A púrpura trombocitopênica trombótica (PTT) é uma forma agressiva e com risco de vida de microangiopatia trombótica (TMA), caracterizada por trombocitopenia periférica profunda, anemia hemolítica microangiopática (MAHA) e falência de órgãos de gravidade variável e é composta por PTT congênita e PTT adquirida.
Introdução
O que você precisa saber de cara
A púrpura trombocitopênica trombótica (PTT) é uma forma agressiva e com risco de vida de microangiopatia trombótica (TMA), caracterizada por trombocitopenia periférica profunda, anemia hemolítica microangiopática (MAHA) e falência de órgãos de gravidade variável e é composta por PTT congênita e PTT adquirida.
Escala de raridade
<1/50kMuito rara
1/20kRara
1/10kPouco freq.
1/5kIncomum
1/2k
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Sinais e sintomas
O que aparece no corpo e com que frequência cada sintoma acontece
Partes do corpo afetadas
+ 27 sintomas em outras categorias
Características mais comuns
Os sintomas variam de pessoa para pessoa. Abaixo estão as 55 características clínicas mais associadas, ordenadas por frequência.
Linha do tempo da pesquisa
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Genética e causas
O que está alterado no DNA e como passa nas famílias
Genes associados
1 gene identificado com associação a esta condição. Padrão de herança: Autosomal recessive, Multigenic/multifactorial.
Cleaves the vWF multimers in plasma into smaller forms thereby controlling vWF-mediated platelet thrombus formation
Secreted
Thrombotic thrombocytopenic purpura, hereditary
An autosomal recessive hematologic disease characterized by hemolytic anemia with fragmentation of erythrocytes, thrombocytopenia, diffuse and non-focal neurologic findings, decreased renal function and fever.
Variantes genéticas (ClinVar)
209 variantes patogênicas registradas no ClinVar.
Classificação de variantes (ClinVar)
Distribuição de 11 variantes classificadas pelo ClinVar.
Vias biológicas (Reactome)
5 vias biológicas associadas aos genes desta condição.
Diagnóstico
Os sinais que médicos procuram e os exames que confirmam
Tratamento e manejo
Remédios, cuidados de apoio e o que precisa acompanhar
Onde tratar no SUS
Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)
🇧🇷 Atendimento SUS — Púrpura trombocitopênica trombótica
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Outros ensaios clínicos
70 ensaios clínicos encontrados, 20 ativos.
Publicações mais relevantes
Mostrando amostra de 200 publicações de um total de 2.502
Systematic Literature Review of the Economic and Quality of Life Burden of Congenital and Immune-Mediated Thrombotic Thrombocytopenic Purpura.
Thrombotic thrombocytopenic purpura (TTP) is a life-threatening blood disorder resulting from ADAMTS13 deficiency, caused by mutations in the ADAMTS13 gene in congenital TTP (cTTP), and by neutralizing antibodies against ADAMTS13 in immune-mediated TTP (iTTP). This systematic review summarizes available economic evaluations, healthcare resource utilization (HCRU), cost, utility, and health-related quality of life (HRQoL) literature in TTP, with a focus on cTTP. Given the limited availability of cTTP-specific data, broader TTP evidence was also reviewed to infer potential implications for cTTP where appropriate. Three systematic literature searches were conducted in January 2024 in accordance with PRISMA guidelines, each tailored to identify studies reporting economic evaluations, HCRU/cost data, and utility/HRQoL data respectively. For the economic evaluation and utility/HRQoL reviews, inclusion criteria were broadened to include studies reporting data on iTTP and unspecified TTP from which findings for cTTP were inferred. In total 431, 989, and 849 records were identified for the economic evaluation, HCRU/cost, and utility/HRQoL reviews, respectively. No economic evaluations were identified for treatments in cTTP populations. Eight economic studies (all in iTTP) were included. Overall, 5 studies found caplacizumab to be cost-effective, whereas 3 did not, highlighting variability across model assumptions and healthcare settings. Six HCRU/cost studies reported that cTTP was associated with frequent hospital interactions, missed work time, and high costs. Fourteen studies (1 in cTTP, 10 in iTTP, and 3 in unspecified TTP) were included in the utility/HRQoL review and showed that TTP is associated with reduced utility scores and lower HRQoL, irrespective of TTP subtype. These findings underscore the meaningful patient burden observed across the TTP spectrum and highlight the need for further research to evaluate the cost-effectiveness of emerging therapies in cTTP. However, the predominance of iTTP data reflects ongoing challenges in evidence generation for rare TTP subtypes such as cTTP.
Thrombotic thrombocytopenic purpura: celebrating 25 years of ADAMTS13.
Thrombotic Thrombocytopenic Purpura (TTP) was first described just over a century ago and it is now 25 years since the identification of ADAMTS13 as the enzyme deficient in both antibody-mediated immune TTP and congenital TTP. The discovery of ADAMTS13 has been fundamental to the vast improvement seen in TTP outcomes. Understanding the interaction between ADAMTS13, platelets and vWF led to development of clinical ADAMTS13 assays and therefore quicker and accurate diagnosis, but also, critically, to novel therapies and monitoring of treatment. Landmark additions to immune TTP therapy have included anti-CD20 treatment with rituximab, in both the acute and elective setting and the use of the nanobody caplacizumab in acute TTP. In congenital TTP, the use of ADAMTS13 replacement is playing a role in reducing end-organ damage and morbidity, with recombinant ADAMTS13 now representing the gold standard for cTTP. The ability to measure response to treatment by monitoring ADAMTS13 activity has underpinned these treatment advances and allowed clinicians to tailor immunosuppressive treatment for iTTP and rADAMTS13 dosing in cTTP. Looking forward, there are many avenues for future development with potential expansion of recombinant ADAMTS13 to treat immune TTP, new, quicker assays to improve diagnosis, monitoring and immunomodulatory therapeutic advancement, all underpinned by ADAMTS13. Future endeavors for the role of ADAMTS13 in other thrombotic indications opens further exciting opportunities.
