Disease · MONDO_0015909

aplastic anemia

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. aplastic anemia. Diseases, record MONDO_0015909. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0015909

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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: aplastic anemia; Open Targets
Open Targets ID: MONDO_0015909; Open Targets
Other names: Aplastic anaemia, Medullar aplasia; Open Targets
Disease Ontology: DOID:12449; Disease Ontology
MeSH: D000741; MeSH
NCI Thesaurus: C2870; NCI Thesaurus
Orphanet: ORPHA:182040; Orphanet
Highest stage of its drugs: Approval, for 11 of its 62 drugs, from DailyMed, EMA Human Drugs, FDA, PMDA and TTD; Open Targets

Drugs62

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
ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE)	CHEMBL2108981		Approval	PMDA and TTD	58	56	4	Drugs and targets
FILGRASTIM	CHEMBL1201567	Protein	Approval	PMDA	23	20	12	Drugs and targets
ELTROMBOPAG	CHEMBL461101	Small molecule	Approval	EMA Human Drugs	21	20	20	Drugs and targets
PREDNISONE	CHEMBL635	Small molecule	Approval	DailyMed	2	1	1	Drugs and targets
CORTISONE ACETATE	CHEMBL1650	Small molecule	Approval	DailyMed	2	0	0	Drugs and targets
DEXAMETHASONE	CHEMBL384467	Small molecule	Approval	DailyMed	2	0	0	Drugs and targets
DEXAMETHASONE SODIUM PHOSPHATE	CHEMBL2021430	Small molecule	Approval	DailyMed	2	0	0	Drugs and targets
ELTROMBOPAG CHOLINE	CHEMBL6068328	Small molecule	Approval	FDA	1	0	0	Drugs and targets
MUPLESTIM	CHEMBL2108469	Protein	Approval	TTD	1	0	0	Drugs and targets
OXYMETHOLONE	CHEMBL1200585	Small molecule	Approval	TTD	1	0	0	Drugs and targets

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Targets58

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
MPL	Thrombopoietin receptor	6	Thrombopoietin receptor agonist	Approval	https://conductscience.org/library/genes/4352	Drugs and targets
NR3C1	Glucocorticoid receptor	6	Glucocorticoid receptor agonist	Approval	https://conductscience.org/library/genes/2908	Drugs and targets
AR	Androgen receptor	3	Androgen Receptor agonist	Approval	https://conductscience.org/library/genes/367	Drugs and targets
CSF3R	Granulocyte colony-stimulating factor receptor	3	Granulocyte colony stimulating factor receptor agonist	Approval	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
JAK1	Tyrosine-protein kinase JAK1	3	Tyrosine-protein kinase JAK1 inhibitor	Phase 1/2	https://conductscience.org/library/genes/3716	Drugs and targets
POLA1	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2/3	https://conductscience.org/library/genes/5422	Drugs and targets
POLA2	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2/3	https://conductscience.org/library/genes/23649	Drugs and targets
POLD1	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2/3	https://conductscience.org/library/genes/5424	Drugs and targets
POLD2	DNA polymerase (alpha/delta/epsilon)	2	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2/3	https://conductscience.org/library/genes/5425	Drugs and targets

