expected. The expectation comes from a model of size, age, breadth and access tier.

  • Analyzed it137
  • Expected~570
Well known, little used
The publication has 695 citations, about 5 for every article that analyzed the data. The finding travelled further than the resource did.
How this count was corrected
Europe PMC returned 181 articles for METHODS:"TARGET-AML", but it indexes the hyphen in TARGET-AML as a word break, so that query also matches the two words in ordinary prose. Of 29 sampled articles whose full text we could read, 22 contained the literal accession (58-88%, 95% interval). The published figure is the raw count scaled by that fraction.
Reuse assessment
  • 137 article(s) reference this dataset's accession in a methods section, fewer than the 569.6 predicted for comparable datasets (index -2.05). It is not flagged as underexplored because more than 25 articles have already analysed it, which is not the position a researcher looking for neglected data is looking for.

Compared against 132 datasets in this corpus with a citable accession and sufficient metadata. See Methods for the model.

Articles that analyzed the data

Examples, not the full list. 129 articles matching this dataset's accession search were retrieved and graded individually; these are the strongest of those.

Deep-review sample: author overlap and funding were checked only for the 10 articles listed here, not for every article examined. Of the 10 that analyzed the data, 3 of the 10 we could check had no author in common with the generating team, and 9 of the 10 we could check were themselves NCI-funded. These are sample counts, not population estimates.

Community examples

Share your example

Analyses someone ran on these data end to end, each with a receipt recording when it ran, against which packages and for how long.

Nobody has shared an analysis of this dataset yet. The examples on other datasets cover the common patterns and can be adapted - and if you adapt one, it belongs here.

Start here

Starter code generated from this record’s own identifiers, then the access steps.

Get the data in code

GDC: list open files and download thempython

Open-access files need no account. Controlled files need an approved dbGaP request and a token passed to gdc-client.

import json, requests

PROJECT = "TARGET-AML"
filters = {"op": "and", "content": [
    {"op": "in", "content": {"field": "cases.project.project_id", "value": [PROJECT]}},
    {"op": "in", "content": {"field": "access", "value": ["open"]}},
]}
params = {"filters": json.dumps(filters),
          "fields": "file_id,file_name,data_category,data_type,file_size",
          "size": "10000", "format": "JSON"}
files = requests.get("https://api.gdc.cancer.gov/files", params=params, timeout=60).json()["data"]["hits"]
print(len(files), "open files")

# Write a manifest and fetch with the GDC client (https://gdc.cancer.gov/access-data/gdc-data-transfer-tool)
with open("manifest.txt", "w") as fh:
    fh.write("id\n" + "\n".join(f["file_id"] for f in files))
# $ gdc-client download -m manifest.txt
GDC: clinical table for every casepython
import json, requests

filters = {"op": "in", "content": {"field": "project.project_id", "value": ["TARGET-AML"]}}
params = {"filters": json.dumps(filters), "size": "5000", "format": "TSV",
          "fields": ",".join([
              "submitter_id", "demographic.vital_status", "demographic.days_to_death",
              "diagnoses.days_to_last_follow_up", "diagnoses.ajcc_pathologic_stage",
              "diagnoses.treatments.treatment_type", "diagnoses.treatments.treatment_outcome"])}
tsv = requests.get("https://api.gdc.cancer.gov/cases", params=params, timeout=60).text
open("TARGET-AML_clinical.tsv", "w").write(tsv)
This page as data: limitations firstbash
curl -s https://cd2s.vercel.app/data/datasets/gdc-target-aml.json | python3 -c '
import json, sys
r = json.load(sys.stdin)
for l in r["limitations"]:
    print(l["severity"].upper(), "-", l["statement"][:120])
print("survival endpoint:", r["longitudinal"]["has_survival_endpoint"], "| treatment response:", r["longitudinal"]["has_treatment_response"])'

Access steps

  1. 1
    Scope with open derived expression filesFor people1 hour
    https://portal.gdc.cancer.gov/projects/TARGET-AML
  2. 2
    Check per-layer coverage before designing a multi-omic analysisFor people30 minutes

    The assay table on this page shows how sharply coverage drops beyond RNA sequencing.