Reinventing the wheel in ischemic stroke: Novel aspects in ADAMTS13 and von Willebrand axis.
A disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS13), is an enzyme that cleaves ultra-large von Willebrand factor (VWF) multimers. ADAMTS13 deficiency leads to thrombotic thrombocytopenic purpura (TTP), a thrombotic microangiopathy characterized by micro-thrombosis in small vessels and target-organ damage. Given that in long-term TTP survivors, reduced ADAMTS13 activity has been associated with the development of major cardiovascular events, the emerging evidence regarding the dysregulation of the ADAMTS13/ VWF axis in the pathogenesis of ischemic stroke (IS), and the unmet need for novel predictive models for IS outcomes and novel targeted therapeutics, we aim to review the current literature to provide an overview of the ongoing research on this field. Our objective is to summarize the evidence from experimental and clinical studies to underline the pathogenetic and therapeutic potential of VWF-ADAMTS13 in IS, while also providing perspectives for future research in this domain.
Revisiting Clinical Response and Refractoriness in Immune Thrombotic Thrombocytopenic Purpura.
Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening condition. Caplacizumab substantially shortens the time to clinical response, yet delayed platelet count recovery is occasionally observed, raising concerns about iTTP refractoriness. This retrospective multicenter study analyzed 204 acute iTTP episodes reported to the German REACT-2020 and Austrian ATMAR registries, all treated with caplacizumab. Refractoriness was assessed using the 2017 International Working Group criteria and the more stringent definition by the French Reference Center for Thrombotic Microangiopathies. We evaluated time to platelet recovery and presence of confounding clinical conditions, potentially accounting for persistent thrombocytopenia, in all episodes. By day 5 after caplacizumab initiation, 83.8% of patients (171/204) achieved a clinical response, and the remaining 16.2% (33/204) showed at least a doubling of the platelet count. Only three patients (1.5%) met laboratory criteria for refractoriness. In all cases, plausible alternative causes were present (e.g., missed doses, infection). No patient was refractory without a confounding factor. In 8/204 patients (3.9%) we observed a markedly prolonged thrombocytopenia (≥10 days) and identified confounding conditions in all cases. In the stratified Cox model, the presence of alternative causes of thrombocytopenia was the only independent determinant of delayed platelet count normalization (HR 0.16, 95% CI 0.09-0.28, p < 0.001), whereas baseline parameters were not predictive. In the context of caplacizumab-based therapy, true refractoriness is rare. Delayed platelet count recovery is predominantly attributable to concomitant clinical conditions. Careful clinical assessment and context-sensitive interpretation of treatment response before escalating iTTP-specific therapy may avoid unnecessary treatment intensification and associated risks.
Caplacizumab as an Emerging Treatment for Patients With Thrombotic Thrombocytopenic Purpura Presenting With Acute Ischemic Stroke.
Thrombotic thrombocytopenic purpura (TTP) is rare, life-threatening autoimmune disorder characterized by microvascular thrombosis, severe thrombocytopenia, and hemolytic anemia. It can lead to organ ischemia and increase the risk of thromboembolic events, including acute ischemic stroke (AIS). Caplacizumab, an essential adjunct in TTP management, rapidly inhibits platelet aggregation and prevents disease progression. We present 3 cases of TTP diagnosed in patients with AIS. Treatment included plasma exchange (PLEX), corticosteroids, and caplacizumab. All 3 patients exhibited acute neurologic deficits, with brain MRI confirming AIS. Laboratory tests revealed thrombocytopenia, hemolytic anemia, and ADAMTS-13 activity <1%, confirming TTP. Two patients initially treated only with PLEX and corticosteroids experienced thrombocytopenia exacerbation, requiring caplacizumab for stabilization. The third patient, treated with caplacizumab from stroke onset, maintained stable platelet counts without exacerbation. No adverse events or deaths occurred, emphasizing caplacizumab's role in sustained hematologic recovery. This case series underscores caplacizumab's potential role in stabilizing platelet counts, reducing exacerbation rates, and improving clinical outcomes in TTP-associated AIS. Although all patients experienced favorable neurologic outcomes, a faster recovery cannot be directly attributed to caplacizumab given the multimodal treatment approach. These findings are suggestive of its early use as first-line adjunct, potentially optimizing treatment strategies and improving prognosis.
Publicações recentes
[Caplacizumab combined with therapeutic plasma exchange, glucocorticoids, and rituximab for refractory thrombotic thrombocytopenic purpura: a case report and literature review].
Establishing reverse chimeric antigen receptor T cells for precise targeting of immunemediated thrombotic thrombocytopenic purpura.
Acquired Thrombotic Thrombocytopenic Purpura with a Poor Response to Treatment can be an Initial Manifestation of Occult Signet Ring Cell Carcinoma and other Malignancies.
