DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for platelet-type bleeding disorder 17 — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease modulePlatelet-type bleeding disorder 17 maps to a 1-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 platelet-type bleeding disorder 17 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.
What the evidence adds up to
Platelet-type bleeding disorder 17 is not mentioned in any of the provided abstracts. The abstracts discuss inherited platelet function defects in general terms, noting that hereditary storage pool disease is common enough to be suspected in a child with characteristic findings, but that most hereditary platelet function defects are rare. No specific molecular defect, gene, or clinical trial data for any named platelet-type bleeding disorder is given.
One abstract from 2017 reports an observational study of 2,256 critically ill patients with thrombocytopenia, of whom 71 (3.1%) received platelet transfusions. Adjusted rates of major bleeding were not statistically different between patients who did and did not receive transfusions (hazard ratio 0.85; 95% confidence interval 0.42 to 1.72). The median post-transfusion platelet count increment was 20 × 10⁹/L at 3.5 hours. The authors state that inferences were limited by the small number of transfused patients and that clinical trials are needed.
A 2007 review of immune thrombocytopenic purpura in adults states that splenectomy remains the best curative treatment for chronic disease, and that other treatments such as anti-D, rituximab, or dexamethasone may allow splenectomy to be postponed if a haemostatic platelet count is attained. Mortality from bleeding may be relevant only in patients refractory to splenectomy. The review recommends treatment for patients with platelet counts below 30 × 10⁹/L or bleeding, but an aggressive approach only for counts below 20 × 10⁹/L and those refractory to splenectomy.
A 1977 review of intravascular coagulation states that if thrombocytopenia is present, heparinisation must be accompanied by platelet transfusion. A 1992 clinical review states that the mainstay of therapy for essentially all platelet function defects, if bleeding is significant, is the liberal infusion of platelet concentrates, and that acquired defects should also be managed by treating the underlying disease and discontinuing offending drugs. No abstract provides data on any specific drug repurposed for platelet-type bleeding disorder 17. What is missing is any clinical trial designed for this specific disorder, any patient stratification by genotype, and any funding for such trials.
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 · 1984 · 375 citations
Molecular Defects in Interactions of Platelets with the Vessel Wall
AbstractThe objectives of this review have been to summarize the recent research on inherited defects involving abnormal platelet function and to illustrate how studies of hemorrhagic syndromes have led to an increased understanding of the molecular events involved in platelet adhesion and aggregation. Emphasis has been placed on the two primary hemostatic reactions: the interaction of platelets with von Willebrand factor to promote adhesion to the subendothelium, and the interaction of platelets with fibrinogen to promote platelet aggregation. Even as these events are more clearly defined, new concepts of molecular structure, function, and heterogeneity are emerging, and the variety of recognized genetic defects is becoming more complex.
Current Opinion in Hematology · 2007 · 86 citations
Immune thrombocytopenic purpura in adults
AbstractPURPOSE OF REVIEW: A review of recent studies was conducted to determine if guidelines promulgated by the American Society of Hematology and the British Committee for Standards in Haematology need to be updated as these were based mainly on expert opinion rather than outcomes derived from clinical trials. RECENT FINDINGS: Recent studies suggest that most patients with immune thrombocytopenic purpura have a disease that is generally well tolerated, with little morbidity. Splenectomy remains the best 'curative' treatment for adults with chronic disease (at least 6 months of follow up). Other treatments such as anti-D, rituximab or dexamethasone may allow the decision of splenectomy to be postponed, possibly indefinitely, if hemostatic platelet count is attained. Mortality from bleeding may be relevant only in patients refractory to splenectomy. Cytotoxic agents should be reserved for patients with bleeding refractory to other treatments. SUMMARY: Patients with platelet counts less than 30 x 10(9)/l or bleeding have to be treated but management decisions should also be based on lifestyle, age, and other medical conditions that may contribute to the risk of serious bleeding. An aggressive therapeutic approach is justified only in patients with platelet counts below 20 x 10(9)/l and those refractory to splenectomy. Newer therapies may be more targeted in their action.
The platelet fibrinogen receptor: from megakaryocyte to the mortuary
AbstractPlatelets are integral to normal haemostatic function and act to control vascular haemorrhage with the formation of a stable clot. The fibrinogen receptor (glycoprotein IIb/IIIa [GPIIb/IIIa]) is the most abundant platelet integrin and, by binding fibrinogen, facilitates irreversible binding of platelets to the exposed extracellular matrix and enables the cross-linking of adjacent platelets. The vital role of GPIIb/IIIa requires tight control of both its synthesis and function. After transcription from distinct domains on chromosome 17, the two subunits of the heterodimer are carefully directed through organelles with intricate regulatory steps designed to prevent the cellular expression of a dysfunctional receptor. Similarly, exquisite control of platelet activation via bidirectional signalling acts to limit the inappropriate and excessive formation of platelet-mediated thrombus. However, the enormous diversity of genetic mutations in the fibrinogen receptor has resulted in a number of allelic variants becoming established. The Pro(33) polymorphism in GPIIIa is associated with increased cardiovascular risk due to a pathological persistence of outside-in signalling once fibrinogen has dissociated from the receptor. The polymorphism has also been associated with the phenomenon of aspirin resistance, although larger epidemiological studies are required to establish this conclusively. A failure of appropriate receptor function due to a diverse range of mutations in both structural and signalling domains, results in the bleeding diathesis Glanzmann's thrombasthaenia. GPIIb/IIIa inhibitors were the first rationally designed anti-platelet drugs and have proven to be a successful therapeutic option in high-risk primary coronary intervention. As our understanding of bidirectional signalling improves, more subtle and directed therapeutic strategies may be developed.
