Disease · EFO_0004251

myeloproliferative disorder

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. myeloproliferative disorder. Diseases, record EFO_0004251. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/EFO_0004251

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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: myeloproliferative disorder; Open Targets
Open Targets ID: EFO_0004251; Open Targets
Other names: CMPD, CMPD, U, MPD, MPN, NCI CTEP SDC Chronic Myeloproliferative Disease Sub-Category Terminology, chronic myeloproliferative disease, chronic myeloproliferative disorder, chronic myeloproliferative neoplasm, myeloproliferative neoplasm, myeloproliferative neoplasm, chronic, myeloproliferative tumor, myeloproliferative tumour; Open Targets
Disease Ontology: DOID:2226; Disease Ontology
NCI Thesaurus: C4345; NCI Thesaurus
Orphanet: ORPHA:98274; Orphanet
Highest stage of its drugs: Approval, for 4 of its 97 drugs, from DailyMed and EMA Human Drugs; Open Targets

Drugs97

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
RUXOLITINIB	CHEMBL1789941	Small molecule	Approval	EMA Human Drugs	13	12	12	Drugs and targets
FEDRATINIB	CHEMBL1287853	Small molecule	Approval	EMA Human Drugs	4	3	3	Drugs and targets
ANAGRELIDE HYDROCHLORIDE	CHEMBL1200759	Small molecule	Approval	DailyMed	1	0	0	Drugs and targets
MOMELOTINIB	CHEMBL1078178	Small molecule	Approval	EMA Human Drugs	1	0	0	Drugs and targets
APREPITANT	CHEMBL1471	Small molecule	Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
ASPIRIN	CHEMBL25	Small molecule	Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
HYDROXYZINE	CHEMBL896	Small molecule	Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
AZACITIDINE	CHEMBL1489	Small molecule	Phase 2	ClinicalTrials.gov	11	11	10	Drugs and targets
CYTARABINE	CHEMBL803	Small molecule	Phase 2	ClinicalTrials.gov	11	11	11	Drugs and targets
CYCLOPHOSPHAMIDE	CHEMBL1200796	Small molecule	Phase 2	ClinicalTrials.gov	10	10	10	Drugs and targets

1 to 10 of 97

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Targets118

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
JAK2	Tyrosine-protein kinase JAK2	6	Tyrosine-protein kinase JAK2 inhibitor	Approval	https://conductscience.org/library/genes/3717	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
FLT3	Receptor-type tyrosine-protein kinase FLT3	4	Tyrosine-protein kinase receptor FLT3 inhibitor	Approval	https://conductscience.org/library/genes/2322	Drugs and targets
KIT	Mast/stem cell growth factor receptor Kit	4	Stem cell growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/3815	Drugs and targets
PDGFRB	Platelet-derived growth factor receptor	4	Platelet-derived growth factor receptor beta inhibitor; Platelet-derived growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/5159	Drugs and targets
ABL1	Bcr/Abl fusion protein	3	Bcr/Abl fusion protein inhibitor; Tyrosine-protein kinase ABL inhibitor	Phase 2	https://conductscience.org/library/genes/25	Drugs and targets
CSF3R	Granulocyte colony-stimulating factor receptor	3	Granulocyte colony stimulating factor receptor agonist	Phase 2	https://conductscience.org/library/genes/1441	Drugs and targets
FKBP1A	Peptidyl-prolyl cis-trans isomerase FKBP1A	3	FK506-binding protein 1A inhibitor	Phase 2	https://conductscience.org/library/genes/2280	Drugs and targets
IFNAR1	Interferon alpha/beta receptor	3	Interferon alpha/beta receptor agonist; Interferon alpha/beta receptor positive modulator	Phase 1	https://conductscience.org/library/genes/3454	Drugs and targets
IFNAR2	Interferon alpha/beta receptor	3	Interferon alpha/beta receptor agonist; Interferon alpha/beta receptor positive modulator	Phase 1	https://conductscience.org/library/genes/3455	Drugs and targets

