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Disease ยท MONDO_0005059

leukemia

6 sourcesdiseases-2026-09-18-0.1.0

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ConductScience Library. leukemia. Diseases, record MONDO_0005059. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0005059

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name
leukemia
Open Targets
Open Targets ID
MONDO_0005059
Open Targets
Other names
blood (leukemia), leukaemia (disease), leukemia (disease), leukemia, malignant, leukemias, leukemias, general
Open Targets
Disease Ontology
DOID:1240
Disease Ontology
MeSH
D007938
MeSH
NCI Thesaurus
C3161
NCI Thesaurus
Highest stage of its drugs
Approval, for 13 of its 182 drugs, from DailyMed and FDA
Open Targets

Drugs182

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

DrugChEMBL IDTypeOpen Targets' stageStage reported byReportsOf them trialsTrials shown hereSource
BUSULFANCHEMBL820Small moleculeApprovalDailyMed212020Drugs and targets
DEXAMETHASONECHEMBL384467Small moleculeApprovalDailyMed1699Drugs and targets
PREDNISONECHEMBL635Small moleculeApprovalDailyMed655Drugs and targets
RASBURICASECHEMBL1201594EnzymeApprovalDailyMed433Drugs and targets
ALLOPURINOLCHEMBL1467Small moleculeApprovalDailyMed5211Drugs and targets
PREDNISOLONECHEMBL131Small moleculeApprovalDailyMed211Drugs and targets
CORTISONE ACETATECHEMBL1650Small moleculeApprovalDailyMed200Drugs and targets
DEXAMETHASONE SODIUM PHOSPHATECHEMBL2021430Small moleculeApprovalDailyMed200Drugs and targets
HYDROCORTISONECHEMBL389621Small moleculeApprovalDailyMed100Drugs and targets
METHYLPREDNISOLONE ACETATECHEMBL1364144Small moleculeApprovalDailyMed100Drugs and targets

1 to 10 of 182

See all 182 drugs

Targets220

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

TargetProteinIts drugs that act on itMechanismsHighest stage of those drugsGene in GenesSource
NR3C1Glucocorticoid receptor10Glucocorticoid receptor agonistApprovalhttps://conductscience.org/library/genes/2908Drugs and targets
ABL1Bcr/Abl fusion protein8Bcr/Abl fusion protein inhibitor; Tyrosine-protein kinase ABL inhibitorPhase 2https://conductscience.org/library/genes/25Drugs and targets
TOP2ADNA topoisomerase 2-alpha8DNA topoisomerase II alpha inhibitor; DNA topoisomerase II inhibitorPhase 3https://conductscience.org/library/genes/7153Drugs and targets
BCRBcr/Abl fusion protein6Bcr/Abl fusion protein inhibitorPhase 2https://conductscience.org/library/genes/613Drugs and targets
KITMast/stem cell growth factor receptor Kit5Stem cell growth factor receptor inhibitorPhase 2https://conductscience.org/library/genes/3815Drugs and targets
PDGFRBPlatelet-derived growth factor receptor5Platelet-derived growth factor receptor beta inhibitor; Platelet-derived growth factor receptor inhibitorPhase 2https://conductscience.org/library/genes/5159Drugs and targets
POLA1DNA polymerase (alpha/delta/epsilon)5DNA polymerase (alpha/delta/epsilon) inhibitorPhase 3https://conductscience.org/library/genes/5422Drugs and targets
POLA2DNA polymerase (alpha/delta/epsilon)5DNA polymerase (alpha/delta/epsilon) inhibitorPhase 3https://conductscience.org/library/genes/23649Drugs and targets
POLD1DNA polymerase (alpha/delta/epsilon)5DNA polymerase (alpha/delta/epsilon) inhibitorPhase 3https://conductscience.org/library/genes/5424Drugs and targets
POLD2DNA polymerase (alpha/delta/epsilon)5DNA polymerase (alpha/delta/epsilon) inhibitorPhase 3https://conductscience.org/library/genes/5425Drugs and targets

1 to 10 of 220

See all 220 targets

Trials215

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 7 more are not shown, since the checks do not confirm them.

