Disease · MONDO_0005170

myeloid neoplasm

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. myeloid neoplasm. Diseases, record MONDO_0005170. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0005170

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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 neoplasm; Open Targets
Open Targets ID: MONDO_0005170; Open Targets
Other names: myeloid malignancy, myeloid tumor, myeloid tumour; Open Targets
Disease Ontology: DOID:0070004; Disease Ontology
NCI Thesaurus: C9290; NCI Thesaurus
Highest stage of its drugs: Phase 2/3, for 6 of its 51 drugs, from ClinicalTrials.gov; Open Targets

Drugs51

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
BUSULFAN	CHEMBL820	Small molecule	Phase 2/3	ClinicalTrials.gov	9	9	9	Drugs and targets
AZACITIDINE	CHEMBL1489	Small molecule	Phase 2/3	ClinicalTrials.gov	7	7	6	Drugs and targets
VENETOCLAX	CHEMBL3137309	Small molecule	Phase 2/3	ClinicalTrials.gov	6	6	6	Drugs and targets
FLUDARABINE	CHEMBL1568	Small molecule	Phase 2/3	ClinicalTrials.gov	4	4	4	Drugs and targets
DECITABINE	CHEMBL1201129	Small molecule	Phase 2/3	ClinicalTrials.gov	2	2	2	Drugs and targets
CEDAZURIDINE	CHEMBL3237547	Small molecule	Phase 2/3	ClinicalTrials.gov	1	1	1	Drugs and targets
CYTARABINE	CHEMBL803	Small molecule	Phase 2	ClinicalTrials.gov	5	5	4	Drugs and targets
ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE)	CHEMBL2108981		Phase 2	ClinicalTrials.gov	4	4	0	Drugs and targets
FLUDARABINE PHOSPHATE	CHEMBL1096882	Small molecule	Phase 2	ClinicalTrials.gov	4	4	4	Drugs and targets
CLADRIBINE	CHEMBL1619	Small molecule	Phase 2	ClinicalTrials.gov	3	3	3	Drugs and targets

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Targets105

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
CSF3R	Granulocyte colony-stimulating factor receptor	3	Granulocyte colony stimulating factor receptor agonist	Phase 2	https://conductscience.org/library/genes/1441	Drugs and targets
TOP2A	DNA topoisomerase 2-alpha	3	DNA topoisomerase II alpha inhibitor; DNA topoisomerase II inhibitor	Phase 2	https://conductscience.org/library/genes/7153	Drugs and targets
BCL2	Apoptosis regulator Bcl-2	2	Apoptosis regulator Bcl-2 inhibitor	Phase 2/3	https://conductscience.org/library/genes/596	Drugs and targets
DNMT1	DNA (cytosine-5)-methyltransferase 1	2	DNA (cytosine-5)-methyltransferase 1 inhibitor	Phase 2/3	https://conductscience.org/library/genes/1786	Drugs and targets
DNMT3A	DNA (cytosine-5)-methyltransferase 3A	2	DNA (cytosine-5)-methyltransferase 3A inhibitor	Phase 2/3	https://conductscience.org/library/genes/1788	Drugs and targets
FGFR1	Fibroblast growth factor receptor	2	Fibroblast growth factor receptor 1 inhibitor; Fibroblast growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/2260	Drugs and targets
FGFR2	Fibroblast growth factor receptor	2	Fibroblast growth factor receptor 2 inhibitor; Fibroblast growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/2263	Drugs and targets
FGFR3	Fibroblast growth factor receptor	2	Fibroblast growth factor receptor 3 inhibitor; Fibroblast growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/2261	Drugs and targets
FKBP1A	Peptidyl-prolyl cis-trans isomerase FKBP1A	2	FK506-binding protein 1A inhibitor	Phase 2	https://conductscience.org/library/genes/2280	Drugs and targets
FLT3	Receptor-type tyrosine-protein kinase FLT3	2	Tyrosine-protein kinase receptor FLT3 inhibitor	Phase 2	https://conductscience.org/library/genes/2322	Drugs and targets

