DeCure for Congenital vitamin K-dependent coagulation factors deficiency
DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for congenital vitamin K-dependent coagulation factors deficiency — screening already-approved drugs against its 2-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCongenital vitamin K-dependent coagulation factors deficiency maps to a 2-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 congenital vitamin k-dependent coagulation factors deficiency 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
vitamin K epoxide reductase complex subunit 1 (VKORC1) — VKORC1 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 ua7drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6WVH · 1.99 Å · ligand Brodifacoum (UA7). Experimental structure, not a prediction.
What the evidence adds up to
Congenital vitamin K-dependent coagulation factor deficiency is a very rare bleeding disorder that usually presents with intracerebral bleeding in the first weeks of life and can be fatal. A 2005 case report describes one patient who presented with both intracerebral bleeding and possible thrombosis, and who responded to vitamin K supplement plus fresh frozen plasma.
A 2012 analysis of 12 patients with occult poisoning by long-acting anticoagulant rodenticides found that bleeding was controlled effectively by administering vitamin K1 daily. Laboratory examinations showed prolonged prothrombin time and activated partial prothrombin time, and there was a statistical difference between these values before and after treatment (P<0.01). The authors note that coagulation disorders from reduced vitamin K-dependent factors tend to be neglected due to hidden medical history and delayed signs of poisoning.
A 2025 report describes two Japanese adults with vitamin K-dependent coagulation factor deficiency of unidentified cause. Both showed markedly decreased levels of factors VII, IX, X, II, protein C, and protein S, with elevated protein induced by vitamin K absence/antagonist II. The clinical course ruled out congenital deficiency in both cases. The authors propose a new disease entity called idiopathic acquired VKCFD.
What is still missing is a systematic understanding of how many patients with congenital deficiency exist, whether vitamin K alone is sufficient for long-term management in that population, and a clear diagnostic pathway for idiopathic acquired cases in adults. No controlled trials have been done, no standardised dosing regimen exists, and patient stratification by underlying cause remains 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.
Congenital vitamin K-dependent coagulation factor deficiency: a case report
AbstractCongenital vitamin K-dependent coagulation factor deficiency is a very rare bleeding disorder, which usually presents with episodes of intracerebral bleed in the first few weeks of life, sometimes leading to a fatal outcome. We report a case of combined factor deficiency of vitamin K-dependent factors in which the patient presented with both intracerebral bleeding, and possibly also thrombosis, and responded to a vitamin K supplement along with fresh frozen plasma.
Clinical analysis of 12 patients caused by long-acting anticoagulant rodenticide occult poisoning.
AbstractOBJECTIVE: To explore the clinical characteristics and diagnosis and treatment of occult poisoning caused by long-acting anticoagulant rodenticides. METHOD: Records of 12 patients from July 2008 to April 2011 diagnosed as anticoagulant rodenticide occult poisoning, who had been misdiagnosed initially at other hospitals were analyzed retrospectively. Elements from the records included clinical symptoms and signs, laboratory findings for prothrombin time (PT) and activated partial prothrombin time (APTT), and initial misdiagnosis and treatment outcome at our hospital. RESULTS: The clinical presentations of patients were insidious and serious, often presented as skin ecchymose, hematuria, menorrhagia and gastrointestine bleeding. Laboratory examinations showed prolonged PT and APTT; bleeding was controlled effectively by administoring vitamin K1 daily. There were statistical difference between PT and APTT before and after the treatment (P<0.01). CONCLUSION: Coagulation disorders might be caused by the reduced acquisition in vitamin K dependent coagulation factors, which tends to be neglected due to a hidden medical history, delayed signs of poisoning, and various organs involved. A detailed patient history and systematic review may improve the diagnostic accuracy. Once diagnosed is made, vitamin K1 should be given as soon as possible.
Internal Medicine · 2025 · 0 citations · open access
Adult-onset Vitamin K-dependent Coagulation Factor Deficiency of Unidentified Etiology
AbstractSymptoms of bleeding caused by vitamin K-dependent coagulation factor deficiency (VKCFD) are rare in adults. We present two Japanese cases of adult-onset VKCFD, in which the cause of vitamin K deficiency remains unknown despite comprehensive evaluation. Both patients showed markedly decreased levels of coagulation factors (F) VII, FIX, FX, FII, protein C, and protein S, along with elevated levels of protein induced by vitamin K absence/antagonist II. The clinical course in both cases ruled out congenital VKCFD. Based on these findings and a similar case in the relevant literature, we propose a new disease entity, idiopathic acquired VKCFD.
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.
