DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for Evans syndrome — 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 moduleEvans syndrome 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 evans syndrome 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
Evans syndrome is defined by the coexistence of autoimmune haemolytic anaemia and immune thrombocytopenia, with or without immune neutropenia, in the absence of a known underlying cause. The condition can develop spontaneously or secondary to underlying autoimmune diseases, immunodeficiency states, malignancies, or following stem cell or solid organ transplantation. A 1999 report describes three siblings diagnosed in childhood: a 4-month-old girl with a life-threatening relapsing course unresponsive to corticosteroids, intravenous gamma-globulin, thymectomy, and cyclophosphamide, who eventually responded to splenectomy. Her two brothers had milder disease that responded to corticosteroids. Cytogenetic analyses revealed a familial Y;15 translocation in all three children and their father, though the authors note the unusual finding of three siblings with the disease and no known congenital abnormality.
The disease is described as chronic and recurrent, often resistant to multiagent therapy. A 2003 case report details the fatal course of a child with chronic thrombocytopenia who presented a spontaneous intracranial haemorrhage, indicating that this potentially fatal event may occur even in children with a moderate grade of chronic thrombocytopenia. The authors call for early recognition and aggressive management of this rare complication. Two separate abstracts from 2016 and 2021 report cases of severe Evans syndrome secondary to common variable immune deficiency, presenting as altered mental status, a rare presenting sign, and highlight the difficulty in diagnosing the disorder.
Pregnancies complicated by Evans syndrome are uncommon, with only a few published cases. A 2019 report notes that management poses a therapeutic challenge because of limited options due to concerns about possible teratogenic effects of available pharmacologic agents. Across these reports, no controlled trials are described, no consistent treatment protocol emerges, and the evidence base consists entirely of case reports and small case series. What is still missing are prospective registries, standardised diagnostic criteria, and any randomised trial that could guide therapy for this rare, heterogeneous, and often treatment-resistant 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.
Journal of Pediatric Hematology/Oncology · 2017 · 39 citations
Natural History, Pathogenesis, and Treatment of Evans Syndrome in Children
AbstractPrimary Evans syndrome (ES) is defined by the concurrent or sequential occurrence of immune thrombocytopenia and autoimmune hemolytic anemia in the absence of an underlying etiology. The syndrome is characterized by a chronic, relapsing, and potentially fatal course requiring long-term immunosuppressive therapy. Treatment of ES is hardly evidence-based. Corticosteroids are the mainstay of therapy. Rituximab has emerged as the most widely used second-line treatment, as it can safely achieve high response rates and postpone splenectomy. An increasing number of new genetic defects involving critical pathways of immune regulation identify specific disorders, which explain cases of ES previously reported as "idiopathic".
Journal of Pediatric Hematology/Oncology · 1999 · 15 citations
Familial Evans Syndrome
AbstractPURPOSE: This report describes the clinical course of three siblings, all of whom had Evans syndrome in childhood. PATIENTS: The coexistence of autoimmune hemolytic anemia and thrombocytopenia, in the absence of a known underlying cause, led to the diagnosis of Evans syndrome in a 4-month-old girl and subsequently in her two brothers when they were 4 and 13 years old. RESULTS: The 4-month-old girl had a life-threatening relapsing course unresponsive to corticosteroids, intravenous gamma-globulin, thymectomy, and cyclophosphamide. She eventually responded to splenectomy. Her two brothers had milder disease that responded to corticosteroids. Cytogenetic analyses revealed the presence of a familial Y;15 translocation in all three children and their father. CONCLUSION: There are few reported cases of familial Evans syndrome, and they are usually associated with an inherited congenital abnormality. We report the unusual finding of three siblings with the disease and no known congenital abnormality.