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Trials147

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

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00003816	Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer	Phase 2/3	361	2021-08-13	Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Nonmalignant Neoplasm, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific	BUSULFAN, CARBOPLATIN, CYCLOPHOSPHAMIDE, ETOPOSIDE, FLUDARABINE PHOSPHATE, MELPHALAN, THIOTEPA	Its record names the disease	ClinicalTrials.gov
NCT00003838	Specialized Blood Cell Transplants for Cancers of the Blood and Bone Marrow	Phase 2	202	2022-10-07	Myeloproliferative Disorders, Acute Myelogenous Leukemia, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia	CYCLOSPORINE, METHOTREXATE	Its record names the disease	ClinicalTrials.gov
NCT00580034	Allo Non-Myeloablative SCT Utilizing Mis-Matched Family Member Stem Cells Purged Using Campath	Phase 2	176	2014-06-03	Lymphoma, Myeloma, Leukemia, Myelodysplasia, Solid Tumors	ALEMTUZUMAB	Its record names the disease	ClinicalTrials.gov
NCT01010217	Mismatched Transplantation Using High-dose Post-transplant Cyclophosphamide	Phase 2	176	2020-01-07	Blood Stem Cell Transplant Failure, Leukemia, Hematologic Malignancies	CYCLOPHOSPHAMIDE, FILGRASTIM, FLUDARABINE, MELPHALAN, MESNA, MYCOPHENOLATE MOFETIL, RITUXIMAB, TACROLIMUS ANHYDROUS, THIOTEPA	Its record names the disease	ClinicalTrials.gov
NCT00260689	Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia	Phase 2	136	2017-06-08	Immunosuppresion, Thrombocytopenia, Pancytopenia, Neutropenia	ALEMTUZUMAB, ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE), CYCLOSPORINE	Its record names the disease	ClinicalTrials.gov
NCT00631163	Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis	Phase 2	102	2021-06-29	Chronic Anemia, Transfusional Hemosiderosis	DEFERASIROX	Its record names the disease	ClinicalTrials.gov
NCT00001962	A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure	Phase 2	100	2015-05-14	Aplastic Anemia, Pure Red Cell Aplasia, Diamond Blackfan Anemia	DACLIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT00326417	Fludarabine-based Conditioning for Severe Aplastic Anemia (BMT CTN 0301)	Phase 1/2	97	2016-10-04	Anemia, Aplastic	CYCLOPHOSPHAMIDE, FLUDARABINE	Its record names the disease	ClinicalTrials.gov
NCT00582933	Phase II Trial of a Chemotherapy Alone Regimen of IV Busulfan (Busulfex), Melphalan and Fludarabine as Myeloablative Regimen Followed by an Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplant From an HLA-Identical, or HLA-Non Identical Related or Unrelated Donor	Phase 2	96	2016-02-01	Leukemia, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma, Allogeneic Marrow Transplant	BUSULFAN, FLUDARABINE, MELPHALAN	Its record names the disease	ClinicalTrials.gov
NCT04304820	Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)	Phase 2	80	2026-01-09	Severe Aplastic Anemia	CYCLOSPORINE, ELTROMBOPAG	Its record names the disease	ClinicalTrials.gov

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Comparisons in its trials23

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
Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality vs Group B: Stem Cell Transplant in Debilitating Hematologic Diseases: Number of Participants That Remained Disease-free Survival	26 of 43	56 of 57	100	NCT00003838	Trial comparisons
Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality vs Group B: Stem Cell Transplant in Debilitating Hematologic Diseases: Number of Participants Who Experienced Transplant Related Mortality	5 of 43	4 of 57	100	NCT00003838	Trial comparisons
Group A: Stem Cell Transplant in High Risk for Transplant Related Complications and Mortality vs Group B: Stem Cell Transplant in Debilitating Hematologic Diseases: Number of Participants Overall Survival	17 of 43	48 of 57	100	NCT00003838	Trial comparisons
Eltrombopag vs Eltrombopag (Post-treatment Long Term Follow-up): deaths from any cause	1 of 36	2 of 34	70	NCT04328727	Trial comparisons
r-ATG /Cyclosporine vs Alemtuzumab (Campath-1H): deaths from any cause	9 of 27	4 of 27	54	NCT00065260	Trial comparisons
r-ATG /Cyclosporine vs Alemtuzumab (Campath-1H): serious adverse events	7 of 27	12 of 27	54	NCT00065260	Trial comparisons
r-ATG /Cyclosporine vs Alemtuzumab (Campath-1H): Number of Participants With Robust Hematologic Recovery With Reticulocyte or Platelet Count	9 of 27	9 of 26	53	NCT00065260	Trial comparisons
Refractory vs Relapsed: Number of Participants With Hematological Response at 6 Months	0 of 5	18 of 42	47	NCT00195624	Trial comparisons
Refractory vs Relapsed: deaths from any cause	5 of 5	13 of 42	47	NCT00195624	Trial comparisons
Refractory vs Relapsed: serious adverse events	2 of 5	22 of 42	47	NCT00195624	Trial comparisons

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Papers from its trials68

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
35907408	RESULT	1	PubMed
30504345	RESULT	2	PubMed
30262561	RESULT	1	PubMed
28423296	RESULT	1	PubMed
28219216	RESULT	1	PubMed
26685770	RESULT	1	PubMed
26359881	RESULT	1	PubMed
26138856	RESULT	1	PubMed
25797174	RESULT	1	PubMed
24842530	RESULT	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_0015909

GET https://conductscience.org/api/diseases/v1/MONDO_0015909/drugs?page=2

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