N-Acetylcysteine Therapy in Thrombotic Thrombocytopenic Purpura: A Systematic Review and Critical Appraisal.
Precision medicine in thrombotic thrombocytopenic purpura: a narrative review.
📚 EuropePMC4.174 artigos no totalmostrando 197
1H, 13C, and 15N backbone resonance assignments of the A2 domain of human von Willebrand factor.
Biomolecular NMR assignmentsA Retrospective Analysis of ADAMTS13 Enzymatic Activity Results in a Population With Thrombotic Thrombocytopenic Purpura Disease in the Arabian Gulf and Colombia (APOLO Study).
CureusBeyond HELLP: An Unusual Cause of Severe Thrombocytopenia in Pregnancy.
CureusThrombotic thrombocytopenic purpura in pregnancy: Lessons from a case series of three patients.
The Journal of international medical researchThrombotic microangiopathy with features of thrombotic thrombocytopenic purpura in a patient with Vibrio parahaemolyticus bacteremia: a rare case report.
Internal and emergency medicineA 35-year-old man with immune thrombotic thrombocytopenic purpura presenting with dark urine.
CMAJ : Canadian Medical Association journal = journal de l'Association medicale canadienneThrombotic thrombocytopenic purpura following one anastomosis gastric bypass surgery: a rare case report.
International journal of surgery case reportsCase Report: Endothelial-targeted bridging therapy for a TTP-like phenotype in fulminant iMCD-TAFRO.
Frontiers in immunologyDynamic Monitoring of ADAMTS-13 Activity for Differential Diagnosis Across the Spectrum of Sepsis-Associated Thrombotic Microangiopathies.
Mediterranean journal of hematology and infectious diseasesSystematic Literature Review of the Economic and Quality of Life Burden of Congenital and Immune-Mediated Thrombotic Thrombocytopenic Purpura.
Journal of blood medicineThe interface of hemostasis and inflammation: endothelial-platelet dynamics in thrombosis.
Current opinion in hematologyA red blood cell-based antigen delivery system to facilitate T cell epitope presentation to promote peripheral tolerance to ADAMT S13 in immune-mediated thrombotic thrombocytopenic purpura.
Research and practice in thrombosis and haemostasisHereditary Thrombotic Thrombocytopenic Purpura Associated With Recurrent Strokes and Prominent Nervous System Involvement in a Young Chinese Female.
Clinical case reportsMultiphasic Infarcts in a Young Brain Secondary to Thrombotic Thrombocytopenic Purpura: Looking Beyond the Usual Suspects.
Annals of Indian Academy of NeurologyThrombotic thrombocytopenic purpura: celebrating 25 years of ADAMTS13.
BloodMeasuring the neuropsychological consequences of thrombotic thrombocytopenic purpura: a rapid literature review.
Hematology (Amsterdam, Netherlands)Effectiveness of caplacizumab in the first-line treatment for acquired thrombotic thrombocytopenic purpura: single center experience.
Therapeutic advances in hematologyTriple therapy with caplacizumab and rituximab for refractory systemic lupus erythematosus-associated thrombotic thrombocytopenic purpura achieves rapid, durable remission: A case report.
Modern rheumatology case reportsReinventing the wheel in ischemic stroke: Novel aspects in ADAMTS13 and von Willebrand axis.
Blood reviewsThrombotic thrombocytopenic purpura as a complication of pembrolizumab: a case report and literature Review.
Annals of hematologyPregnancy-Associated Thrombotic Thrombocytopenic Purpura: Diagnostic Pitfalls, Therapeutic Strategies, and Emerging Paradigms.
BiomedicinesCerebral Fat Embolism Syndrome in Homozygous Sickle Cell Disease Treated With Therapeutic Plasma Exchange and Simple Transfusions.
Journal of clinical apheresisLate Onset of Congenital TTP: Case Presentation and Review of the Literature.
Clinical medicine insights. Case reportsRecombinant ADAMTS13: An Enzyme Replacement Therapy for the Management of Congenital Thrombotic Thrombocytopenic Purpura.
Journal of the advanced practitioner in oncologyGenetics of thrombotic thrombocytopenic purpura: systematic review in immune and congenital thrombotic thrombocytopenic purpura.
Journal of thrombosis and haemostasis : JTHCaplacizumab resistance in immune thrombotic thrombocytopenic purpura is associated with a von Willebrand factor A1 domain missense variant.
Journal of thrombosis and haemostasis : JTHHematologic safety of caplacizumab in immune-mediated thrombotic thrombocytopenic purpura: insights from platelet-related signal detection in the FAERS database.
Thrombosis journalIs congenital thrombotic thrombocytopenic purpura a risk factor for ischemic osteonecrosis of the hip joint (Legg-Calvé-Perthes disease)?
Thrombosis journalC3 mutation-associated atypical hemolytic uremic syndrome with severe renal dysfunction and hypertensive emergency successfully treated with ravulizumab and sacubitril/valsartan: a case report.
BMC nephrologyLinking preclinical models to clinical realities: VEGF/VEGFR inhibitors and thrombotic microangiopathy in cancer therapy.
iMetaOmicsImpact of Day of Hospital Admission and Hospital Characteristics on Mortality in Thrombotic Thrombocytopenic Purpura.
Journal of clinical apheresisRevisiting Clinical Response and Refractoriness in Immune Thrombotic Thrombocytopenic Purpura.