1 to 10 of 118

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Trials83

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. 1 more is not shown, since the checks do not confirm it.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT01810588	Targeting the IPA and Matching for the Non-Inherited Maternal Antigen for Haplo-Cord Transplantation	Phase 2	270	2024-10-09	Hematologic Malignancies	FLUDARABINE, MELPHALAN, MYCOPHENOLATE MOFETIL, RITUXIMAB, TACROLIMUS ANHYDROUS	Open Targets' reading of its record	ClinicalTrials.gov
NCT00597714	Efficacy Study of T Cell Depleted Allogeneic Non-myeloablative Stem Cell Transplant	Phase 2	264	2014-06-02	Hodgkin's Disease, Non Hodgkin's Lymphoma, Myeloma, Leukemia, Myelodysplasia	ALEMTUZUMAB	Open Targets' reading of its record	ClinicalTrials.gov
NCT01572662	Allogeneic Transplantation Using Timed Sequential Busulfan and Fludarabine Conditioning	Phase 2	201	2023-06-29	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, FLUDARABINE	Open Targets' reading of its record	ClinicalTrials.gov
NCT00520130	Chemotherapy and Unrelated Donor Stem Cell Transplantation for Patients With Cancers of the Blood and Immune System	Phase 1/2	92	2017-08-24	Myelodysplastic Syndrome, Hodgkin's Lymphoma, Non-Hodgkin's Disease, Acute Leukemia, Multiple Myeloma	ALEMTUZUMAB, CYCLOPHOSPHAMIDE, CYCLOSPORINE, CYTARABINE, DOXORUBICIN, ETOPOSIDE, FILGRASTIM, FLUDARABINE, METHOTREXATE, PREDNISONE, RITUXIMAB, SIROLIMUS, TACROLIMUS, VINCRISTINE	Its record names the disease	ClinicalTrials.gov
NCT05223920	An Extension Study of Bomedemstat (IMG-7289/MK-3543) in Participants With Myeloproliferative Neoplasms (IMG-7289-CTP-202/MK-3543-005)	Phase 2	81	2025-09-22	Thrombocythemia, Essential, Primary Myelofibrosis	BOMEDEMSTAT	Its record names the disease	ClinicalTrials.gov
NCT00397813	Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders	Phase 2	77	2018-10-24	Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndrome, Essential Thrombocythemia, Myeloproliferative Neoplasm, Paroxysmal Nocturnal Hemoglobinuria, Polycythemia Vera, Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase, Primary Myelofibrosis, Refractory Anemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Ring Sideroblasts, Refractory Cytopenia With Multilineage Dysplasia, Refractory Cytopenia With Multilineage Dysplasia and Ring Sideroblasts	CYCLOSPORINE, FLUDARABINE PHOSPHATE, MYCOPHENOLATE MOFETIL	Its record names the disease	ClinicalTrials.gov
NCT01101880	Clofarabine, Cytarabine, and Filgrastim in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia, Advanced Myelodysplastic Syndrome, and/or Advanced Myeloproliferative Neoplasm	Phase 2	50	2017-10-19	Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Refractory Anemia With Excess Blasts, Untreated Adult Acute Myeloid Leukemia, Myeloproliferative Neoplasm With 10% Blasts or Higher	CLOFARABINE, CYTARABINE, FILGRASTIM	Its record names the disease	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	Its record names the disease	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
NCT00381550	3-AP and Fludarabine in Treating Patients With Myeloproliferative Disorders, Chronic Myelomonocytic Leukemia, or Accelerated Phase or Blastic Phase Chronic Myelogenous Leukemia	Phase 2	35	2014-05-21	Accelerated Phase Chronic Myelogenous Leukemia, Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Blastic Phase Chronic Myelogenous Leukemia, Chronic Eosinophilic Leukemia, Chronic Myelomonocytic Leukemia, Essential Thrombocythemia, Philadelphia Chromosome Negative Chronic Myelogenous Leukemia, Polycythemia Vera, Primary Myelofibrosis, Relapsing Chronic Myelogenous Leukemia	FLUDARABINE PHOSPHATE, TRIAPINE	Open Targets' reading of its record	ClinicalTrials.gov

1 to 10 of 83

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Comparisons in its trials16

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
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 49	47 of 150	199	NCT01572662	Trial 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 49	8 of 150	199	NCT01572662	Trial 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 49	93 of 150	199	NCT01572662	Trial 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 49	93 of 150	199	NCT01572662	Trial 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 49	8 of 150	199	NCT01572662	Trial comparisons
Cohort A - Lymphoid Disease vs Cohort B - Myeloid Disease: serious adverse events	16 of 80	10 of 57	137	NCT00597714	Trial comparisons
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm vs B - Cyclosporine (AC) Arm: Toxicities	43 of 44	42 of 45	89	NCT00520130	Trial comparisons
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm vs B - Cyclosporine (AC) Arm: deaths from any cause	8 of 44	19 of 45	89	NCT00520130	Trial comparisons
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm vs B - Cyclosporine (AC) Arm: serious adverse events	36 of 44	41 of 45	89	NCT00520130	Trial comparisons
A - Tacrolimus, Methotrexate, Sirolimus (TMS) Arm vs B - Cyclosporine (C) Arm: Early Treatment Related Mortality	1 of 39	0 of 42	81	NCT00520130	Trial comparisons

1 to 10 of 16

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Papers from its trials29

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
38877813	RESULT	1	PubMed
32266380	RESULT	1	PubMed
30409796	RESULT	1	PubMed
27774990	RESULT	1	PubMed
24362550	RESULT	1	PubMed
24269380	RESULT	1	PubMed
22234683	RESULT	1	PubMed
22014557	RESULT	1	PubMed
18430462	RESULT	1	PubMed
42304430	DERIVED	1	PubMed

1 to 10 of 29

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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.

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

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GET https://conductscience.org/api/diseases/v1/EFO_0004251/drugs?page=2

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