DeCure for Inherited bleeding disorder, platelet-type
DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for inherited bleeding disorder, platelet-type — screening already-approved drugs against its 3-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleInherited bleeding disorder, platelet-type maps to a 3-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 inherited bleeding disorder, platelet-type 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
CD36 molecule (CD36 blood group) (CD36) — CD36 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 plmdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 5LGD · 2.07 Å · ligand PALMITIC ACID (PLM). Experimental structure, not a prediction.
What the evidence adds up to
Inherited platelet disorders are uncommon and likely under-diagnosed, as a 2006 UK review notes they are difficult to identify and pose management problems. A 2016 diagnostic review states their real frequency is probably underestimated because many patients with excessive subcutaneous and mucosal bleeding have not been recognised. A 2025 Brazilian cohort study of 60 patients, drawn from 857 records reviewed between 1998 and 2023, found that 65% had abnormal platelet function and 35% had thrombocytopenia. The median age was 48 years, 75% were female, and a positive family history was reported in 62% of those with low platelet counts and 51% of those with function abnormalities. The bleeding phenotype was mild, with a median ISTH-BAT score of 6; patients with reduced platelet counts tended to have lower scores. Previous misdiagnoses included immune thrombocytopenia and von Willebrand disease.
No clinical trial in these abstracts tests any drug for efficacy against inherited platelet disorders. The 2017 French expert guidelines, based on literature review and clinical experience rather than controlled studies, discuss the use of platelet concentrates, tranexamic acid, desmopressin, recombinant factor VIIa, von Willebrand factor concentrates, and thrombopoietin receptor agonists for perioperative management. The guidelines explicitly state that clinical studies are scarce due to the rarity of these disorders and did not allow defining strong recommendations. The 2006 UK review similarly offers suggestions for managing bleeding manifestations, surgical interventions, and pregnancy, but provides no trial data.
The 2025 Brazilian study emphasises that identifying these disorders is essential for proper treatment and follow-up, but it is a descriptive cohort, not a treatment trial. The 2016 diagnostic algorithm presents a stepwise clinical and laboratory approach but does not report outcomes from any intervention. Across all four abstracts, there is no evidence that any drug improves survival, reduces bleeding events, or alters the natural history of inherited platelet disorders in a controlled setting.
What is still missing are adequately powered randomised trials, standardised diagnostic criteria that can be applied in low-resource settings without advanced genetic testing, and prospective data linking specific drug use to measurable clinical outcomes such as reduced bleeding or fewer transfusions. The rarity of these disorders makes trial design and funding difficult, and patient stratification by genotype or bleeding phenotype has not been validated in prospective studies.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
British Journal of Haematology · 2006 · 381 citations
A review of inherited platelet disorders with guidelines for their management on behalf of the UKHCDO
AbstractThe inherited platelet disorders are an uncommon cause of symptomatic bleeding. They may be difficult to diagnose (and are likely to be under-diagnosed) and pose problems in management. This review discusses the inherited platelet disorders summarising the current state of the art with respect to investigation and diagnosis and suggests how to manage bleeding manifestations with particular attention to surgical interventions and the management of pregnancy.
Management of invasive procedures in patients with platelet disorders or thrombocytopenia Guidelines by French expert group on inherited platelet diseases
AbstractInherited platelet disorders (thrombocytopenia, functional defects or both) are rare and extremely heterogeneous with variable risk of spontaneous bleeding, but they are frequently diagnosed after haemorrhages following a surgical procedure or a trauma. Clinical studies focusing on these disorders are scarce due to their paucity, and thus they did not allow defining strong recommendations on the haemostasis treatment of these patients. On the basis of literature review and the experience of his members, the Reference Centre on inherited platelet disorders in France provides practical recommendations on the perioperative management, according to the type of underlying disease, to the bleeding risk associated to the invasive procedure, and to the clinical history of the patient. The indication and use of platelet concentrates and of various drugs i.e. tranexamic acid, desmopressin, recombinant factor VIIa, von Willebrand factor concentrates, and thrombopoietin receptor agonists are discussed in this text.
Hematology Transfusion and Cell Therapy · 2025 · 0 citations · open access
Clinical and laboratorial characterization of a cohort of patients with hereditary platelet disorders in Brazil
AbstractINTRODUCTION: Inherited platelet disorders are rare conditions characterized by altered platelet function and/or reduced platelet counts. Diagnosing these disorders is challenging and may result in delays, misdiagnosis, and inappropriate treatment. In low- and middle-income countries, data are scarce. Here, we describe a cohort of patients at a reference center in Brazil. METHODS: A descriptive analysis was conducted on patients followed at the Thrombosis and Hemostasis outpatient clinic of the Hospital das Clinicas, University of São Paulo, Brazil.Medical records of 857 patients with thrombocytopenia or bleeding disorders of unknown cause, evaluated between 1998 and 2023, were reviewed. Of these, 60 patients had a confirmed or suspected diagnosis of an inherited platelet disorder and were included in the study. RESULTS: Among the 60 patients, the majority were female (75 %), with a median age of 48 years. The suspicion of a platelet disorder was based on clinical presentation, family history, and laboratory findings. Overall, 65 % of the patients had abnormal platelet function, while 35 % presented with thrombocytopenia. A positive family history was reported in 62 % of those with low platelet counts and in 51 % of patients with platelet function abnormalities. Previous misdiagnoses included immune thrombocytopenia and von Willebrand disease. Overall, the bleeding phenotype was mild, with a median ISTH-BAT (International Society on Thrombosis and Haemostasis Bleeding Assessment Tool) score of 6. Patients with reduced platelet counts tended to have lower ISTH-BAT score. CONCLUSIONS: Identifying inherited platelet disorders is essential for proper treatment and follow-up. This study emphasizes the need for careful assessment of family history, bleeding risk, platelet count, morphology, and function for diagnosis, particularly in low-resource settings without access to advanced genetic testing.
Slovenian Medical Journal · 2016 · 0 citations · open access
Sodobni pristop za diagnosticiranje prirojenih motenj delovanja trombocitov
AbstractInherited platelet function disorders (IPFD) comprise a heterogeneous group of diseases. Their real frequency is probably underestimated as in many patients with excessive subcutaneous and mucosal bleedings the disorder has not been recognized. The presented diagnostic recommendations are based on a systematic review of the scientific literature and describe the role of platelets in the clotting process and the most common congenital disorders of platelet function. A diagnostic algorithm for clinical and laboratory stepwise management of patients with IPFD is presented.
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.
