Disease · MONDO_0004643

myeloid leukemia

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. myeloid leukemia. Diseases, record MONDO_0004643. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0004643

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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: myeloid leukemia; Open Targets
Open Targets ID: MONDO_0004643; Open Targets
Other names: leukaemia granulocytic, leukaemia myeloid, leukemia granulocytic, leukemia myelogenous, leukemia myeloid, leukemia, granulocytic, malignant, myelocytic leukaemia, myelocytic leukemia, myelogenous leukaemia, myelogenous leukemia, non-lymphoblastic leukaemia, non-lymphoblastic leukemia, non-lymphocytic leukaemia, non-lymphocytic leukemia; Open Targets
Disease Ontology: DOID:8692; Disease Ontology
MeSH: D007951; MeSH
NCI Thesaurus: C3172; NCI Thesaurus
Highest stage of its drugs: Approval, for 7 of its 47 drugs, from DailyMed and EMA Human Drugs; Open Targets

Drugs47

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.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
DECITABINE	CHEMBL1201129	Small molecule	Approval	EMA Human Drugs	11	9	9	Drugs and targets
CEDAZURIDINE	CHEMBL3237547	Small molecule	Approval	EMA Human Drugs	4	3	3	Drugs and targets
QUIZARTINIB	CHEMBL576982	Small molecule	Approval	EMA Human Drugs	4	3	3	Drugs and targets
GEMTUZUMAB OZOGAMICIN	CHEMBL1201506	Antibody drug conjugate	Approval	DailyMed	3	2	2	Drugs and targets
PONATINIB	CHEMBL1171837	Small molecule	Approval	EMA Human Drugs	2	1	1	Drugs and targets
BOSUTINIB	CHEMBL288441	Small molecule	Approval	EMA Human Drugs	1	0	0	Drugs and targets
GILTERITINIB FUMARATE	CHEMBL3301603	Small molecule	Approval	DailyMed	1	0	0	Drugs and targets
LESTAURTINIB	CHEMBL603469	Small molecule	Phase 3	TTD	1	0	0	Drugs and targets
VENETOCLAX	CHEMBL3137309	Small molecule	Phase 2	ClinicalTrials.gov	17	17	17	Drugs and targets
CYTARABINE	CHEMBL803	Small molecule	Phase 2	ClinicalTrials.gov	11	11	11	Drugs and targets

1 to 10 of 47

See all 47 drugs

Targets114

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.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
FLT3	Receptor-type tyrosine-protein kinase FLT3	6	Tyrosine-protein kinase receptor FLT3 inhibitor	Approval	https://conductscience.org/library/genes/2322	Drugs and targets
TOP2A	DNA topoisomerase 2-alpha	6	DNA topoisomerase II alpha inhibitor; DNA topoisomerase II inhibitor	Phase 2	https://conductscience.org/library/genes/7153	Drugs and targets
ABL1	Bcr/Abl fusion protein	4	Bcr/Abl fusion protein inhibitor; Tyrosine-protein kinase ABL inhibitor	Approval	https://conductscience.org/library/genes/25	Drugs and targets
BCR	Bcr/Abl fusion protein	3	Bcr/Abl fusion protein inhibitor	Approval	https://conductscience.org/library/genes/613	Drugs and targets
CSF3R	Granulocyte colony-stimulating factor receptor	3	Granulocyte colony stimulating factor receptor agonist	Phase 1/2	https://conductscience.org/library/genes/1441	Drugs and targets
KIT	Mast/stem cell growth factor receptor Kit	3	Stem cell growth factor receptor inhibitor	Approval	https://conductscience.org/library/genes/3815	Drugs and targets
PDGFRB	Platelet-derived growth factor receptor	3	Platelet-derived growth factor receptor beta inhibitor; Platelet-derived growth factor receptor inhibitor	Approval	https://conductscience.org/library/genes/5159	Drugs and targets
RET	Proto-oncogene tyrosine-protein kinase receptor Ret	3	Tyrosine-protein kinase receptor RET inhibitor	Approval	https://conductscience.org/library/genes/5979	Drugs and targets
TOP2B	DNA topoisomerase II	3	DNA topoisomerase II inhibitor	Phase 2	https://conductscience.org/library/genes/7155	Drugs and targets
AXL	Tyrosine-protein kinase receptor UFO	2	Tyrosine-protein kinase receptor UFO inhibitor	Approval	https://conductscience.org/library/genes/558	Drugs and targets

