Rare & Orphan Lab · DeCure for X

DeCure for Pure red-cell aplasia

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for pure red-cell aplasia — screening already-approved drugs against its 5-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module5 genesLead labRare & Orphan
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Rare & OrphanDOID:1340$DeCureRare

The disease map

Disease modulePure red-cell aplasia maps to a 5-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.

Research record

01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash

Current lead

No approved-drug candidate for pure red-cell aplasia is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.

Molecular view

inosine monophosphate dehydrogenase 1 (IMPDH1)IMPDH1 is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.

Loading structure…
helix sheet cprdrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 1JCN · 2.5 Å · ligand 6-CHLOROPURINE RIBOSIDE, 5'-MONOPHOSPHATE (CPR). Experimental structure, not a prediction.

What the evidence adds up to

Pure red-cell aplasia is a syndrome of severe anaemia, reticulocytopenia, and absent erythroid precursors in otherwise normal bone marrow. In the adult form, antibody-mediated mechanisms have been demonstrated in almost half of patients. Immunosuppressive agents such as corticosteroids, cyclophosphamide, and antithymocyte globulin have been used for immunologically mediated cases, and in vitro assays for haematopoietic progenitor cells may help predict response to such therapy. In a 1987 report, high-dose intravenous gamma globulin was investigated as a treatment for antibody-mediated pure red-cell aplasia, but the abstract provides no numerical outcomes, response rates, or survival data for that intervention.

A 1985 case report describes a 22-year-old woman with idiopathic pure red-cell aplasia who had failed to respond to high-dose corticosteroids. After a series of 10 plasma exchange procedures, bone marrow erythropoiesis and reticulocyte count returned to normal, and haematological remission persisted for 12 months. A 2004 report describes two cases of pure red-cell aplasia occurring after ABO major-mismatched allogeneic peripheral blood stem cell transplantation, a complication for which there is no standard treatment. Both cases were successfully treated with plasma exchange and low-dose steroid, though again no specific numerical outcomes such as transfusion reduction or duration of remission are given.

The evidence for plasma exchange in pure red-cell aplasia is limited to single case reports and two-case series, with no controlled trials. The 1987 gamma globulin study does not report any measurable results. What remains missing are prospective trials with adequate sample sizes, standardised outcome measures such as transfusion independence and haemoglobin recovery, and stratification by underlying aetiology (idiopathic, post-transplant, antibody-mediated). Funding for such trials and a consensus on treatment protocols are also absent.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

New England Journal of Medicine · 1987 · 98 citations

Treatment of Antibody-Mediated Pure Red-Cell Aplasia with High-Dose Intravenous Gamma Globulin

AbstractPURE red-cell aplasia is a syndrome characterized by severe anemia, reticulocytopenia, and an absence of erythroid precursor cells in bone marrow that is otherwise normal. This failure of erythropoiesis has been shown to be antibody-mediated in almost half the patients with the adult form of red-cell aplasia.1 2 3 4 Immunosuppressive agents such as corticosteroids, cyclophosphamide, and antithymocyte globulin have been used to treat immunologically mediated red-cell aplasia.2 , 5 6 7 8 Results of in vitro assays for hematopoietic progenitor cells may assist in predicting which patients will respond to immunosuppressive therapy.9 10 11 Red-cell aplasia in children usually differs from the disorder in adults. There is a constitutional . . .

https://doi.org/10.1056/nejm198710153171606
Acta Haematologica · 2009 · 25 citations

Pure Red Cell Aplasia Associated with Systemic Lupus erythematosus: Remission after a Single Course of Intravenous Immunoglobulin

AbstractPure red cell aplasia is characterized by severe anemia, with reticulocytopenia and absence of precursor cells in the bone marrow. Many modes of treatment have been described, including the use of immunosuppressive agents. Recently repeated courses of high-dose intravenous immunoglobulins have been used successfully in patients with idiopathic pure red cell aplasia. We here describe a 22-year-old woman who developed pure red cell aplasia in the course of systemic lupus erythematosus. After failure of corticosteroid therapy the patient was treated with one course of high-dose intravenous immunoglobulins with complete remission. No further therapy was required.

