Disease · MONDO_0001705

pure red-cell aplasia

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. pure red-cell aplasia. Diseases, record MONDO_0001705. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0001705

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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: pure red-cell aplasia; Open Targets
Open Targets ID: MONDO_0001705; Open Targets
Other names: PRCA, Pure Red Cell Aplasia, Acquired, Red cell aplasia; Open Targets
Disease Ontology: DOID:1340; Disease Ontology
MeSH: D012010; MeSH
NCI Thesaurus: C34974; NCI Thesaurus
Highest stage of its drugs: Phase 3, for 4 of its 20 drugs, from ClinicalTrials.gov; Open Targets

Drugs20

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
CYCLOPHOSPHAMIDE	CHEMBL1200796	Small molecule	Phase 3	ClinicalTrials.gov	3	3	3	Drugs and targets
CYCLOSPORINE	CHEMBL160	Protein	Phase 3	ClinicalTrials.gov	2	2	2	Drugs and targets
MYCOPHENOLATE MOFETIL	CHEMBL1456	Small molecule	Phase 3	ClinicalTrials.gov	2	2	2	Drugs and targets
PREDNISOLONE	CHEMBL131	Small molecule	Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
FILGRASTIM	CHEMBL1201567	Protein	Phase 2	ClinicalTrials.gov	2	2	1	Drugs and targets
RITUXIMAB	CHEMBL1201576	Antibody	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
ALEMTUZUMAB	CHEMBL1201587	Antibody	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
BORTEZOMIB	CHEMBL325041	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
DACLIZUMAB	CHEMBL1201605	Antibody	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
DEXAMETHASONE	CHEMBL384467	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets

1 to 10 of 20

See all 20 drugs

Targets51

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
NR3C1	Glucocorticoid receptor	4	Glucocorticoid receptor agonist	Phase 3	https://conductscience.org/library/genes/2908	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
ADRM1	26S proteasome	1	26S proteasome inhibitor	Phase 2	https://conductscience.org/library/genes/11047	Drugs and targets
CD38	ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1	1	Lymphocyte differentiation antigen CD38 inhibitor	Phase 2	https://conductscience.org/library/genes/952	Drugs and targets
CD52	CAMPATH-1 antigen	1	CAMPATH-1 antigen inhibitor	Phase 2	https://conductscience.org/library/genes/1043	Drugs and targets
EPOR	Erythropoietin receptor	1	Erythropoietin receptor agonist	Phase 2	https://conductscience.org/library/genes/2057	Drugs and targets
FKBP1A	Peptidyl-prolyl cis-trans isomerase FKBP1A	1	FK506-binding protein 1A inhibitor	Phase 2	https://conductscience.org/library/genes/2280	Drugs and targets
IL2RA	Interleukin-2 receptor	1	Interleukin-2 receptor inhibitor	Phase 2	https://conductscience.org/library/genes/3559	Drugs and targets
IL2RB	Interleukin-2 receptor	1	Interleukin-2 receptor inhibitor	Phase 2	https://conductscience.org/library/genes/3560	Drugs and targets
IL2RG	Interleukin-2 receptor	1	Interleukin-2 receptor inhibitor	Phase 2	https://conductscience.org/library/genes/3561	Drugs and targets

1 to 10 of 51

See all 51 targets

Trials10

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
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
NCT00314795	Efficacy and Safety of Peginesatide (AF37702) in the Treatment of Anemia in Participants With Chronic Kidney Disease	Phase 2	22	2019-03-14	Anemia, Chronic Kidney Disease, Chronic Renal Failure, Pure Red Cell Aplasia	PEGINESATIDE	Its record names the disease	ClinicalTrials.gov
NCT00229619	Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia	Phase 2	11	2014-07-28	Anemia, Aplastic, Red-Cell Aplasia, Pure, Anemia, Diamond-Blackfan	RITUXIMAB	Its record names the disease	ClinicalTrials.gov
NCT05559827	Efficacy of the antiCD38 Monoclonal Antibody Isatuximab in the Treatment of PCRA by Major ABO Mismatch After Allogeneic Hematopoietic Stem Cell Transplantation	Phase 2	90		Immunological Pure Red Cell Aplasia	ISATUXIMAB	Its record names the disease	ClinicalTrials.gov
NCT06412497	MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.	Phase 2	60		Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia, Acquired Pure Red Cell Aplasia, Paroxysmal Nocturnal Hemoglobinuria	CYCLOPHOSPHAMIDE, FLUDARABINE, MYCOPHENOLATE MOFETIL, RITUXIMAB, TACROLIMUS ANHYDROUS	Its record names the disease	ClinicalTrials.gov
NCT00472433	The Efficacy and Safety of Alemtuzumab in Auotoimmune Cytopenias	Phase 2	30		Autoimmune Cytopenias	ALEMTUZUMAB	Its record names the disease	ClinicalTrials.gov
NCT04423367	Bortezomib Plus Dexamethasone for Acquired Pure Red Cell Aplasia Failure or Relapse After First-line Treatment	Phase 2	18		Acquired Pure Red Cell Aplasia	BORTEZOMIB, DEXAMETHASONE	Open Targets' reading, confirmed by NLM's indexing of its papers	ClinicalTrials.gov
NCT00006055	Autologous Peripheral Blood Stem Cell Transplantation in Patients With Life Threatening Autoimmune Diseases	Not applicable	10		Purpura, Schoenlein-Henoch, Graft Versus Host Disease, Anemia, Hemolytic, Autoimmune, Rheumatoid Arthritis, Churg-Strauss Syndrome, Hypersensitivity Vasculitis, Wegener's Granulomatosis, Systemic Lupus Erythematosus, Giant Cell Arteritis, Pure Red Cell Aplasia, Juvenile Rheumatoid Arthritis, Polyarteritis Nodosa, Autoimmune Thrombocytopenic Purpura, Takayasu Arteritis	CYCLOPHOSPHAMIDE, CYCLOSPORINE, FILGRASTIM, METHYLPREDNISOLONE, PREDNISONE	Its record names the disease	ClinicalTrials.gov
NCT01288131	Study of Anti-r-HuEpo Associated Pure Red Cell Aplasia (PRCA) Treatment	Phase 3	8		Anti-r-HuEpo Associated PRCA Subjects	CYCLOPHOSPHAMIDE, CYCLOSPORINE, MYCOPHENOLATE MOFETIL, PREDNISOLONE	Its record names the disease	ClinicalTrials.gov
NCT07031115	Linperlisib in the Treatment of aPRCA	Phase 2	0		Acquired Pure Red Cell Aplasia	LINPERLISIB	Its record names the disease	ClinicalTrials.gov

Comparisons 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 trials2

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
19890127	RESULT	1	PubMed
40772317	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_0001705

GET https://conductscience.org/api/diseases/v1/MONDO_0001705/targets?page=2

API reference For AI assistants