Disease · EFO_0006927
severe aplastic anemia
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. severe aplastic anemia. Diseases, record EFO_0006927. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/EFO_0006927
On this page
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
- severe aplastic anemia
- Open Targets ID
- EFO_0006927
- Other names
- SAA
- Highest stage of its drugs
- Approval, for 1 of its 17 drugs, from DailyMed
Source Open Targets
Drugs17
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 |
|---|---|---|---|---|---|---|---|---|
| ELTROMBOPAG OLAMINE | CHEMBL3989691 | Small molecule | Approval | DailyMed | 1 | 0 | 0 | Drugs and targets |
| ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE) | CHEMBL2108981 | Phase 3 | ClinicalTrials.gov | 6 | 6 | 2 | Drugs and targets | |
| CYCLOSPORINE | CHEMBL160 | Protein | Phase 3 | ClinicalTrials.gov | 6 | 6 | 6 | Drugs and targets |
| ELTROMBOPAG | CHEMBL461101 | Small molecule | Phase 3 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets |
| RECOMBINANT HUMAN THROMBOPOIETIN | CHEMBL4802180 | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets | |
| SIROLIMUS | CHEMBL413 | Small molecule | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| TACROLIMUS | CHEMBL3989887 | Small molecule | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| CYCLOPHOSPHAMIDE | CHEMBL1200796 | Small molecule | Phase 2 | ClinicalTrials.gov | 5 | 5 | 5 | Drugs and targets |
| FLUDARABINE | CHEMBL1568 | Small molecule | Phase 2 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets |
| MELPHALAN | CHEMBL852 | Small molecule | Phase 2 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
1 to 10 of 17
See all 17 drugsTargets11
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 | 3 | Thrombopoietin receptor agonist | Approval | https://conductscience.org/library/genes/4352 | Drugs and targets |
| FKBP1A | Peptidyl-prolyl cis-trans isomerase FKBP1A | 2 | FK506-binding protein 1A inhibitor | Phase 3 | https://conductscience.org/library/genes/2280 | Drugs and targets |
| CD2 | T-cell surface antigen CD2 | 1 | T-cell surface antigen CD2 inhibitor | Unknown | https://conductscience.org/library/genes/914 | Drugs and targets |
| CD52 | CAMPATH-1 antigen | 1 | CAMPATH-1 antigen inhibitor | Phase 2 | https://conductscience.org/library/genes/1043 | Drugs and targets |
| DHFR | Dihydrofolate reductase | 1 | Dihydrofolate reductase inhibitor | Unknown | https://conductscience.org/library/genes/1719 | Drugs and targets |
| JAK1 | Tyrosine-protein kinase JAK1 | 1 | Tyrosine-protein kinase JAK1 inhibitor | Phase 1/2 | https://conductscience.org/library/genes/3716 | Drugs and targets |
| JAK2 | Tyrosine-protein kinase JAK2 | 1 | Tyrosine-protein kinase JAK2 inhibitor | Phase 1/2 | https://conductscience.org/library/genes/3717 | Drugs and targets |
| JAK3 | Tyrosine-protein kinase JAK3 | 1 | Tyrosine-protein kinase JAK3 inhibitor | Phase 1/2 | https://conductscience.org/library/genes/3718 | Drugs and targets |
| NR3C1 | Glucocorticoid receptor | 1 | Glucocorticoid receptor agonist | Phase 2 | https://conductscience.org/library/genes/2908 | Drugs and targets |
| PPIA | Peptidyl-prolyl cis-trans isomerase A | 1 | Cyclophilin A modulator | Phase 3 | https://conductscience.org/library/genes/5478 | Drugs and targets |
1 to 10 of 11
See all 11 targetsTrials15
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 |
|---|---|---|---|---|---|---|---|---|
| NCT01623167 | Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia | Phase 1/2 | 207 | 2020-06-05 | Severe Aplastic Anemia | ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE), CYCLOSPORINE, ELTROMBOPAG | Its record names the disease | ClinicalTrials.gov |
| NCT01319851 | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation | Not applicable | 3 | 2015-03-09 | Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic-granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Schwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Fanconi Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia | ALEFACEPT | Its record names the disease | ClinicalTrials.gov |
| NCT01187017 | A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia | Phase 1/2 | 1 | 2016-03-02 | Aplastic Anemia, Neutropenia, Pancytopenia, Severe Aplastic Anemia | CYCLOPHOSPHAMIDE, FLUDARABINE | Its record names the disease | ClinicalTrials.gov |
| NCT00993343 | Randomized Trial Comparing Sirolimus and Tacrolimus Versus Cyclosporine and Methotrexate as Graft-versus-host Disease (GVHD) Prophylaxis After Allogeneic Stem Cell Transplantation | Phase 3 | 215 | Graft-versus-host Disease, Survival | SIROLIMUS, TACROLIMUS | Its record names the disease | ClinicalTrials.gov | |
| NCT02099747 | hATG+CsA vs hATG+CsA+Eltrombopag for SAA | Phase 3 | 202 | Severe Aplastic Anemia | CYCLOSPORINE, ELTROMBOPAG | Its record names the disease | ClinicalTrials.gov | |
| NCT00003662 | Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Other Hematologic or Metabolic Diseases | Phase 2 | 90 | Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes, Thymic Carcinoma | BUSULFAN, CYCLOPHOSPHAMIDE, CYCLOSPORINE, MELPHALAN, METHYLPREDNISOLONE | Its record names the disease | ClinicalTrials.gov | |
| NCT07001397 | Study on the Short-term Efficacy and Safety of Recombinant Human Thrombopoietin Combined With Immunosuppressant Sequential Eltrombopag Ethanolamine Dry Suspension in the Treatment of SAA/TD-NSAA | Phase 3 | 70 | Aplastic Anemias | ELTROMBOPAG, RECOMBINANT HUMAN THROMBOPOIETIN | Its record names the disease | ClinicalTrials.gov | |
| NCT03025698 | A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia | Phase 2 | 51 | Aplastic Anemia | ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE), CYCLOSPORINE, ELTROMBOPAG | Its record names the disease | ClinicalTrials.gov | |
| NCT00895739 | Alemtuzumab and Low-Dose Cyclosporine in Treating Patients With Severe Aplastic Anemia or Acquired Marrow Failure | Phase 2 | 50 | Nonmalignant Neoplasm | ALEMTUZUMAB, CYCLOSPORINE | Its record names the disease | ClinicalTrials.gov | |
| NCT02845596 | Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia | Not applicable | 40 | Severe Aplastic Anemia | CYCLOPHOSPHAMIDE, CYCLOSPORINE, FLUDARABINE, METHOTREXATE | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 15
See all 15 trialsComparisons in its trials0
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.
None in this release.
Papers from its trials22
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
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/EFO_0006927
GET https://conductscience.org/api/diseases/v1/EFO_0006927/papers?page=2