DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for platelet-type bleeding disorder 14 — 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 14 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 14 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
The four abstracts provided do not describe any original research on platelet-type bleeding disorder 14. They are review articles or clinical guidance pieces. One abstract from 2011 states there are no guidelines for managing patients on antiplatelet agents who need reversal of drug effect, and the author’s approach is based solely on personal experience and very limited published data. A 2010 article on bleeding disorder in a child describes initial diagnostic work-up as prothrombin time, activated partial thromboplastin time, and blood platelet concentration, and recommends referral to a haematologist for confirmation and definitive treatment. A 2001 chapter outlines a systematic clinical approach to the bleeding patient. A 2013 article on phenotyping and genotyping of platelet disorders notes that gene mutations require phenotypic support to assign causation, citing the observational GAPP study, but gives no results specific to platelet-type bleeding disorder 14.
No concrete numbers — survival, response rates, or sample sizes — appear in any of these abstracts. No drug is mentioned by name as a treatment for platelet-type bleeding disorder 14. The abstracts contain no efficacy data, no trial results, and no evidence that any intervention has been tested in this condition.
What is still missing is any original research on platelet-type bleeding disorder 14 itself. There are no published trials, no patient stratification data, and no funding directed at this specific disorder in these abstracts. The clinical guidance that exists is generic and based on expert opinion, not on evidence from studies of this disease.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Transfusion · 2011 · 39 citations
How do I transfuse platelets (PLTs) to reverse anti‐PLT drug effect? (CME)
AbstractAntiplatelet agents (APAs) are commonly used in clinical practice to either treat or prevent arterial thrombotic disorders in patients at high risk. The newer APAs are more potent with higher bleeding risk profiles. Patients who present with serious bleeds or need urgent surgical interventions while on APAs may require reversal of these agents' effect on PLTs. Currently, there are no guidelines for management of such patients. This article describes my approach to PLT transfusion or use of pharmacologic agents in such clinical scenarios based solely on personal experience and very limited published data.
American Journal of Clinical Pathology · 1987 · 31 citations
Essential Thrombocythemia with Acquired von Willebrand’s Disease
AbstractTwo patients with essential thrombocythemia showed a marked decrease in ristocetin-induced platelet aggregation in addition to other aggregation defects during routine aggregation studies for prolonged bleeding times. Further investigation revealed type I von Willebrand's disease (vWD) in one patient and a variant of vWD compatible with type IIA defect in the second patient. The latter patient had minor episodes of epistaxis clinically, while the patient with type I disease experienced excessive blood loss during menstrual periods. Both patients gave negative history for abnormal bleeding in the past or in their families. Both have had uneventful surgical procedures in the past, at which time bleeding times and basic coagulation test results were normal.
Diagnosis and Management of Bleeding Disorder in a Child
AbstractChildren with symptoms of bleeding and bruising are commonly seen in clinical practice. Primary care providers should be able to decide when and whether evaluation for bleeding disorder is warranted. This decision depends on one's index of suspicion for bleeding disorder based on history, physical examination, and screening laboratory investigations. Knowledge of the hemostatic physiology is essential to be able to order appropriate laboratory investigations and their accurate interpretation. Prothrombin time (PT), activated partial thromboplastin time (aPTT), and blood platelet concentration constitute the initial diagnostic work up of any bleeding disorder. Abnormality in any of these parameters in a child with excessive bleeding should lead to presumptive diagnosis of bleeding disorder and trigger referral to a hematologist for confirmation and definitive treatment. Awareness of basic treatment principles for management of bleeding/clotting disorders may prepare the provider to develop appropriate management plans, especially in a life threatening situation.
Cambridge University Press eBooks · 2001 · 1 citations
Clinical approach to the bleeding patient
AbstractReduced platelet count or function is typically expressed clinically as a bleeding problem; further chapters of this book discuss platelet disorders in more detail. The clinician faced with a patient presenting hemorrhagic symptoms, however, must consider a much broader differential diagnosis than just a platelet problem. The present chapter describes a systematic approach to such a bleeding patient. This involves taking initial urgent measures if required and then performing a brief inspection for subcutaneous bleeding, taking a focused medical history, completing the physical examination, and reviewing the initial laboratory tests.