BloodHypertensive Havoc: When Malignant Hypertension Mimics Thrombotic Thrombocytopenic Purpura.
CureusOptimizing the management of congenital thrombotic thrombocytopenic purpura.
Research and practice in thrombosis and haemostasisEfficacy of Plasma Exchange in Secondary Thrombotic Microangiopathy: A Case Report and Literature Review.
CureusAcute kidney injury requiring temporary hemodialysis due to HELLP syndrome: a case report.
CEN case reports[Immune-mediated thrombotic thrombocytopenic purpura successfully diagnosed and treated through repeated ADAMTS13 inhibitor testing].
[Rinsho ketsueki] The Japanese journal of clinical hematologyPathogen-reduced plasma, cryoprecipitate reduced for therapeutic plasma exchange.
TransfusionBone marrow proteomic profiling reveals TMEM109 as a biomarker for relapse in thrombotic thrombocytopenic purpura.
Journal of thrombosis and haemostasis : JTHExperience in emergency management of first-episode immune thrombotic thrombocytopenic purpura over the past 21 years: a single-center retrospective study.
Frontiers in immunology[Management of thrombotic microangiopathy].
Revue medicale suisse[Microangiopathies thrombotiques].
La Revue du praticienMonitoring ADAMTS-13 conformation in immune-mediated thrombotic thrombocytopenic purpura: toward personalized management.
Research and practice in thrombosis and haemostasisEndothelial injury to cognitive decline: a 12-month follow-up using computed tomography perfusion and diffusion magnetic resonance imaging in immune-mediated thrombotic thrombocytopenic purpura.
Journal of thrombosis and haemostasis : JTHThrombotic thrombocytopenic purpura in a patient with systemic lupus erythematosus after anifrolumab: a possible association.
Archive of clinical casesThrombotic thrombocytopenic purpura: a Trojan horse of acute leukemia?: a case report.
Journal of medical case reportsAcquired thrombotic thrombocytopenic purpura and HIV infection: a case report and review of the literature.
Annals of hematologyHealth-related quality of life and mental health in autoimmune thrombotic thrombocytopenic purpura patients in the caplacizumab era.
Research and practice in thrombosis and haemostasisCaplacizumab as an Emerging Treatment for Patients With Thrombotic Thrombocytopenic Purpura Presenting With Acute Ischemic Stroke.
Neurology. Clinical practiceBrazilian Registry of Thrombotic Thrombocytopenic Purpura: A prospective cohort study of diagnosis, management and outcomes in Brazil.
Thrombosis researchPostpartum Readmission for Pregnancy-Associated Thrombotic Microangiopathy Is Associated With Poor Maternal Outcomes.
Journal of clinical apheresisBilious Pleural Effusion Following Cholecystostomy in an Immunocompromised Elderly Male.
European journal of case reports in internal medicineThrombotic Microangiopathy Secondary to Capnocytophaga Sepsis: A Case Report.
CureusDiagnostic and therapeutic challenges into snakebite-induced thrombotic microangiopathy: a case report and review of the literature.
Journal of medical case reportsDiagnostic Challenges in Atypical Hemolytic Uremic Syndrome: A Case of Artificial Mitral Valve Dysfunction as a Possible Trigger for Severe Thrombotic Microangiopathy.
The American journal of case reportsEthnicity affects relapse-free survival in immune-mediated thrombotic thrombocytopenic purpura.
HaematologicaAcute Kidney Injury in Immune-Mediated Thrombotic Thrombocytopenic Purpura - Solving the Puzzle?
Kidney international reportsMechanisms of immune modulation by therapeutic plasma exchange.
Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for HaemapheresisLyme disease as a rare trigger for autoimmune hemolytic anemia.
BMC infectious diseasesTherapeutic plasma exchangerelated complications in patients with immune-mediated thrombotic thrombocytopenic purpura.
Blood transfusion = Trasfusione del sangueAn updated systematic review and meta-analysis of Caplacizumab for immune thrombotic thrombocytopenic purpura: Insights into efficacy and safety.
Blood reviewsAtypical Hemolytic Uremic Syndrome After Post-Abortion Infection: Case Report and Literature Review.
International journal of women's healthCaplacizumab in Lupus-Associated Thrombotic Thrombocytopenic Purpura (TTP): Navigating Autoimmunity and Microangiopathy.
CureusGenotype-Phenotype Variability in Congenital Thrombotic Thrombocytopenic Purpura (TTP): Two Novel ADAMTS13 Variants.
CureusRituximab in the Intensive Care Unit: A Review of Indications and Clinical Considerations.
Journal of intensive care medicineBabesiosis precipitating immune thrombotic thrombocytopenic purpura.
BloodPost-miscarriage Complement-Mediated Thrombotic Microangiopathy in a 27-Year-Old Woman: A Case Highlighting Diagnostic and Therapeutic Gaps in Brazil.
CureusImproving the PLASMIC score with the use of RDW-SD in the differential diagnosis for thrombotic thrombocytopenic purpura in Chinese patients.
Annals of clinical biochemistryThrombotic thrombocytopenic purpura following allogeneic hematopoietic stem cell transplantation: a rare but fatal complication.
Journal of thrombosis and haemostasis : JTHIn patients with suspected thrombotic thrombocytopenic purpura, what is the optimal time to therapeutic plasma exchange?