Annals of Saudi Medicine · 2009 · 7 citations · open access
Neonatal Familial Evans Syndrome Associated with Joint Hypermobility and Mitral Valve Regurgitation in Three Siblings in a Saudi Arab Family
AbstractThe occurrence of autoimmune hemolytic anemia and immune thrombocytopenia in the absence of a known underlying cause led to the diagnosis of Evans syndrome in a 9-month-old male. Subsequently, a similar diagnosis was made in two siblings (a 3-year-old boy and a 1-day-old girl). The 9-month-old had a chronic course with exacerbations. He was treated with steroids, intravenous immunoglobulin and colchiccine with a variable response. He died of congestive heart failure at the age of 8 years. The brother's disease course was one of remission and exacerbation. With time, remissions were prolonged and paralleled an improvement in joint hypermobility. The sister died of sepsis after a chronic course with severe exacerbattions. Only two families with Evans syndrome have been reported in the English medical literature. In one report (in a Saudi Arab family), the disease was associated with hereditary spastic paraplegia.
Journal of Pediatric Hematology/Oncology · 2016 · 6 citations
Evans Syndrome Secondary to Common Variable Immune Deficiency
AbstractEvans syndrome is an underdiagnosed condition consisting of simultaneous or sequential combination of autoimmune hemolytic anemia and immune-mediated thrombocytopenia. We report a case of severe Evans syndrome presenting as altered mental status, a rare presenting sign of the disease. This case highlights the difficulty in diagnosing Evans syndrome and provides a review of the literature and management strategies for treating the disorder.
Pediatric Hematology and Oncology · 2003 · 4 citations
Spontaneous Fatal Intracranial Hemorrhage in a Child with Evans Syndrome
AbstractEvans syndrome is a chronic and recurrent disease often resistant to multiagent therapy. Spontaneous intracranial hemorrhage is a rare but life-threatening complication in children with Evans syndrome. The authors report the fatal course of a child with chronic thrombocytopenia who presented a spontaneous intracranial hemorrhage. The case indicates that this potentially fatal event may occur even in children with a moderate grade of chronic thrombocytopenia. Early recognition and aggressive management is required for an adequate management of this rare complication.
Pediatric Hematology and Oncology · 2003 · 0 citations
Spontaneous Fatal Intracranial Hemorrhage in a Child with Evans Syndrome
AbstractEvans syndrome is a chronic and recurrent disease often resistant to multiagent therapy. Spontaneous intracranial hemorrhage is a rare but life-threatening complication in children with Evans syndrome. The authors report the fatal course of a child with chronic thrombocytopenia who presented a spontaneous intracranial hemorrhage. The case indicates that this potentially fatal event may occur even in children with a moderate grade of chronic thrombocytopenia. Early recognition and aggressive management is required for an adequate management of this rare complication.
Successful Therapeutic Management and Delivery in a Pregnancy Complicated by Secondary Evans Syndrome from Systemic Lupus Erythematosus and Concurrent Antiphospholipid Syndrome
AbstractObjectives: Evans syndrome is characterized by the coexistence of autoimmune hemolytic anemia and immune thrombocytopenia and/or immune neutropenia. Its usual clinical course is chronic with relapses and may develop spontaneously or secondary to underlying autoimmune diseases, immunodeficiency states, malignancies or following stem cell or solid organ transplantation. Pregnancies complicated by Evans syndrome are uncommon with only few published cases. It poses a therapeutic challenge since we are left with limited therapeutic options due to concerns of possible teratogenic effects of current available pharmacologic agents.
Carolina Digital Repository (University of North Carolina at Chapel Hill) · 2021 · 0 citations · open access
Evans syndrome secondary to common variable immune deficiency
AbstractEvans syndrome is an underdiagnosed condition consisting of simultaneous or sequential combination of autoimmune hemolytic anemia and immune-mediated thrombocytopenia. We report a case of severe Evans syndrome presenting as altered mental status, a rare presenting sign of the disease. This case highlights the difficulty in diagnosing Evans syndrome and provides a review of the literature and management strategies for treating the disorder.
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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