Disease · MONDO_0005570

hematologic disorder

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. hematologic disorder. Diseases, record MONDO_0005570. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0005570

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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: hematologic disorder; Open Targets
Open Targets ID: MONDO_0005570; Open Targets
Other names: Hematologic Diseases, blood disease, blood disorder, disease of hematopoietic system, disease of the blood and blood-forming organs, disease or disorder of haematopoietic system, disease or disorder of hematopoietic system, disorder of haematopoietic system, disorder of hematopoietic system, haematological disease, haematological disorder, haematological system disease, haematological system disorder, haematopoietic disease, haematopoietic system disease or disorder, hematologic and lymphocytic disorder, hematologic disease, hematological disease, hematological disorder, hematological system disease, hematological system disorder, hematopoietic disease, hematopoietic system disease, hematopoietic system disease or disorder, rare hematologic disease; Open Targets
Disease Ontology: DOID:74; Disease Ontology
MeSH: D006402; MeSH
NCI Thesaurus: C26323; NCI Thesaurus
Orphanet: ORPHA:97992; Orphanet
Highest stage of its drugs: Approval, for 1 of its 31 drugs, from TTD; Open Targets

Drugs31

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
HEPARIN	CHEMBL4650299	Oligosaccharide	Approval	TTD	1	0	0	Drugs and targets
CYCLOPHOSPHAMIDE	CHEMBL1200796	Small molecule	Phase 2	ClinicalTrials.gov	18	18	18	Drugs and targets
FLUDARABINE	CHEMBL1568	Small molecule	Phase 2	ClinicalTrials.gov	12	12	12	Drugs and targets
MYCOPHENOLATE MOFETIL	CHEMBL1456	Small molecule	Phase 2	ClinicalTrials.gov	10	10	10	Drugs and targets
BUSULFAN	CHEMBL820	Small molecule	Phase 2	ClinicalTrials.gov	9	9	9	Drugs and targets
FLUDARABINE PHOSPHATE	CHEMBL1096882	Small molecule	Phase 2	ClinicalTrials.gov	9	9	9	Drugs and targets
CYCLOSPORINE	CHEMBL160	Protein	Phase 2	ClinicalTrials.gov	8	8	8	Drugs and targets
THIOTEPA	CHEMBL671	Small molecule	Phase 2	ClinicalTrials.gov	5	5	5	Drugs and targets
ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE)	CHEMBL2108981		Phase 2	ClinicalTrials.gov	4	4	0	Drugs and targets
MELPHALAN	CHEMBL852	Small molecule	Phase 2	ClinicalTrials.gov	4	4	4	Drugs and targets

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See all 31 drugs

Targets72

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
FKBP1A	Peptidyl-prolyl cis-trans isomerase FKBP1A	3	FK506-binding protein 1A inhibitor	Phase 2	https://conductscience.org/library/genes/2280	Drugs and targets
MPL	Thrombopoietin receptor	2	Thrombopoietin receptor agonist	Unknown	https://conductscience.org/library/genes/4352	Drugs and targets
POLA1	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5422	Drugs and targets
POLA2	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/23649	Drugs and targets
POLD1	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5424	Drugs and targets
POLD2	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5425	Drugs and targets
POLD3	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/10714	Drugs and targets
POLD4	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/57804	Drugs and targets
POLE	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5426	Drugs and targets
POLE2	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5427	Drugs and targets