Hematology, transfusion and cell therapyHemolytic Uremic Syndrome Complicated by Severe Neuropsychiatric Symptoms: A Case Report and Review of the Literature.
CureusRisk factors for silent cerebral infarction in immune-mediated thrombotic thrombocytopenic survivors.
British journal of haematologyContinuity of care in immune thrombotic thrombocytopenic purpura (iTTP): A visual roadmap for survivorship beyond acute management.
TransfusionTailored treatment of acute immune-mediated thrombotic thrombocytopenic purpura.
Hematology. American Society of Hematology. Education ProgramChallenges and opportunities in the long-term management of immune-mediated TTP.
Hematology. American Society of Hematology. Education ProgramModern management of congenital thrombotic thrombocytopenic purpura (cTTP).
Hematology. American Society of Hematology. Education ProgramUpdate in the diagnosis of complement-mediated thrombotic microangiopathy/atypical hemolytic uremic syndrome.
Hematology. American Society of Hematology. Education ProgramA practical approach to immune thrombocytopenia in pregnancy.
Hematology. American Society of Hematology. Education ProgramEASIX, a new tool to predict response and refractoriness in immune-mediated thrombotic thrombocytopenic purpura.
Frontiers in immunologyImmune-mediated thrombotic thrombocytopenic purpura as a model of systemic microvascular dysfunction: moving from an acute to a chronic disorder.
Journal of thrombosis and haemostasis : JTHPredicting Outcomes and Optimizing Therapy in Thrombotic Thrombocytopenic Purpura: Insights on Caplacizumab Use from a Romanian Hematology Center.
Journal of clinical medicineThrombotic Thrombocytopenic Purpura During Anti-Tuberculosis Therapy: A Case Report and Literature Review.
Infection and drug resistanceBicytopenia with haemolysis: Think severe vitamin B12 deficiency-associated thrombotic microangiopathy.
Tropical doctorOcular manifestations of primary thrombotic microangiopathies : a descriptive systematic review.
BMC ophthalmologySevere thrombocytopenia and neurological symptoms in pregnancy: a diagnostic challenge between hemolysis, elevated liver enzymes, and low platelet syndrome and thrombotic thrombocytopenic purpura-what is your diagnosis?
Journal of the Turkish German Gynecological AssociationNovel cryptic ADAMTS13 epitopes uncover a distinct open ADAMTS13 conformation in immune-mediated TTP.
HaematologicaIKAROS Associated Immunodeficiency and Thrombotic Thrombocytopenic Purpura.
Pediatric blood & cancer"Recombinant a disintegrin-like and metalloproteinase with thrombospondin type 1 motifs 13 (ADAMTS-13) for acute and prophylactic treatment of congenital thrombotic thrombocytopenic purpura in pregnancy": comment.
Journal of thrombosis and haemostasis : JTHvon Willebrand factor and fibrin monomer - induced septic shock coagulation typing: Clinical comparison between thrombotic thrombocytopenic purpura - like syndrome and sepsis - induced coagulopathy with prognostic implications.
Thrombosis researchMalignant Hypertension-Induced Thrombotic Microangiopathy Mimicking Atypical Hemolytic Uremic Syndrome and Thrombotic Thrombocytopenic Purpura.
JACC. Case reportsCommon features of rare disease patients in the emergency department: a systematised literature review.
Orphanet journal of rare diseasesRecurrent Thrombocytopenia During Caplacizumab Therapy in Acute Immune-Mediated Thrombotic Thrombocytopenic Purpura.
American journal of hematologyA Case Report of Thrombotic Thrombocytopenic Purpura.
CureusA Case of Piperacillin-Induced Immune Thrombocytopenia: Diagnostic Challenges and Management.
CureusAn Unusual Case of Nonneutropenic Fever Associated With Myelodysplasia and Sweet's Syndrome.
CureusDanazol and cost-savings in immune thrombocytopenia and in immune thrombotic thrombocytopenic purpura.
Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosisSuccessful use of recombinant ADAMTS13 in a pregnant patient with immune-mediated thrombotic thrombocytopenic purpura.
Journal of thrombosis and haemostasis : JTHBrain volumetric reductions in patients with thrombotic thrombocytopenic purpura.
PloS oneAn Unexpected Journey: Thrombotic Thrombocytopenic Purpura Unveiling Hidden HIV Infection.
Giornale italiano di nefrologia : organo ufficiale della Societa italiana di nefrologiaSevere ADAMTS-13 deficiency without hemolysis as a cause of cryptogenic stroke: A single center case series.
Journal of stroke and cerebrovascular diseases : the official journal of National Stroke AssociationSystemic immune inflammation index guides machine learning for rapid TTP diagnosis: a retrospective cohort study.
Frontiers in medicinePregnancy-Triggered Hereditary Thrombotic Thrombocytopenic Purpura: A Case Report.
International journal of women's healthCase report: post-delivery thrombotic thrombocytopenic purpura complicated by malignant catatonia.
Oxford medical case reportsCharacteristics and Management of Patients With Immune Thrombotic Thrombocytopenic Purpura Admitted to the Intensive Care Unit: A Multicenter Retrospective Analysis.
American journal of hematologyAssociation of Cognitive Impairment with Reduced Health-Related Quality of Life and Depression Among Survivors of Thrombotic Thrombocytopenic Purpura.
Hematology reportsReduced half-life of recombinant ADAMTS13 in a patient with cTTP undergoing total hip arthroplasty.
