DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for Quebec platelet disorder — 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 moduleQuebec platelet disorder 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
approvedAminocaproic AcidApproved drug
Structures already discussed alongside quebec platelet disorder in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
B1-8 FV fragment — Aminocaproic Acid has a real, experimentally solved structure in complex with this target (PDB 1A6V, 1.8 Å). This is the drug's own deposited structure, not a prediction, and confirms it is a structurally characterised molecule rather than an untested guess.
Loading structure…
helix sheet npcdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 1A6V · 1.8 Å · ligand Aminocaproic Acid (NPC). Experimental structure, not a prediction.
What the evidence adds up to
Quebec platelet disorder is a rare inherited thrombopathy characterised by severe bleeding after trauma or surgery despite normal platelet counts, and none of the five abstracts provided concerns this condition directly. The 2010 randomised trial of romiplostim enrolled 234 adults with immune thrombocytopenia, not Quebec platelet disorder, and found a platelet response rate 2.3 times that of standard care (95% CI 2.0 to 2.6), with treatment failure in 11% of romiplostim patients versus 30% on standard care, and splenectomy in 9% versus 36%. Serious adverse events occurred in 23% of romiplostim patients and 37% of standard-care patients. This evidence cannot be extrapolated to Quebec platelet disorder, where platelet counts are typically normal and the defect is in platelet function and granule content, not production.
The 1980 report on aminocaproic acid describes 13 patients with amegakaryocytic thrombocytopenia, a condition of absent megakaryocytes, not Quebec platelet disorder. Four patients on long-term therapy had striking reductions in platelet transfusion requirements for capillary bleeding, with orthostatic hypotension as the only noted adverse effect. The authors could not demonstrate quantitative platelet function changes, and no data exist from this paper on the specific platelet storage pool defect seen in Quebec platelet disorder. The 2016 case report concerns a single patient with aplastic anaemia and placenta previa managed surgically, and the 1999 study on gentamicin-associated thrombocytopenia reports drug-dependent platelet antibodies in 4% of 659 eligible hospitalised patients, rising to 7.7% among those with platelet count changes; neither has any bearing on Quebec platelet disorder.
The 2025 differential diagnosis review confirms that thrombocytopenia is defined as a platelet count below 150,000/μL and lists immune, drug-induced, infectious, and haematologic causes, but Quebec platelet disorder does not typically present with thrombocytopenia and is not mentioned in any abstract. No abstract in this set tests a drug in Quebec platelet disorder, reports a response rate for it, or provides any survival or bleeding-outcome data specific to that disease. What is missing is any clinical trial of antifibrinolytic agents, thrombopoietin receptor agonists, or other haemostatic drugs in patients with Quebec platelet disorder, along with validated bleeding-assessment tools and patient stratification by genetic status; without such studies, no drug can be claimed to alter the natural history of this condition.
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 · 2010 · 424 citations · open access
Romiplostim or Standard of Care in Patients with Immune Thrombocytopenia
AbstractBACKGROUND: Romiplostim, a thrombopoietin mimetic, increases platelet counts in patients with immune thrombocytopenia, with few adverse effects. METHODS: In this open-label, 52-week study, we randomly assigned 234 adult patients with immune thrombocytopenia, who had not undergone splenectomy, to receive the standard of care (77 patients) or weekly subcutaneous injections of romiplostim (157 patients). Primary end points were incidences of treatment failure and splenectomy. Secondary end points included the rate of a platelet response (a platelet count >50×10(9) per liter at any scheduled visit), safety outcomes, and the quality of life. RESULTS: The rate of a platelet response in the romiplostim group was 2.3 times that in the standard-of-care group (95% confidence interval [CI], 2.0 to 2.6; P<0.001). Patients receiving romiplostim had a significantly lower incidence of treatment failure (18 of 157 patients [11%]) than those receiving the standard of care (23 of 77 patients [30%], P<0.001) (odds ratio with romiplostim, 0.31; 95% CI, 0.15 to 0.61). Splenectomy also was performed less frequently in patients receiving romiplostim (14 of 157 patients [9%]) than in those receiving the standard of care (28 of 77 patients [36%], P<0.001) (odds ratio, 0.17; 95% CI, 0.08 to 0.35). The romiplostim group had a lower rate of bleeding events, fewer blood transfusions, and greater improvements in the quality of life than the standard-of-care group. Serious adverse events occurred in 23% of patients (35 of 154) receiving romiplostim and 37% of patients (28 of 75) receiving the standard of care. CONCLUSIONS: Patients treated with romiplostim had a higher rate of a platelet response, lower incidence of treatment failure and splenectomy, less bleeding and fewer blood transfusions, and a higher quality of life than patients treated with the standard of care. ( ClinicalTrials.gov number, NCT00415532.).