Seminars in Thrombosis and Hemostasis · 1992 · 38 citations
Platelet Function Defects: A Clinical Review
AbstractPlatelet dysfunction, especially acquired forms, are common causes of hemorrhage, especially in association with trauma and surgery. Although the hereditary platelet function defects are generally quite rare, hereditary storage pool disease is common enough to be suspected in an individual, usually a child, with characteristic historical and clinical findings. The acquired platelet function defects, especially those resulting from drugs, are very common and should promptly be suspected in patients developing easy and spontaneous bruising, mild to moderate mucosal membrane hemorrhage, or unexplained bleeding associated with trauma or surgery. The template bleeding time is generally useful as a screening test of platelet function, but a normal template bleeding time, in the presence of a suggestive history, suggestive clinical findings, or in the patient frankly bleeding, is not reliable and platelet aggregation or lumi-aggregation should be done in appropriate clinical situations. The mainstay of therapy for essentially all these defects, if bleeding is significant, is the liberal infusion of appropriate numbers of platelet concentrates. The acquired platelet function defects, of course, should also be managed by attempts to treat or control the underlying disease, if possible, and offending drugs or potentially offending drugs should promptly be discontinued.
Research and Practice in Thrombosis and Haemostasis · 2017 · 14 citations · open access
The association between platelet transfusions and bleeding in critically ill patients with thrombocytopenia
AbstractBackground Platelet transfusions are commonly used to treat critically ill patients with thrombocytopenia. Whether platelet transfusions are associated with a reduction in the risk of major bleeding is unknown. Patients/Methods Observational cohort study nested in a previous multicenter, randomized thromboprophylaxis trial in the intensive care unit (ICU). The objective was to evaluate the association between platelet transfusions and adjudicated major bleeding events. Platelet transfusion episodes were reviewed for timing of administration, product type, and dose. Major bleeding with and without platelet transfusions was adjusted for severity of thrombocytopenia, use of anti‐platelet agents, surgery and other covariates. Secondary outcomes were thrombosis, death in ICU and platelet count increment. Results Among 2,256 patients, 71 (3.1%) received 190 platelet transfusions. Of those, 121 (63.7%) were administered to 54 non‐bleeding, thrombocytopenic patients. Adjusted rates of major bleeding were not statistically different with or without the administration of platelet transfusions (hazard ratio for transfused patients 0.85; 95% confidence interval, 0.42‐1.72). We did not find a significant association between platelet transfusion use and thrombosis or death in ICU in adjusted analyses. Thrombocytopenia, anemia, major or minor bleeding and use of anticoagulants were associated with platelet transfusion administration. The median post‐transfusion platelet count increment was 20×10 9 /L at 3.5 hours post‐transfusion. Conclusions Rates of major bleeding were not different for patients who did and did not receive platelet transfusions. Inferences were limited by the small number of transfused patients. Clinical trials are needed to better investigate the potential hemostatic benefit and potential harms of platelet transfusions for this high‐risk population.
Scandinavian Journal of Haematology · 1981 · 14 citations
Prolonged Bleeding Time with Adequate Platelet Count in Hospital Patients
Abstract100 patients with a modified Ivy bleeding time longer than 10 min in the presence of more than 80 x 10(9)/l blood platelets were studied retrospectively. 72 patients had repeated bleeding times between 10 and 20 min or one or more measurements exceeding 20 min, and were considered to have an unquestionable platelet dysfunction. 39 (54%) of these 72 patients were receiving large doses of antibiotics. Nearly one half of the patients on antibiotics had a bleeding tendency, but most of these patients had additional features that may have interfered with platelet function, or a coagulation defect. In the remaining 33 patients, the prolonged bleeding time was associated with von Willebrand's disease, liver disease, leukaemia/myeloproliferative disease, paraproteinaemia or aspirin ingestion. High doses of penicillin seem to be the most common cause of qualitative platelet disorders in general hospital practice.
AbstractTherapy for intravascular coagulation is directed primarily against the underlying disorder. If the latter is not readily correctable or if the patient is bleeding actively, anticoagulation with intermittent administration of heparin by the intravenous route is indicated. If thrombocytopenia is present, heparinization must be accompanied by platelet transfusion. The efficacy of therapy is judged by the cessation of bleeding or thrombosis, improvement of organic dysfunction, and correction of the levels of the coagulation factors, particularly factor V and fibrinogen.
The Primary Care Companion For CNS Disorders · 2018 · 1 citations
Mirtazapine-Induced Epistaxis in an Australian Indigenous Man
AbstractArticle AbstractBecause this piece does not have an abstract, we have provided for your benefit the first 3 sentences of the full text.To the Editor: It is well known that serotonin reuptake inhibitors (SSRIs) can cause abnormal bleeding by decreasing the availability of serotonin within the platelet and, in turn, inhibiting platelet aggregation and blocking the coagulation cascade. If there is a risk of abnormal bleeding, mirtazapine and bupropion are generally considered to be safer treatment options because they lack the serotonin reuptake mechanism. We present the case of an 18-year-old North Australian indigenous man, with no prior history of bleeding disorder, who developed frequent epistaxis after starting mirtazapine.
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.
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