1 to 10 of 72

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Trials30

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

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT01119066	HLA-Compatible Related or Unrelated Donors With CD34+ Enriched, T-cell Depleted Peripheral Blood Stem Cells Isolated by the CliniMACS System in the Treatment of Patients With Hematologic Malignancies	Phase 2	422	2022-08-05	Acute Lymphoblastic Leukemia, Acute Myelogenous Leukemia, Myelodysplastic Syndrome, Multiple Myeloma	BUSULFAN, CLOFARABINE, CYCLOPHOSPHAMIDE, FLUDARABINE, MELPHALAN, THIOTEPA	Open Targets' reading of its record	ClinicalTrials.gov
NCT00544115	Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders	Phase 2	260	2023-05-10	Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Precancerous/Nonmalignant Condition	BUSULFAN, CYCLOPHOSPHAMIDE, CYCLOSPORINE, ETOPOSIDE, FLUDARABINE PHOSPHATE, MELPHALAN, METHOTREXATE, MYCOPHENOLATE MOFETIL, SIROLIMUS, TACROLIMUS ANHYDROUS	Open Targets' reading of its record	ClinicalTrials.gov
NCT03733249	Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study	Phase 1/2	187	2023-09-26	Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Primary Immunodeficiency, Anemia, Aplastic, Hemoglobinopathies, Cytopenia, Fanconi Anemia, Diamond Blackfan Anemia, Thalassemia, Anemia, Sickle Cell	RIVOGENLECLEUCEL	Open Targets' reading of its record	ClinicalTrials.gov
NCT00719888	Umbilical Cord Blood Transplant, Cyclophosphamide, Fludarabine, and Total-Body Irradiation in Treating Patients With Hematologic Disease	Phase 2	135	2025-03-07	Acute Biphenotypic Leukemia, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Burkitt Lymphoma, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Follicular Lymphoma, Lymphoblastic Lymphoma, Lymphoplasmacytic Lymphoma, Mantle Cell Lymphoma, Marginal Zone Lymphoma, Myelodysplastic Syndrome, Myelofibrosis, Non-Hodgkin Lymphoma, Plasma Cell Myeloma, Prolymphocytic Leukemia, Refractory Anemia, Small Lymphocytic Lymphoma	CYCLOPHOSPHAMIDE, CYCLOSPORINE, FLUDARABINE, MYCOPHENOLATE MOFETIL, THIOTEPA	Its record names the disease	ClinicalTrials.gov
NCT00448357	Allogeneic Hematopoietic Cell Transplantation for Patients With Busulfex-based Regimen	Phase 1/2	54	2017-07-17	Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes	BUSULFAN, FLUDARABINE PHOSPHATE, METHOTREXATE, TACROLIMUS ANHYDROUS	Its record names the disease	ClinicalTrials.gov
NCT00950846	Umbilical Cord Blood Transplant for Congenital Pediatric Disorders	Not applicable	40	2023-10-30	Congenital Pediatric Disorders	BUSULFAN, CYCLOPHOSPHAMIDE, FLUDARABINE	Open Targets' reading of its record	ClinicalTrials.gov
NCT00058825	Stem Cell Transplant for Hematologic Diseases	Phase 1/2	27	2015-10-22	Hematologic Malignancies	ALEMTUZUMAB, CYCLOSPORINE, FLUDARABINE, TACROLIMUS, TACROLIMUS ANHYDROUS	Its record names the disease	ClinicalTrials.gov
NCT02248597	Donor Stem Cell Transplant Followed by Cyclophosphamide in Treating Patients With Hematological Diseases	Phase 2	27	2023-10-11	Graft Versus Host Disease, Hematopoietic/Lymphoid Cancer	BUSULFAN, CYCLOPHOSPHAMIDE, FLUDARABINE PHOSPHATE	Open Targets' reading of its record	ClinicalTrials.gov
NCT00763490	Trial Of Double Umbilical Cord Blood Transplantation	Phase 2	20	2015-01-12	Hematological Malignancies	BUSULFAN, FLUDARABINE, MYCOPHENOLATE MOFETIL	Its record names the disease	ClinicalTrials.gov
NCT03711604	Compassionate Use Study of Tenalisib (RP6530)	Phase 1/2	17	2024-05-08	Hematological Malignancies	TENALISIB	Open Targets' reading of its record	ClinicalTrials.gov

1 to 10 of 30

See all 30 trials

Comparisons in its trials7

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
Rivogenlecleucel (BPX-501) Safety Population vs Rimiducid Safety Population: deaths from any cause	7 of 171	0 of 16	187	NCT03733249	Trial comparisons
Rivogenlecleucel (BPX-501) Safety Population vs Rimiducid Safety Population: serious adverse events	52 of 171	7 of 16	187	NCT03733249	Trial comparisons
Regimen A: High-dose TBI + Fludarabine + Cyclophosphamide vs Regimen B: Middle-intensity TBI + Fludarabine + Cyclophosphamide + Thiotepa: deaths from any cause	24 of 108	9 of 27	135	NCT00719888	Trial comparisons
Regimen A: High-dose TBI + Fludarabine + Cyclophosphamide vs Regimen B: Middle-intensity TBI + Fludarabine + Cyclophosphamide + Thiotepa: serious adverse events	47 of 108	7 of 27	135	NCT00719888	Trial comparisons
Isolex vs CLINIMACs: Number of Patients Who Engrafted With the Isolex/CLINIMACs System	15 of 19	7 of 8	27	NCT00058825	Trial comparisons
No Anti-thymocyte Globulin (ATG) vs Anti-thymocyte Globulin (ATG): deaths from any cause	1 of 5	2 of 10	15	NCT02722668	Trial comparisons
No Anti-thymocyte Globulin (ATG) vs Anti-thymocyte Globulin (ATG): serious adverse events	0 of 5	0 of 10	15	NCT02722668	Trial comparisons

Papers from its trials7

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
26210442	RESULT	1	PubMed
25046834	RESULT	1	PubMed
21873334	RESULT	1	PubMed
42907159	DERIVED	1	PubMed
36453638	DERIVED	1	PubMed
34875023	DERIVED	1	PubMed
34700343	DERIVED	1	PubMed

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_0005570

GET https://conductscience.org/api/diseases/v1/MONDO_0005570/drugs?page=2

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