https://doi.org/10.1159/000204509
American Journal of Hematology · 1985 · 16 citations

Treatment of red cell aplasia with antithymocyte globulin: Repeated inductions of complete remissions in two patients

AbstractTwo patients with red cell aplasia unresponsive to prednisone and cyclophosphamide were treated with antithymocyte globulin (ATG). Both patients developed reticulocytosis within 2-4 days after ATG treatment and had complete remissions. Within 4-6 months, they relapsed, and after retreatment with ATG both again developed reticulocytosis and remission. ATG should be considered for the treatment of patients with red cell aplasia who fail to respond to glucocorticoid/alkylator treatment.

https://doi.org/10.1002/ajh.2830200212
Scandinavian Journal of Haematology · 1985 · 13 citations

Remission of acquired pure red cell aplasia following plasma exchanges:

AbstractA 22-year-old woman had an idiopathic pure red cell aplasia that failed to respond to high doses of corticosteroids. After a series of 10 plasma exchange procedures, bone marrow erythropoiesis and reticulocyte blood count returned to normal; the haematological remission has been now persistent for 12 months. The place of plasma exchange in the management of pure red cell aplasia and its mode of action will be discussed.

https://doi.org/10.1111/j.1600-0609.1985.tb00737.x
The Kaohsiung Journal of Medical Sciences · 2004 · 11 citations · open access

Pure Red Cell Aplasia After ABO Major‐Mismatched Allogeneic Peripheral Blood Stem Cell Transplantation Successfully Treated with Plasma Exchange and Low‐Dose Steroid: Two Case Reports

AbstractPure red cell aplasia (PRCA) is a complication of ABO-incompatible allogeneic stem cell transplantation. The mechanism is not well known, although the isoagglutinin titer before transplantation or cyclosporine use is considered to be the cause. Patients with this complication require more blood transfusions than those without it. There is no standard treatment. We report two cases of PRCA after allogeneic peripheral blood stem cell transplantation that were successfully treated with plasma exchange and low-dose steroid.

https://doi.org/10.1016/s1607-551x(09)70096-8
American Journal of Clinical Pathology · 1989 · 4 citations

Azathloprine as Possible Explanation of Etiology of Pure Red Cell Aplasia in Myasthenics

AbstractJournal Article Azathloprine as Possible Explanation of Etiology of Pure Red Cell Aplasia in Myasthenics Get access N. Smith, N. Smith The Queen Elizabeth Hospital Department of Hematology Queen Elizabeth Medical Centre Edgbaston, Birmingham, B15 2TH United Kingdom Search for other works by this author on: Oxford Academic Google Scholar B. J. Boughton B. J. Boughton The Queen Elizabeth Hospital Department of Hematology Queen Elizabeth Medical Centre Edgbaston, Birmingham, B15 2TH United Kingdom Search for other works by this author on: Oxford Academic Google Scholar American Journal of Clinical Pathology, Volume 91, Issue 1, 1 January 1989, Page 112, https://doi.org/10.1093/ajcp/91.1.112a Published: 01 January 1989

https://doi.org/10.1093/ajcp/91.1.112a
Dialnet (Universidad de la Rioja) · 2000 · 3 citations

Los miembros de la asamblea celta. Notas para su estudio.

AbstractA 22-year-old woman had an idiopathic pure red cell aplasia that failed to respond to high doses of corticosteroids. After a series of 10 plasma exchange procedures, bone marrow erythropoiesis and reticulocyte blood count returned to normal; the haematological remission has been now persistent for 12 months. The place of plasma exchange in the management of pure red cell aplasia and its mode of action will be discussed.

https://doi.org/10.1111/j.1600-0609.1985.tb00737.x

Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.

DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.