TrialTitlePhaseParticipantsResults first postedConditionsDrugsLinked bySource
NCT01190930Risk-Adapted Chemotherapy in Treating Younger Patients With Newly Diagnosed Standard-Risk Acute Lymphoblastic Leukemia or Localized B-Lineage Lymphoblastic LymphomaPhase 39,3502020-06-23
Acute Lymphoblastic Leukemia, Adult B Lymphoblastic Lymphoma, Ann Arbor Stage I B Lymphoblastic Lymphoma, Ann Arbor Stage II B Lymphoblastic Lymphoma, Childhood B Acute Lymphoblastic Leukemia, Childhood B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1, Childhood B Lymphoblastic Lymphoma, Down Syndrome, Hypodiploid B Acute Lymphoblastic Leukemia, Philadelphia Chromosome Positive
CYCLOPHOSPHAMIDE, CYTARABINE, DEXAMETHASONE, DOXORUBICIN HYDROCHLORIDE, LEUCOVORIN CALCIUM, MERCAPTOPURINE, METHOTREXATE, PEGASPARGASE, THIOGUANINE, VINCRISTINE SULFATEOpen Targets' reading, confirmed by NLM's indexing of its papersClinicalTrials.gov
NCT01522976Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic LeukemiaPhase 22822016-03-22
Chronic Myelomonocytic Leukemia, Chronic Myelomonocytic Leukemia-1, Chronic Myelomonocytic Leukemia-2, Myelodysplastic Syndrome, Myelodysplastic Syndrome With Excess Blasts, Myelodysplastic Syndrome With Excess Blasts-1, Myelodysplastic Syndrome/Acute Myeloid Leukemia
AZACITIDINE, LENALIDOMIDE, VORINOSTATOpen Targets' reading, confirmed by NLM's indexing of its papersClinicalTrials.gov
NCT00230178Rasburicase Versus Allopurinol in Tumor Patients at Risk for Hyperuricemia and Tumor Lysis SyndromePhase 32802009-12-31Tumor Lysis Syndrome, Cancer, HyperuricemiaALLOPURINOL, RASBURICASEIts record names the diseaseClinicalTrials.gov
NCT01572662Allogeneic Transplantation Using Timed Sequential Busulfan and Fludarabine ConditioningPhase 22012023-06-29
Leukemia, Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Chronic Myeloid Leukemia, Chronic Lymphocytic Leukemia, Myeloproliferative Diseases, Non-Hodgkins Lymphoma, Hodgkins Lymphoma, Multiple Myeloma, Myelodysplastic Syndrome
BUSULFAN, FLUDARABINEIts record names the diseaseClinicalTrials.gov
NCT01376310GSK1120212 Rollover StudyPhase 21592019-02-19CancerCARBOPLATIN, DOCETAXEL, ERLOTINIB, EVEROLIMUS, GEMCITABINE, PEMETREXED, TRAMETINIBIts record names the diseaseClinicalTrials.gov
NCT00658814Azacitidine and Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid LeukemiaPhase 21332014-01-06
Acute Myeloid Leukemia, Adult Acute Megakaryoblastic Leukemia, Adult Acute Monoblastic Leukemia, Adult Acute Monocytic Leukemia, Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11, Adult Acute Myeloid Leukemia With Maturation, Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11, Adult Acute Myeloid Leukemia With t(8;21)(q22;q22.1); RUNX1-RUNX1T1, Adult Acute Myeloid Leukemia With t(9;11)(p21.3;q23.3); MLLT3-KMT2A, Adult Acute Myeloid Leukemia Without Maturation, Adult Acute Myelomonocytic Leukemia, Adult Erythroleukemia, Adult Pure Erythroid Leukemia, Secondary Acute Myeloid Leukemia
AZACITIDINE, GEMTUZUMAB OZOGAMICINOpen Targets' reading, confirmed by NLM's indexing of its papersClinicalTrials.gov
NCT00390793Combination Chemotherapy and Dasatinib in Treating Participants With Philadelphia Positive or BCR-ABL Positive Acute Lymphoblastic Leukemia.Phase 21072025-02-17
Acute Lymphoblastic Leukemia, BCR-ABL1 Fusion Protein Expression, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Philadelphia Chromosome Positive, Recurrent Acute Lymphoblastic Leukemia, t(9;22)
CYCLOPHOSPHAMIDE, CYTARABINE, DASATINIB ANHYDROUS, DEXAMETHASONE, DOXORUBICIN, METHOTREXATE, PREDNISONE, VINCRISTINEOpen Targets' reading, confirmed by NLM's indexing of its papersClinicalTrials.gov
NCT00357500Etoposide, Cyclophosphamide, Thalidomide, Celecoxib, and Fenofibrate in Relapsed or Progressive CancerPhase 21012014-09-09Central Nervous System Tumor, Pediatric, Leukemia, Lymphoma, Neuroblastoma, Sarcoma, Unspecified Childhood Solid Tumor, Protocol SpecificCELECOXIB, CYCLOPHOSPHAMIDE, ETOPOSIDE, FENOFIBRATE, THALIDOMIDEIts record names the diseaseClinicalTrials.gov
NCT00792948Combination Chemotherapy With or Without Donor Stem Cell Transplant in Treating Patients With Acute Lymphoblastic LeukemiaPhase 2972017-04-26
Acute Lymphoblastic Leukemia, Adult B Acute Lymphoblastic Leukemia, Adult B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1, Adult L1 Acute Lymphoblastic Leukemia, Adult L2 Acute Lymphoblastic Leukemia, Adult T Acute Lymphoblastic Leukemia, Recurrent Adult Acute Lymphoblastic Leukemia
CYCLOPHOSPHAMIDE, CYTARABINE, DASATINIB ANHYDROUS, DEXAMETHASONE, DOXORUBICIN HYDROCHLORIDE, ETOPOSIDE, FILGRASTIM, LEUCOVORIN CALCIUM, METHOTREXATE, METHYLPREDNISOLONE, PREDNISONE, SIROLIMUS, TACROLIMUS ANHYDROUS, VINCRISTINE SULFATE
Open Targets' reading, confirmed by NLM's indexing of its papersClinicalTrials.gov
NCT00943592Clofarabine-Melphalan-Alemtuzumab Conditioning in Patients With Advanced Hematologic MalignanciesPhase 1/2822014-02-27Advanced Hematologic Malignancies, Leukemia, PreleukemiaALEMTUZUMAB, CLOFARABINE, MELPHALANIts record names the diseaseClinicalTrials.gov