AbstractThe bleeding complications of some forms of thrombocytopenia are difficult to control. Many patients become refractory to platelet transfusions even when HLA-matched. We have successfully used aminocaproic acid to control bleeding in 13 patients with amegakaryocytic thrombocytopenia. Four patients receiving long-term therapy with this drug had striking reductions in the number of platelet transfusions required for capillary bleeding. No adverse effects have been noted save for orthostatic hypotension, which is ameliorated by a reduction in dosage. Quantitative platelet function changes have been impossible to demonstrate, but no changes were noted in four patients with normal platelet counts who were receiving high-dose aminocaproic acid for treatment of subarachnoid hemorrhage. Aminocaproic acid has proved to be a valuable agent in the management of patients with amegakaryocytic thrombocytopenia, especially in decreasing the need for platelet transfusions. (<i>JAMA</i>243:35-37, 1980)
Clinical and Experimental Obstetrics & Gynecology · 2016 · 3 citations · open access
Cesarean delivery via a transverse uterine fundal incision for the successful management of a low-lying placenta and aplastic anemia
AbstractPURPOSE: To present a case report on the successful management of a low-lying placenta and aplastic anemia. Aplastic anemia is a rare but serious disorder that is often characterized by severe pancytopenia. Because of the rarity of aplastic anemia, a pregnancy complicated by it is rarely encountered by obstetricians. Moreover, placenta previa (low-lying placenta) complicated by aplastic anemia has not been previously reported. MATERIALS AND METHODS: The authors present the first reported case of placenta previa with aplastic anemia in a patient who had undergone a previous cesarean delivery. RESULTS: They successfully managed this case by making a transverse uterine fundal incision during an elective cesarean delivery. This incision minimized blood loss and enabled good visualization of the source of bleeding in the lower uterine segment. Bleeding was stemmed by suturing the source of bleeding. CONCLUSION: The authors propose that this procedure should be considered for patients with low platelet counts and abnormal placentation.
Immunohematology · 1999 · 2 citations · open access
Frequency of thrombocytopenia associated with gentamicin therapy
AbstractMany drugs, including antibiotics such as gentamicin, have been associated with the development of a drug-induced thrombocytopenia. Serologic methods for detection of drug-dependent platelet antibodies (DDPAs) are not routinely performed, and the incidence of such antibody-mediated thrombocytopenia is not known. As we routinely perform solid-phase red cell adherence assays for the detection of DDPAs, a study was designed to determine the incidence of gentamicin- associated platelet antibodies (GAPAs) in our institution. Adult patients who received gentamicin from 1/1/98 to 7/31/98 were evaluated for inclusion in the study. Testing for the presence of GAPAs was performed if the patient had a decrease in platelet count while receiving gentamicin or if the platelet count increased or decreased within 3 days of the last gentamicin therapy. Patients receiving gentamicin without development of thrombocytopenia were tested as controls. During the study period, 926 patients received gentamicin, with 324 (35%) being evaluated for the presence of GAPAs; GAPAs were identified in 25 of 659 patients (4%) eligible for the study. All control samples were found to lack GAPAs. If only patients exhibiting changes in platelet counts are considered, the incidence increases to 7.7 percent, with females apparently being almost twice as likely to develop GAPAs than are males. Gentamicin-associated thrombocytopenia is not an infrequent occurrence in hospitalized patients.
Deutsches Ärzteblatt international · 2025 · 1 citations
The differential diagnosis of thrombocytopenia
AbstractBACKGROUND: Thrombocytopenia is defined as a platelet count below 150 000/μL. It increases the risk of bleeding, often due to an existing underlying condition. A meticulous diagnostic evaluation is needed so that specific treatment can be initiated and complications avoided. METHODS: This review is based on clinical studies up to June 2025 that were retrieved by a selective search with pertinent key words in the MEDLINE/PubMed database, and on current guidelines. RESULTS: Thrombocytopenia often arises in association with pregnancy (7-10 %), immune-mediated diseases such as idiopathic thrombocytopenic purpura (ITP), certain drugs (e.g., heparin-induced thrombocytopenia [HIT] in as many as 1% of patients treated with unfractionated heparin), infections, and systemic and hematologic diseases. The diagnostic evaluation is by an algorithm involving the clinical history, a complete blood count, and other, specific tests. Emergencies such as thrombotic microangiopathy (TMA) and disseminated intravascular coagulation require rapid therapeutic intervention. The treatment depends on the cause and severity of thrombocytopenia; it can include the discontinuation of precipitating drugs, the use of immunosuppressive drugs or thrombopoietin receptor agonists in immune-mediated cases, and specific measures against infection, TMA, or malignant diseases. Prophylactic platelet transfusion can be considered if the platelet count drops below 10 000-20 000/μL, or below 50 000/μL before an intervention or operation, but not in cases of ITP or thrombotic thrombocytopenic purpura (TTP). CONCLUSION: The treatment depends on the clinical manifestations, platelet count, and underlying cause.
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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