AbstractInvestigation of patients with mild bleeding disorders might provide novel information on the regulation and role of platelet proteins. It might even identify new targets for prevention of thrombosis. However, gene mutations require phenotypic support to assign causation, according to findings from the observational study, Genotyping and Platelet Phenotyping [GAPP; ISRCTN77951167; UKCRN ID 9858]. This article presents results to date from the study.
P1600: USE OF RITUXIMAB PLUS STANDARD OF CARE FOR TREATMENT OF PRIMARY IMMUNE THROMBOCYTOPENIA: A SYSTEMATIC REVIEW AND META-ANALYSIS
AbstractTopic: 32. Platelet disorders Background: Immune thrombocytopenia (ITP) is a common autoimmune disease associated with decreased platelet counts and increased risk of bleeding. The exact pathophysiology of ITP is still poorly understood, with multiple immune mediated pathways being theorized to contribute. Treatment of this condition is done with the goal of sustained recovery of platelet counts and prevention or cessation of bleeding. If treatment is deemed necessary, first line treatment consists of high dose corticosteroids or intravenous immunoglobulin. Unfortunately for many patients, these treatments produce rapid but transient effects. Rituximab, a monoclonal anti-CD20 antibody, has been shown to improve platelet count response, but evidence for sustained response beyond 6 months is limited. Aims: The objective of this systematic review and meta-analysis is to establish the effect of rituximab on short-term and sustained platelet response in adults with primary ITP. Methods: A comprehensive search of MEDLINE, Embase, and the grey literature was done from inception to September 2022. Studies were screened at abstract and full-text level independently and in duplicate. All English language randomized controlled studies investigating the use of rituximab in addition to standard of care as compared to standard of care were included. Baseline trial characteristics, rituximab dosing regimen, standard of care regimen, and follow up time were collected. Efficacy outcome measures were short-term partial and complete response, and long-term partial and complete response. Partial and complete response were defined by primary study. Short-term was defined as <3 months, whereas long term was defined as >6 months. If multiple time points were presented, longest follow up time was used in analysis. Safety outcomes included bleeding and infection as defined by primary study. Meta-analysis was done using DerSimonioan and Laird random effects model and reported as relative risks (RR) with 95% confidence intervals (CI) Results: Of the 3693 studies screened, five studies with a total of 463 unique patients were included in the analysis. Four trials used standard dosing of rituximab (375 mg/m2), while one trial used low dose rituximab (100 mg). Standard of care in each trial consisted of corticosteroids +- IVIG. Median short-term follow up was 28 days (Range 10 days, 1 month). Median long-term follow up was 12 months (range 6 months, 18 months). Complete response was defined as platelet count >100*109 platelets/L in all studies. Partial response definition varied between >30-50*109 platelets/L. Rituximab is associated with increased short-term partial response (RR 1.34, 95%CI 1.01, 1.77, I2= 68%, N=3), long-term complete response (RR 1.71, 95%CI 1.11, 2.64, I2= 48%, N=4), and long-term partial response (RR 1.58, 95%CI 1.02, 2.44, I2= 74%, N=5). There was insufficient data to analyze short-term complete response. There was no difference between rituximab and standard of care with regards to bleeding (RR 1.06, 95%CI 0.52, 2.13, I2= 3%, N=4). There was a trend towards more infections with rituximab as compared to standard of care (RR 1.41, 95%CI 0.94, 2.14, I2= 0%, N=4). Summary/Conclusion The addition of rituximab may increase both short-term and long-term platelet response rate, with no difference with regards to bleeding as compared to standard of care. There is a trend towards increased infection as compared to standard of care. Evidence regarding sustained response beyond 12 months is limited in this study. Further research including more primary data regarding long-term response and pooled analyses of long-term response are required. Keywords: Immune thrombocytopenia (ITP), Rituximab
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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