1 to 10 of 215

See all 215 trials

Comparisons in its trials37

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

ComparisonGroup 1, with the outcomeGroup 2, with the outcomeParticipantsTrialSource
Arm 1: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 16,000 Umol/l vs Arm 2: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 20,000umol/l.: Overall Survival
16 of 4947 of 150199NCT01572662Trial comparisons
Arm 1: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 16,000 Umol/l vs Arm 2: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 20,000umol/l.: Non-Relapse Mortality Rate (NRM)
2 of 498 of 150199NCT01572662Trial comparisons
Arm 1: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 16,000 Umol/l vs Arm 2: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 20,000umol/l.: Overall Survival
29 of 4993 of 150199NCT01572662Trial comparisons
Arm 1: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 16,000 Umol/l vs Arm 2: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 20,000umol/l.: deaths from any cause
31 of 4993 of 150199NCT01572662Trial comparisons
Arm 1: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 16,000 Umol/l vs Arm 2: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 20,000umol/l.: serious adverse events
6 of 498 of 150199NCT01572662Trial comparisons
Cohort A (GSK1120212 < 24 Weeks) vs Cohort B (GSK1120212 >= 24 Weeks): deaths from any cause13 of 1263 of 33159NCT01376310Trial comparisons
Cohort A (GSK1120212 < 24 Weeks) vs Cohort B (GSK1120212 >= 24 Weeks): serious adverse events26 of 12613 of 33159NCT01376310Trial comparisons
G-CSF (Granulocyte Colony- Stimulating Factor) vs G-CSF and GM-CSF (Granulocyte Macrophage): serious adverse events7 of 257 of 2550NCT00208949Trial comparisons
Arm I: Glutamine vs Arm II: Placebo: Incidence of Vincristine-induced Peripheral Neuropathy24 of 2425 of 2549NCT00365768Trial comparisons
Arm I: Glutamine vs Arm II: Placebo: Number of Participants With Progression of Neuropathy11 of 2419 of 2549NCT00365768Trial comparisons

Papers from its trials101

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMIDListed asTrials listing itSource
33150834RESULT1PubMed
28396162RESULT1PubMed
27507877RESULT1PubMed
26899273RESULT1PubMed
25046834RESULT1PubMed
24862638RESULT2PubMed
23893953RESULT1PubMed
21910214RESULT1PubMed
21527516RESULT2PubMed
20860038RESULT1PubMed

Data dates

The date of each source this release read.

Open Targets
2026-09-18
Open Targets
Drug links and their checks, from Drugs and targets
2026-09-18
Drugs and targets
ClinicalTrials.gov, from Trials
2026-11-12
Trials
Comparisons, from Trial comparisons
2026-03-23
Trial comparisons
PubMed, for the checks
2026-11-06
PubMed
MeSH, for the checks
2026-11-10
MeSH

Sources 6

  • ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
  • ChEMBL, through Open TargetsCC BY-SA 3.0
  • ClinicalTrials.govNo license stated
  • NLM's MeSHNLM's terms
  • Open TargetsCC0 1.0
  • PubMedNLM's terms
Each source's terms
  • ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
  • ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
  • ClinicalTrials.govnone stated
  • NLM's MeSHNLM's
  • Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
  • PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0005059

GET https://conductscience.org/api/diseases/v1/MONDO_0005059/drugs?page=2