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Trials38

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. 3 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00185640	Allogeneic Transplantation Using Total Lymphoid Irradiation (TLI) and Anti-Thymocyte Globulin (ATG) for Older Patients With Hematologic Malignancies	Phase 2	303	2017-10-03	Blood Cancer, Leukemia	CYCLOSPORINE, FILGRASTIM, MYCOPHENOLATE MOFETIL	Open Targets' reading of its record	ClinicalTrials.gov
NCT04189445	Futibatinib in Patients With Specific FGFR Aberrations	Phase 2	115	2025-10-08	Advanced or Metastatic Solid Tumor, Advanced or Metastatic Gastric or Gastroesophageal Cancer, Myeloid or Lymphoid Neoplasms (MLN)	FUTIBATINIB	Open Targets' reading of its record	ClinicalTrials.gov
NCT02520427	A Phase 1 Study of AMG 330 in Subjects With Myeloid Malignancies	Phase 1	95	2024-07-31	Relapsed/Refractory AML, Minimal Residual Disease Positive AML, Myelodysplastic Syndrome	ELUVIXTAMAB	Open Targets' reading of its record	ClinicalTrials.gov
NCT01731951	Imetelstat Sodium in Treating Participants With Primary or Secondary Myelofibrosis	Phase 2	80	2021-09-21	Primary Myelofibrosis, Secondary Myelofibrosis, Myeloid Malignancies	IMETELSTAT, IMETELSTAT SODIUM	Open Targets' reading of its record	ClinicalTrials.gov
NCT01823198	Donor Natural Killer Cells and Donor Stem Cell Transplant in Treating Patients With High Risk Myeloid Malignancies	Phase 1/2	63	2023-11-07	Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Erythroid Leukemia, Acute Megakaryoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Acute Myeloid Leukemia in Remission, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Blasts Under 20 Percent of Bone Marrow Nucleated Cells, Blasts Under 20 Percent of Peripheral Blood White Cells, Chronic Myelomonocytic Leukemia, High Risk Myelodysplastic Syndrome, Myelodysplastic Syndrome, Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Therapy-Related Acute Myeloid Leukemia, Therapy-Related Myelodysplastic Syndrome	ALDESLEUKIN, BUSULFAN, FLUDARABINE PHOSPHATE, TROCULEUCEL	Open Targets' reading of its record	ClinicalTrials.gov
NCT04214860	APR-246 in Combination With Venetoclax and Azacitidine in TP53-Mutant Myeloid Malignancies	Phase 1	51	2025-02-21	Myeloid Malignancy	AZACITIDINE, EPRENETAPOPT, VENETOCLAX	Its record names the disease	ClinicalTrials.gov
NCT03012672	Higher or Lower Dose Cladribine, Cytarabine, and Mitoxantrone in Treating Medically Less Fit Patients With Newly Diagnosed Acute Myeloid Leukemia or Myeloid Neoplasm	Phase 2	50	2021-11-01	Acute Leukemia of Ambiguous Lineage, Acute Myeloid Leukemia, Myeloid Neoplasm	CLADRIBINE, CYTARABINE, MITOXANTRONE HYDROCHLORIDE	Its record names the disease	ClinicalTrials.gov
NCT03011372	A Study to Evaluate the Efficacy and Safety of Pemigatinib (INCB054828) in Subjects With Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement - (FIGHT-203)	Phase 2	47	2025-11-18	MPN (Myeloproliferative Neoplasms)	PEMIGATINIB	Open Targets' reading of its record	ClinicalTrials.gov
NCT01392989	Post T-plant Infusion of Allogeneic Cytokine Induced Killer (CIK) Cells as Consolidative Therapy in Myelodysplastic Syndromes/Myeloproliferative Disorders	Phase 2	44	2019-05-07	Neural Tube Defects, Anemia, Leukemia, Myeloid, Bone Marrow Transplant Failure, Myelodysplastic Syndromes (MDS), Myeloproliferative Disorders	CYCLOSPORINE, MYCOPHENOLATE MOFETIL	Open Targets' reading of its record	ClinicalTrials.gov
NCT00014495	Chemotherapy and Monoclonal Antibody Therapy in Treating Patients With Advanced Myeloid Cancer	Phase 1/2	32	2016-01-22	Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms	CYTARABINE, LINTUZUMAB	Open Targets' reading of its record	ClinicalTrials.gov

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Comparisons in its trials24

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
Futibatinib (Cohort A) vs Futibatinib (Cohort B): deaths from any cause	54 of 87	24 of 28	115	NCT04189445	Trial comparisons
Futibatinib (Cohort A) vs Futibatinib (Cohort B): serious adverse events	23 of 87	6 of 28	115	NCT04189445	Trial comparisons
ARM I (HIGHER-DOSE): vs ARM II (LOWER-DOSE):: deaths from any cause	16 of 20	25 of 30	50	NCT03012672	Trial comparisons
ARM I (HIGHER-DOSE): vs ARM II (LOWER-DOSE):: serious adverse events	9 of 20	14 of 30	50	NCT03012672	Trial comparisons
Cohort 1 vs Cohort 2: To Evaluate the Tolerabililty and the Incidence of Treatment-Emergent Adverse Events of Administration of APR 246 in Combination With Venetoclax and Azacitidine in Patients With TP53 Mutant Myeloid Malignancies.	5 of 6	32 of 43	49	NCT04214860	Trial comparisons
Cohort 1 vs Cohort 2: deaths from any cause	6 of 6	26 of 43	49	NCT04214860	Trial comparisons
Cohort 1 vs Cohort 2: serious adverse events	5 of 6	32 of 43	49	NCT04214860	Trial comparisons
Post-Chemotherapy vs Post-Transplant: deaths from any cause	5 of 30	2 of 9	39	NCT02756572	Trial comparisons
Post-Chemotherapy vs Post-Transplant: serious adverse events	30 of 30	9 of 9	39	NCT02756572	Trial comparisons
Arm I: 1200cGy vs Arm II: 1350cGy: deaths from any cause	19 of 23	2 of 2	25	NCT00540995	Trial comparisons

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Papers from its trials21

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
26885686	RESULT	1	PubMed
26146806	RESULT	1	PubMed
24607552	RESULT	1	PubMed
20858843	RESULT	1	PubMed
20528878	RESULT	1	PubMed
19423725	RESULT	1	PubMed
16192477	RESULT	1	PubMed
41180818	DERIVED	1	PubMed
40856555	DERIVED	1	PubMed
39892383	DERIVED	1	PubMed

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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_0005170

GET https://conductscience.org/api/diseases/v1/MONDO_0005170/drugs?page=2

API reference For AI assistants