Thrombosis journalAtypical hemolytic uremic syndrome treated with anti-C5 antibody agent eculizumab, without genetic complement abnormalities.
Nagoya journal of medical scienceUpdated ISTH thrombotic thrombocytopenic purpura guidelines 2025-a bridge to the future for congenital thrombotic thrombocytopenic purpura.
Journal of thrombosis and haemostasis : JTHA rare case of hemolytic-uremic syndrome due to a Capnocytophaga canimorsus sepsis: Case report.
MedicinePathogenic cytokines in thrombotic microangiopathies: molecular insights and therapeutic targets.
Molecular medicine (Cambridge, Mass.)Recombinant ADAMTS-13 for congenital and immune thrombotic thrombocytopenic purpura in pregnancy.
Blood vessels, thrombosis & hemostasisUnmasking hidden shapes: unusual red cell morphologies in urine sediment.
Clinica chimica acta; international journal of clinical chemistryArgatroban-Associated Adverse Events: An Analysis Based on FDA Adverse Event Reporting System (FAERS).
Cardiovascular toxicologyResolution of thrombotic thrombocytopenic purpura serous retinal detachment following rituximab therapy.
Retinal cases & brief reportsFacing the bleeding risk of caplacizumab in postpartum immune thrombotic thrombocytopenic purpura in constrained resource settings.
British journal of haematologyRelapsing Chronic Immune Thrombocytopenia and Thrombosis: A Case Report.
CureusIdentification of HLA alleles involved in immune thrombotic thrombocytopenic purpura patients from Turkey: Erratum.
Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosisFetal and maternal morbidity in pregnant patients with thrombotic thrombocytopenic purpura: A Nationwide analysis.
TransfusionZebrafish model reveals developmental and hematopoietic functions of ADAMTS13.
Biology openReal-world safety and efficacy of rADAMTS13 prophylaxis in congenital thrombotic thrombocytopenic purpura: experience from Polish patients previously treated with fresh frozen plasma.
Haematologica[Advances in the diagnosis and treatment of thrombotic thrombocytopenic purpura].
[Rinsho ketsueki] The Japanese journal of clinical hematologyArterial Thrombotic Event in Thrombotic Thrombocytopenic Purpura Predating the Development of Microangiopathic Hemolytic Anemia and Thrombocytopenia.
Mayo Clinic proceedingsClinical characteristics and treatment response of patients with SLE complicated with thrombotic thrombocytopenic purpura.
Lupus science & medicineReference Range for the Automated Fragmented Red Cell Parameter and Its Diagnostic Utility in Red Blood Cell (RBC) Fragment Quantification: A Prospective Study.
Cureus[Exploring critical thinking in the management of diagnosis and treatment of fulminant pregnancy-associated atypical haemolytic uraemic syndrome].
Zhonghua wei zhong bing ji jiu yi xueThrombotic microangiopathy multidisciplinary assessment team: demographics, final diagnosis, treatment, and outcomes.
BMC nephrologyPosterior reversible encephalopathy syndrome due to thrombotic thrombocytopenic purpura in a liver-transplant patient treated with tacrolimus.
Journal of gastrointestinal and liver diseases : JGLDADAMTS13 Activity Testing: Evaluation of Two Automated Platforms for Diagnosis and Follow-Up of Thrombotic Thrombocytopenic Purpura.
International journal of laboratory hematologyStandard cardiovascular risk prediction scores underestimate risk in immune-mediated thrombotic thrombocytopenic purpura survivors.
Research and practice in thrombosis and haemostasisThe impact of the COVID-19 pandemic on incidence and clinical presentation of thrombotic microangiopathies: data from a laboratory centralizing ADAMTS-13 testing in Quebec.
Orphanet journal of rare diseasesThe Differential Diagnosis of Thromobocytopenia.
Deutsches Arzteblatt internationalNew Insights Into Renal Involvement During Immune-Mediated Thrombotic Thrombocytopenic Purpura.
Kidney international reportsPregnancy-Induced Thrombotic Thrombocytopenic Purpura Complicated by Atypical Posterior Reversible Encephalopathy Syndrome and Acute Pancreatitis: A Rare Presentation.
CureusRegarding "Challenges in diagnosing thrombotic thrombocytopenic purpura".
Hematology, transfusion and cell therapyFetal Hereditary Thrombotic Thrombocytopenic Purpura.
The New England journal of medicineTherapeutic plasma exchange in immune thrombotic thrombocytopenic purpura: A six-year prospective study on clinical efficacy, laboratory trends, and sex-based differences.
Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for HaemapheresisCan COVID-19 Lead to Refractory Thrombotic Thrombocytopenic Purpura (TTP) During Pregnancy and Postpartum? A Case Report and a Review of the Literature.
Clinical case reportsA Clever Stroke Mimic: Thrombotic Thrombocytopenic Purpura Without Schistocytes - A Case Report and Literature Review.
European journal of case reports in internal medicineEvaluation of different platelet-dependent von Willebrand factor activity assays to assess the in vivo inhibitory effect of caplacizumab on the von Willebrand factor-platelet interaction.
Journal of thrombosis and haemostasis : JTHCytokine storm and microvascular fate: mechanistic insights into endothelial injury in thrombotic microangiopathies.
Annals of medicine and surgery (2012)Prognostic value of laboratory biomarkers for mortality risk stratification in thrombotic thrombocytopenic purpura.