1 to 10 of 114

See all 114 targets

Trials36

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.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00658814	Azacitidine and Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia	Phase 2	133	2014-01-06	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 OZOGAMICIN	Open Targets' reading, confirmed by NLM's indexing of its papers	ClinicalTrials.gov
NCT04802161	Comparing the Addition of an Anti-Cancer Drug, Pomalidomide, to the Usual Chemotherapy Treatment (Daunorubicin and Cytarabine Liposome) in Newly Diagnosed Acute Myeloid Leukemia With Myelodysplastic Syndrome-Related Changes	Phase 2	49	2025-12-19	Acute Myeloid Leukemia, Acute Myeloid Leukemia Post Cytotoxic Therapy, Acute Myeloid Leukemia With Multilineage Dysplasia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome, Myeloproliferative Neoplasm	CYTARABINE, DAUNORUBICIN, POMALIDOMIDE	Open Targets' reading of its record	ClinicalTrials.gov
NCT02658487	Vosaroxin and Infusional Cytarabine in Treating Patients With Untreated Acute Myeloid Leukemia	Phase 2	42	2020-06-09	Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia With Multilineage Dysplasia, Myeloid Sarcoma, Secondary Acute Myeloid Leukemia, Therapy-Related Acute Myeloid Leukemia, Therapy-Related Myelodysplastic Syndrome	CYTARABINE, VOSAROXIN	Open Targets' reading of its record	ClinicalTrials.gov
NCT00301834	Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders	Phase 2	35	2013-05-16	Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia	ALEMTUZUMAB, BUSULFAN, FLUDARABINE PHOSPHATE	Its record names the disease	ClinicalTrials.gov
NCT03912064	A Phase 1 Trial of CD25/Treg-depleted DLI Plus Ipilimumab for Myeloid Disease Relapse After Matched-HCT	Phase 1	25	2026-01-05	Acute Myeloid Leukemia, Myelodysplastic Syndromes, Myeloproliferative Neoplasms, Chronic Myelomonocytic Leukemia, Myelofibrosis	IPILIMUMAB	Open Targets' reading of its record	ClinicalTrials.gov
NCT00354172	Donor Umbilical Cord Blood Natural Killer Cells, Aldesleukin and Umbilical Cord Blood Transplant in Patients With Refractory Hematologic Cancers.	Phase 2	16	2009-12-31	Leukemia, Myelodysplastic Syndromes	ALDESLEUKIN, CYCLOPHOSPHAMIDE, FLUDARABINE	Its record names the disease	ClinicalTrials.gov
NCT03953898	Using the Anticancer Drug Olaparib to Treat Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome With an Isocitrate Dehydrogenase (IDH) Mutation	Phase 2	14	2025-03-30	Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia Post Cytotoxic Therapy, Recurrent Acute Myeloid Leukemia, Recurrent Myelodysplastic Syndrome, Refractory Acute Myeloid Leukemia, Refractory Myelodysplastic Syndrome	OLAPARIB	Open Targets' reading of its record	ClinicalTrials.gov
NCT01464359	T-Cell Depleted Double UCB for Refractory AML	Phase 2	3	2015-05-15	Acute Myelogenous Leukemia, Refractory Acute Myelogenous Leukemia	ALDESLEUKIN, BUSULFAN, CYCLOPHOSPHAMIDE, FLUDARABINE	Its record names the disease	ClinicalTrials.gov
NCT02115295	Cladribine, Idarubicin, Cytarabine, and Venetoclax in Treating Patients With Acute Myeloid Leukemia, High-Risk Myelodysplastic Syndrome, or Blastic Phase Chronic Myeloid Leukemia	Phase 2	508		Acute Biphenotypic Leukemia, Acute Myeloid Leukemia, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Blasts 10 Percent or More of Bone Marrow Nucleated Cells, Blasts 10 Percent or More of Peripheral Blood White Cells, de Novo Myelodysplastic Syndrome, Myelodysplastic Syndrome, Previously Treated Myelodysplastic Syndrome, Recurrent Acute Myeloid Leukemia, Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Refractory Acute Myeloid Leukemia, Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Secondary Acute Myeloid Leukemia, Untreated Adult Acute Myeloid Leukemia	CLADRIBINE, CYTARABINE, GILTERITINIB, IDARUBICIN, MIDOSTAURIN, VENETOCLAX	Open Targets' reading, confirmed by NLM's indexing of its papers	ClinicalTrials.gov
NCT05735184	A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML	Phase 1	420		Acute Myeloid Leukemia, Mixed Lineage Leukemia Gene Mutation, Refractory AML, AML With Mutated NPM1, Acute Myeloid Leukemia Recurrent, Acute Myeloid Leukemia, in Relapse, NPM1 Mutation, KMT2Ar, Myeloid Sarcoma, Nucleophosmin 1-mutated Acute Myeloid Leukemia	AZACITIDINE, VENETOCLAX, ZIFTOMENIB	Open Targets' reading, confirmed by NLM's indexing of its papers	ClinicalTrials.gov

1 to 10 of 36

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Comparisons in its trials2

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.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Treatment (Vosaroxin, Cytarabine) Induction 1 vs Induction 2: deaths from any cause	18 of 33	4 of 9	42	NCT02658487	Trial comparisons
Treatment (Vosaroxin, Cytarabine) Induction 1 vs Induction 2: serious adverse events	10 of 33	5 of 9	42	NCT02658487	Trial comparisons

Papers from its trials24

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

PMID	Listed as	Trials listing it	Source
42227701	DERIVED	1	PubMed
42013378	DERIVED	1	PubMed
41796974	DERIVED	1	PubMed
41075807	DERIVED	1	PubMed
40535475	DERIVED	1	PubMed
40513054	DERIVED	2	PubMed
40500616	DERIVED	1	PubMed
40193193	DERIVED	1	PubMed
40073835	DERIVED	1	PubMed
39303729	DERIVED	1	PubMed

1 to 10 of 24

See all 24 papers from its trials

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.

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