  3. 3
    Submit a dbGaP data access requestFor peopledays to a few weeks

    Requires: eRA Commons account, institutional signing official approval

    https://gdc.cancer.gov/access-data/obtaining-access-controlled-data
  4. 4
    Query the project from code, with no credentialsFor agents

    The file index is public even where files are not, so an agent can size the cohort before anyone requests access.

    curl -s 'https://api.gdc.cancer.gov/files' --get \
      --data-urlencode 'filters={"op":"in","content":{"field":"cases.project.project_id","value":["TARGET-AML"]}}' \
      --data-urlencode 'fields=file_id,file_name,data_type' \
      --data-urlencode 'size=10000' --data-urlencode 'format=TSV'
    https://docs.gdc.cancer.gov/API/Users_Guide/Getting_Started/

    Generated from repository policy. Check the linked policy before downloading.

  5. 5
    Pass the token when the agent needs controlled filesFor agents

    Without a token, an API query may return only the open subset. Compare returned counts with the record before treating them as the whole cohort.

    Requires: GDC authentication token

    curl -H "X-Auth-Token: $GDC_TOKEN" https://api.gdc.cancer.gov/data/<file_id>
    https://docs.gdc.cancer.gov/API/Users_Guide/Getting_Started/

    Generated from repository policy. Check the linked policy before downloading.

Provenance

All of it, and more, is in this record’s JSON.

Identifiers

Versions and verification

Released
1 Jan 2009
Metadata retrieved
18 Sept 2026
Review status
Project-curatedCD2S project curation on 18 Sept 2026
Data generation funding
Funded data generation

Funding

Awards resolved through NIH RePORTER. Awards that paid to generate these data are listed apart from awards that paid to reuse them. Both are returns on NCI investment, but different ones.

Funded data generation

  • U10CA098543Children's Oncology Group Chair's GrantPETER C. ADAMSON, GREGORY H REAMANFY2003-2013API2
  • U24CA114766Support for Human Specimen Banking in NCI-Sponsored Clinical Trials-(CGB)Nilsa Del Carmen Ramirez Milan, GREGORY H REAMANFY2005-2014API2
  • U10CA180899Children's Oncology Group Statistics and Data CenterTODD A ALONZO, Meenakshi DevidasFY2014-2026API2
  • U10CA180886COG NCTN Network Group Operations CenterDouglas S. Hawkins, Richard AplencFY2014-2026API2
  • U10CA098413Children's Oncology Group Statistics and Data Center GrantJAMES R ANDERSON, Meenakshi DevidasFY2003-2013API2
  • R01CA114563Biology and Prognostic Implications of FLT3 Mutations in AMLSOHEIL MESHINCHIFY2005-2016API2

Funded reuse of these data

  • T32CA009351Pediatric Oncology Research Training ProgramElizabeth R Lawlor, SOHEIL MESHINCHIFY1985-2026API
  • R01CA204396ABERRANT SIGNALING IN ACUTE MYELOID LEUKEMIAAlex KentsisFY2016-2025API
  • K08CA256489Co-targeting BET Bromodomain Proteins and Aberrant Signaling in AML.Benjamin HuangFY2021-2025API
  • T32CA236748Childhood Hematological Malignancies Training ProgramCharles G MullighanFY2019-2026API
  • R01CA139158MiRNAs in hepatocellular carcinoma development and treatmentWENDONG HUANG, Hua E YuFY2011-2021API
  • R50CA211481MULTIMODAL PRECLINICAL CANCER IMAGING AND THERAPY RESEARCH SPECIALISTWalter John AkersFY2016-2020API

Funded infrastructure or harmonization

  • P30CA021765Cancer Biology (CBP)Douglas R. Green, Michael A DyerFY1985-2026API
  • P30CA008748Cancer Center AdministrationALEX MACDONALD, SELWYN M VICKERSFY1985-2026API
  • U54CA243124Experimental and preclinical modeling of NUP98-rearranged acute leukemiaCharles G Mullighan, RICHARD W KRIWACKIFY2019-2023API
  • P30CA016058Shared Resource 10: Pharmacoanalytical Shared Resource (PhASR)Mitch A Phelps, David M GoskyFY1985-2026API
  • P01CA096832CORE A - AdministrationSUZANNE J. BAKER, Brent Alan OrrFY2003-2025API
  • P30CA015704Prostate CancerPETER S NELSON, Marion Shonn DorerFY1985-2026API

For softwarerecord JSON