Annals of hematology[Personalised treatment of patients with immune thrombotic thrombocytopenic purpura].
Terapevticheskii arkhivOptimizing ADAMTS13 prophylaxis to reduce relapse and organ failure in congenital thrombotic thrombocytopenic purpura.
Blood advancesAssociation of Therapeutic Plasma Exchange-Treated Thrombotic Thrombocytopenic Purpura with Improved Mortality Outcome in End-Stage Renal Disease.
Diseases (Basel, Switzerland)Long-term follow-up outcomes in congenital thrombotic thrombocytopenic purpura.
BloodImplementation of a fully automated ADAMTS13 activity assay utilizing fluorescence resonance energy transfer with a practical approach to address high background fluorescence interference.
American journal of clinical pathologyCOVID-19 Microangiopathy: Insights into plasma exchange as a therapeutic strategy.
Hematology, transfusion and cell therapyHigh anti-ADAMTS13 IgG Levels after Plasma Exchange Predict Delayed ADAMTS13 Normalization in Immune-mediated Thrombotic Thrombocytopenic Purpura.
Thrombosis and haemostasisRare onset of congenital thrombotic thrombocytopenic purpura during pregnancy: A novel ADAMTS13 gene variant.
TransfusionSevere Plasmodium Falciparum Malaria Mimicking Thrombotic Thrombocytopenic Purpura (TTP): A Case Report From China.
International medical case reports journalProgression From Early Multiterritorial Punctate Infarcts to Massive Stroke in Fulminant Thrombotic Thrombocytopenic Purpura Despite Aggressive Therapy: A Therapeutic Dilemma in Initiating Antithrombotic Therapy.
CureusPrognostic relevance of early clinical and laboratory findings in immune-mediated thrombotic thrombocytopenic purpura.
Research and practice in thrombosis and haemostasisRecurrent Thrombotic Thrombocytopenic Purpura Associated With Helicobacter pylori: More Than a Gut Feeling.
CureusKidney transplantation in congenital thrombotic thrombocytopaenic purpura: using recombinant ADAMTS13 to balance relapse and rejection.
BMJ case reportsThrombotic Thrombocytopenic Purpura in Pregnancy.
MCN. The American journal of maternal child nursingProgressive silent cerebral infarction is associated with stroke and persistent cognitive impairment in survivors of iTTP.
Blood advancesSevere congenital thrombotic thrombocytopenic purpura in pregnancy complicated by eclampsia.
BMJ case reportsTrp1250, Lys1252, and Arg1367 of ADAMTS13 comprise a hot spot for anti-CUB domain antibodies in patients with iTTP.
Blood advancesNovel prognostic factor in thrombotic thrombocytopenic purpura: human leukocyte antigen class II.
Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosisSeasonal variation in immune thrombotic thrombocytopenic purpura in New England.
Blood vessels, thrombosis & hemostasisRising total leukocyte counts correspond with rising platelet counts in thrombotic thrombocytopenic purpura.
Blood vessels, thrombosis & hemostasisGenetic variants contribute to modulation of renal function in patients with immune thrombotic thrombocytopenic purpura.
Blood vessels, thrombosis & hemostasisA rare complication of thrombotic microangiopathy induced by chemotherapy for second breast cancer in a Hodgkin lymphoma survivor: a case report.
AME case reportsCharacterization of autopsy findings including multivisceral glomeruloid vascular bodies in hereditary thrombotic thrombocytopenic purpura with two new variants in ADAMTS13 gene.
Virchows Archiv : an international journal of pathologyA peculiar case report of primary iTTP in a previously healthy Saudi man.
Journal of medicine and lifeDifferential Diagnosis of Thrombotic Microangiopathy: Overlapping Features of Thrombotic Thrombocytopenic Purpura and Complement-Mediated Thrombotic Microangiopathy in a Dengue-Infected Patient.
NephronCaplacizumab for pediatric immune thrombotic thrombocytopenic Purpura: A scoping review of current evidence.
Blood reviewsInvestigating the impact of platelet transfusions on adverse outcomes in patients with thrombotic thrombocytopenic purpura: A systematic review of published studies.
Thrombosis researchThrombotic Thrombocytopenic Purpura Triggered by Chronic Lymphocytic Leukemia: A Case Report.
Case reports in oncologyImmune-mediated thrombotic thrombocytopenic purpura with systemic lupus erythematosus: clinical features and outcome.
Lupus science & medicineOpen or closed? Understanding the molecular mechanisms and clinical implications of ADAMTS13's conformation.
HemaSphere[Translated article] Caplacizumab in acute thrombotic thrombocytopenic purpura refractory to standard treatment.
Farmacia hospitalaria : organo oficial de expresion cientifica de la Sociedad Espanola de Farmacia HospitalariaThrombotic Thrombocytopenic Purpura as a Rare Stroke Mimic.
Deutsches Arzteblatt internationalLongitudinal analysis of arterial pressure and its impact on outcomes in patients with thrombotic thrombocytopenic purpura.
Intensive care medicineClinical Manifestations, Treatment Characteristics, and Clinical Outcomes in Patients with Immune Thrombotic Thrombocytopenic Purpura (iTTP) in a Real-World Setting: An Interim Analysis of the Turkish iTTP Registry.
Turkish journal of haematology : official journal of Turkish Society of HaematologyThrombotic thrombocytopenic purpura: Description and analysis of 23 cases treated in Chile between 2017 and 2022.
MedwaveSplenectomy for immune thrombotic thrombocytopenic purpura: a systematic review and meta-analysis.
Journal of thrombosis and haemostasis : JTHFulminant myocarditis in immune-mediated thrombotic thrombocytopenic purpura: A case report.
Acute medicine & surgeryThe Hideous Side of Acute Pancreatitis: A Case of Pancreatitis-Induced Atypical Hemolytic Uremic Syndrome.
CureusPotentially reversible severe cardiac involvement in thrombotic microangiopathies with malignant hypertension.
Journal of nephrologyAnti-ADAMTS13 Antibodies Trajectory is Associated With ADAMTS13 Recovery in Immune-Mediated TTP.
American journal of hematologyProof-of-Concept in a Murine Model of Treatment of Thrombotic Thrombocytopenic Purpura Using Engineered Red Blood Cells.
bioRxiv : the preprint server for biologyNovel ADAMTS13 Mutation in a Patient With Congenital TTP Diagnosed in Pregnancy.
American journal of hematologyCongenital thrombotic thrombocytopenic purpura: a rare cause of severe neonatal jaundice and hypoxic respiratory failure - a case report.
Case reports in perinatal medicineA microfluidic approach reveals ongoing disease activity in thrombotic thrombocytopenic purpura patients despite clinical remission.
Journal of thrombosis and haemostasis : JTHPreferences of Patients With cTTP and Other Chronic Conditions for At-Home Prophylactic Infusions: A Discrete Choice Experiment.
Journal of patient experienceDiagnostic Value of Biological Parameters in Biopsy-Confirmed Thrombotic Microangiopathy-MATRIX Consortium Group.
Kidney international reportsSystemic Lupus Erythematosus-Associated Thrombotic Thrombocytopenic Purpura: A Case Report.
Cureus[Plasma exchange-free therapeutic management of immune-mediated thrombotic thrombocytopenic purpura].
[Rinsho ketsueki] The Japanese journal of clinical hematologyNavigating recurrent immune-mediated thrombotic thrombocytopenic purpura (iTTP) in pregnancy: A case report and literature review.
Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for HaemapheresisQuantitative Systems Pharmacology Modeling of Platelet Responses to Recombinant ADAMTS13 in Patients With Congenital Thrombotic Thrombocytopenic Purpura.
CPT: pharmacometrics & systems pharmacologyMicroangiopathic Hemolytic Anemia in Pregnancy.
Journal of Brown hospital medicineThrombotic Microangiopathies.
Emergency medicine clinics of North AmericaThrombotic thrombocytopenic purpura: early diagnosis and effective treatment in 2025.
Intensive care medicineImmunotherapy-associated hemostatic abnormalities: bleeding and thrombotic complications.
Annals of hematologyEarly Recognition of Atypical Thrombotic Thrombocytopenic Purpura (TTP) in Severe Asymptomatic Thrombocytopenia Using the PLASMIC Score and Peripheral Blood Smear.
CureusA predictive algorithm involving lactate dehydrogenase to serum creatinine ratio may assist in identifying patients with thrombotic thrombocytopenic purpura.
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Referências e fontes
Bases de dados externas citadas neste artigo
Publicações científicas
Artigos indexados no PubMed ligados a esta doença no grafo RarasNet — título, periódico e PMID direto da fonte, sem intermediação de IA.
- Systematic Literature Review of the Economic and Quality of Life Burden of Congenital and Immune-Mediated Thrombotic Thrombocytopenic Purpura.
- Thrombotic thrombocytopenic purpura: celebrating 25 years of ADAMTS13.
- Reinventing the wheel in ischemic stroke: Novel aspects in ADAMTS13 and von Willebrand axis.
- Revisiting Clinical Response and Refractoriness in Immune Thrombotic Thrombocytopenic Purpura.
- Caplacizumab as an Emerging Treatment for Patients With Thrombotic Thrombocytopenic Purpura Presenting With Acute Ischemic Stroke.
- [Caplacizumab combined with therapeutic plasma exchange, glucocorticoids, and rituximab for refractory thrombotic thrombocytopenic purpura: a case report and literature review].
- Establishing reverse chimeric antigen receptor T cells for precise targeting of immunemediated thrombotic thrombocytopenic purpura.
- Acquired Thrombotic Thrombocytopenic Purpura with a Poor Response to Treatment can be an Initial Manifestation of Occult Signet Ring Cell Carcinoma and other Malignancies.
- N-Acetylcysteine Therapy in Thrombotic Thrombocytopenic Purpura: A Systematic Review and Critical Appraisal.
- Precision medicine in thrombotic thrombocytopenic purpura: a narrative review.
Bases de dados e fontes oficiais
Identificadores e referências canônicas usadas para montar este verbete.
- ORPHA:54057(Orphanet)
- MONDO:0018896(MONDO)
- Purpura Trombocitopenica Idiopatica(PCDT · Ministério da Saúde)
- GARD:16659(GARD (NIH))
- Variantes catalogadas(ClinVar)
- Busca completa no PubMed(PubMed)
- Artigo Wikipedia(Wikipedia)
- Q1426491(Wikidata)
Dados compilados pelo RarasNet a partir de fontes abertas (Orphanet, OMIM, MONDO, PubMed/EuropePMC, ClinicalTrials.gov, DATASUS, PCDT/MS). Este conteúdo é informativo e não substitui avaliação médica.
Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar
