Seminars in thrombosis and hemostasis最新文献_第7页

Factor Replacement Treatment for Hemophilia A: Achievements and Perspectives. 血友病 A 的因子替代治疗：成就与展望》。

IF 3.6 2区医学

Seminars in thrombosis and hemostasis Pub Date : 2025-02-01 Epub Date: 2024-10-14 DOI: 10.1055/s-0044-1791778

Maria Elisa Mancuso

{"title":"Factor Replacement Treatment for Hemophilia A: Achievements and Perspectives.","authors":"Maria Elisa Mancuso","doi":"10.1055/s-0044-1791778","DOIUrl":"10.1055/s-0044-1791778","url":null,"abstract":"The mainstay of treatment for persons with hemophilia A (PwHA) with severe bleeding phenotype is prophylaxis. The pharmacokinetic (PK) profile of native factor VIII (FVIII) imposes the need for rather frequent intravenous injections to ensure effective prophylaxis, but this represents a relevant treatment burden and is associated with suboptimal adherence to treatment. In this light, the advent of extended half-life (EHL) FVIII molecules has improved prophylaxis feasibility and outcomes by favoring treatment individualization and tailoring protection according to specific clinical and nonclinical needs. Different technologies have been used to enhance FVIII PK properties including Fc-fusion and conjugation with polyethylene glycol. Data from clinical development programs for such molecules, together with growing real-world experience, have shown numerous benefits related to the use of EHL FVIII in PwHA. Recently a new class of ultra-long-acting EHL FVIII has been developed to further improve protection against bleeding episodes and achieve the ambitious goal of providing PwHA with hemostatic protection in the nonhemophilia range over longer time periods, hence ensuring very low bleeding rates and improving joint health and quality of life. In this review, the achievements and perspectives of replacement therapies for PwHA are summarized and discussed.","PeriodicalId":21673,"journal":{"name":"Seminars in thrombosis and hemostasis","volume":" ","pages":"18-22"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142473886","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Non-factor Therapies for Hemophilia: Achievements and Perspectives. 血友病的非因素治疗：成就与展望。

IF 3.6 2区医学

Seminars in thrombosis and hemostasis Pub Date : 2025-02-01 Epub Date: 2024-11-29 DOI: 10.1055/s-0044-1796651

Victor Jiménez-Yuste

{"title":"Non-factor Therapies for Hemophilia: Achievements and Perspectives.","authors":"Victor Jiménez-Yuste","doi":"10.1055/s-0044-1796651","DOIUrl":"10.1055/s-0044-1796651","url":null,"abstract":"Non-factor replacement therapies (NFTs) have been developed to address the limitations of conventional replacement therapies, aiming to improve hemostasis and provide enhanced protection against bleeding episodes and long-term joint damage for patients both with and without inhibitors. Factor VIII (FVIII)-mimetic agents, such as emicizumab, have transformed the management of hemophilia A with inhibitors, offering a lower treatment burden and an effective alternative for those without inhibitors as well. Rebalancing agents, including anti-tissular factor pathway inhibitor agents (concizumab and marstacimab) and serpin inhibitors like fitusiran, have shown promising efficacy for patients with hemophilia B with inhibitors and other hemophilia subtypes. Administered subcutaneously, NFTs generate stable thrombin levels and feature a long half-life, which can shift severe hemophilia toward a milder phenotype. These therapies are effective regardless of inhibitor status and hold potential for application in other bleeding disorders. Evaluating the potential thrombotic risk after implementing mitigation measures, along with the development of anti-drug antibodies (ADAs), remain critical areas for further analysis. NFTs pose additional challenges due to their complex mechanism of action and the absence of a standardized laboratory assessment method. Unresolved issues include optimal management strategies for major surgeries and tailored approaches for safe use in older populations. This review highlights the progress and future potential of NFTs in treating persons with hemophilia.","PeriodicalId":21673,"journal":{"name":"Seminars in thrombosis and hemostasis","volume":" ","pages":"23-27"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142755481","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Innovative Therapies for Acquired Hemophilia A. 后天性血友病 A 的创新疗法。

IF 3.6 2区医学

Seminars in thrombosis and hemostasis Pub Date : 2025-02-01 Epub Date: 2024-02-23 DOI: 10.1055/s-0044-1779737

Massimo Franchini, Daniele Focosi

引用次数: 0

Inflammation and Coagulation in Neurologic and Psychiatric Disorders. 神经和精神疾病中的炎症和凝血。

IF 3.6 2区医学

Seminars in thrombosis and hemostasis Pub Date : 2025-01-23 DOI: 10.1055/s-0044-1801824

Rabee Khoury, Joab Chapman

{"title":"Inflammation and Coagulation in Neurologic and Psychiatric Disorders.","authors":"Rabee Khoury, Joab Chapman","doi":"10.1055/s-0044-1801824","DOIUrl":"https://doi.org/10.1055/s-0044-1801824","url":null,"abstract":"Coagulation factors are intrinsically expressed in various brain cells, including astrocytes and microglia. Their interaction with the inflammatory system is important for the well-being of the brain, but they are also crucial in the development of many diseases in the brain such as stroke and traumatic brain injury. The cellular effects of coagulation are mediated mainly by protease-activated receptors. In this review, we sum up the role of the coagulation cascade in the development of different diseases including psychiatric disorders. In inflammatory diseases such as multiple sclerosis, fibrinogen activates microglia and suppresses the differentiation of oligodendrocytes, leading to axonal damage and suppression of remyelination. In ischemic stroke, thrombin activity is associated with the size of infarction, and the inhibition of either thrombin- or protease-activated receptor 1 promotes neuronal survival and reduces the size of infarction. Patients suffering from Alzheimer's disease express higher levels of thrombin, which in turn damages the endothelium, increases blood-brain barrier permeability, and induces cell apoptosis. In major depressive disorder, a positive correlation is present between prothrombotic states and suicidality. Moreover, both protein S deficiency and antiphospholipid antibodies are associated with schizophrenia and there is an effect of warfarin on psychosis-free intervals. Studying the coagulation in the brain could open a new door in understanding and treating neurological and psychiatric disorders, and extensive research should be conducted in this field.","PeriodicalId":21673,"journal":{"name":"Seminars in thrombosis and hemostasis","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143029365","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Venous Thromboembolism Occurrence and Association with Gastrointestinal Disorders in Children with Cystic Fibrosis: An Analysis from the TriNetX Research Network Global Multicenter Real-World Dataset. 囊性纤维化儿童静脉血栓栓塞的发生及其与胃肠道疾病的关联：来自TriNetX研究网络全球多中心真实世界数据集的分析

IF 3.6 2区医学

Seminars in thrombosis and hemostasis Pub Date : 2025-01-13 DOI: 10.1055/s-0044-1801825

Carolena Trocchia, Dina Ashour, Maua Mosha, Bailey Hamner, Marisol Betensky, Neil Goldenberg, Racha Khalaf

{"title":"Venous Thromboembolism Occurrence and Association with Gastrointestinal Disorders in Children with Cystic Fibrosis: An Analysis from the TriNetX Research Network Global Multicenter Real-World Dataset.","authors":"Carolena Trocchia, Dina Ashour, Maua Mosha, Bailey Hamner, Marisol Betensky, Neil Goldenberg, Racha Khalaf","doi":"10.1055/s-0044-1801825","DOIUrl":"https://doi.org/10.1055/s-0044-1801825","url":null,"abstract":"The purpose of this study is to (1) estimate and compare the prevalence of venous thromboembolism (VTE) in children (age 0 to ≤21) with versus without cystic fibrosis (CF); (2) investigate putative associations between specific gastrointestinal (GI) manifestations and the development of VTE among children with CF. This was a multicenter case-control analysis among patients aged 0 to ≤ 21 years between 2010 and 2020, using the TriNetX Research Network. Data queries included ICD-9/10 (International Classification of Diseases-9th/10th Revision) diagnosis codes. Bivariate associations with VTE among CF patients were compared using Chi-square testing for categorical variables and Student's t-test for continuous variables. We used multivariable logistic regression to test for independent associations of GI manifestations with VTE among children with CF, with adjustment for other salient covariates. There was a total of 7,689 children with and 22,327,660 without CF. The frequency of occurrence of VTE was increased nearly 20-fold among those with, as compared with without CF (130 vs. 7 per 10,000 patients). Acute pancreatitis (adjusted odd ratio [aOR] = 3.80, [95% confidence interval, CI: 2.00-7.22]), biliary disease (aOR = 2.17 [95% CI: 1.17-4.03]), gastrostomy status (aOR = 2.01 [95% CI: 1.27-3.18]), and malabsorption/malnutrition (aOR = 2.41 [95% CI: 1.52-3.82]) were each associated with a higher likelihood of VTE among children with CF. In conclusion, we found a significantly increased frequency of VTE occurrence and association of specific GI diseases as independent risk factors for VTE among children with CF compared with those without.","PeriodicalId":21673,"journal":{"name":"Seminars in thrombosis and hemostasis","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-01-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142979684","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Inherited or Immunological Thrombocytopenia: The Complex Nature of Platelet Disorders in 22q11.2 Deletion Syndrome. 遗传性或免疫性血小板减少：22q11.2缺失综合征中血小板疾病的复杂性质。

IF 3.6 2区医学

Seminars in thrombosis and hemostasis Pub Date : 2025-01-13 DOI: 10.1055/s-0044-1801383

Bartosz Urbański, Zuzanna Urbańska, Katarzyna Bąbol-Pokora, Ewelina Subocz, Wojciech Młynarski, Szymon Janczar

{"title":"Inherited or Immunological Thrombocytopenia: The Complex Nature of Platelet Disorders in 22q11.2 Deletion Syndrome.","authors":"Bartosz Urbański, Zuzanna Urbańska, Katarzyna Bąbol-Pokora, Ewelina Subocz, Wojciech Młynarski, Szymon Janczar","doi":"10.1055/s-0044-1801383","DOIUrl":"https://doi.org/10.1055/s-0044-1801383","url":null,"abstract":"22q11.2 deletion syndrome (22q11.2DS) is one of the most common congenital malformation syndromes resulting from disrupted embryonic development of pharyngeal pouches. The classical triad of symptoms described by Angelo DiGeorge is frequently accompanied by hematological and immune disorders. While it is well-established that patients with 22q11.2DS have an increased risk of recurrent autoimmune cytopenias, including immune thrombocytopenia, the platelet abnormalities in this population are more complex and multifaceted. Given this issue, we conducted a comprehensive literature review on platelet disorders in 22q11.2DS using accessible databases (PubMed and Scopus). We aimed to outline previous studies limitations and most urgent challenges concerning thrombocytopenia in these patients. One characteristic finding frequently observed in 22q11.2DS is mild macrothrombocytopenia caused presumably by the loss of one GP1BB allele, encoding the element of the GPIb-IX-V complex. This structure plays a central role in thrombocyte adhesion, aggregation, and subsequent activation. Recent studies suggest that defective megakaryopoiesis and impaired vasculogenesis may strongly influence platelet and hemostasis disorders in 22q11.2DS. Furthermore, the phenotypic manifestation may be modulated by epigenetic factors and gene expression modifiers located outside the deletion region. Although the final hemorrhagic phenotype is typically mild, these patients may require more frequent transfusions following major surgical procedures. Despite the risk of thrombocytopenia and thrombocytopathy, there is a lack of large-scale research on hematological anomalies in 22q11.2DS, and the available results are often inconclusive. Given the complexity of hemostatic disorders, it is essential to establish specific recommendations for perioperative management and autoimmune cytopenias treatment within this population.","PeriodicalId":21673,"journal":{"name":"Seminars in thrombosis and hemostasis","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-01-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142979683","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

The Overlooked Risk of Venous Thromboembolism in Psychiatric Patients: Epidemiology, Pathophysiology, and Implications for Clinical Care. 精神病人静脉血栓栓塞被忽视的风险：流行病学、病理生理学和临床护理的意义。

IF 3.6 2区医学

Seminars in thrombosis and hemostasis Pub Date : 2024-12-13 DOI: 10.1055/s-0044-1800968

Giris Jacob, Yoab Ocytil, Benjamin Brenner

{"title":"The Overlooked Risk of Venous Thromboembolism in Psychiatric Patients: Epidemiology, Pathophysiology, and Implications for Clinical Care.","authors":"Giris Jacob, Yoab Ocytil, Benjamin Brenner","doi":"10.1055/s-0044-1800968","DOIUrl":"https://doi.org/10.1055/s-0044-1800968","url":null,"abstract":"Psychiatric patients face a significantly shorter life expectancy than the general population due to a complex interplay of medical, behavioral, and social factors. Venous thromboembolism (VTE), encompassing both pulmonary embolism and deep vein thrombosis, is an underrecognized yet critical contributor to morbidity and mortality in this population. Evidence suggests a two to three times higher prevalence of VTE in psychiatric patients compared to the general population, with incidence rates up to 4.5 per 1,000 person-years. This elevated risk is attributed to a hypercoagulable-hypofibrinolytic state. It is influenced by metabolic abnormalities, pro-inflammatory pathways, antipsychotic medications, and genetic factors. Health care biases and reduced treatment compliance further exacerbate the burden. This review explores the epidemiology, pathophysiology, and mechanistic underpinnings of VTE in psychiatric populations, emphasizing the role of metabolic syndrome and antipsychotic therapy. To mitigate mortality and enhance outcomes for these high-risk individuals, it is imperative to address this issue through improved risk stratification and preventive strategies.","PeriodicalId":21673,"journal":{"name":"Seminars in thrombosis and hemostasis","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142822691","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Regulatory Effect of PDGF/PDGFR on Hematopoiesis. PDGF/PDGFR对造血功能的调节作用。

IF 3.6 2区医学

Seminars in thrombosis and hemostasis Pub Date : 2024-11-28 DOI: 10.1055/s-0044-1796630

Yong Liu, Junbin Huang, Lindi Li, Yifei Duan, Beng H Chong, Liang Li, Mo Yang

{"title":"Regulatory Effect of PDGF/PDGFR on Hematopoiesis.","authors":"Yong Liu, Junbin Huang, Lindi Li, Yifei Duan, Beng H Chong, Liang Li, Mo Yang","doi":"10.1055/s-0044-1796630","DOIUrl":"https://doi.org/10.1055/s-0044-1796630","url":null,"abstract":"Platelet-derived growth factor (PDGF) is a critical cytokine with substantial regulatory effects on hematopoiesis. Recent research highlights the essential role of PDGF in the modulation of hematopoietic stem/progenitor cells (HSPCs), megakaryocytes/platelets, and thrombopoietin (TPO) synthesis within the bone marrow microenvironment. PDGF directly stimulates the proliferation and differentiation of HSPCs while also inhibiting apoptosis. In addition, PDGF indirectly enhances the production of other growth factors, including granulocyte-macrophage colony-stimulating factors. Further, PDGF regulates TPO production and influences the bone marrow milieu, thus impacting hematopoiesis and platelet formation. Mechanistically, PDGF binds to its receptor, PDGF receptor (PDGFR), thus activating the PDGF/PDGFR signaling pathway. This pathway subsequently activates phosphoinositide 3-kinase/protein kinase B, leading to the activation of downstream cytokines, including c-Fos and NF-E2, while inhibiting caspase-3 activation. Collectively, these actions have prodifferentiation and antiapoptotic effects on megakaryocytes, thereby regulating platelet production. This review provides a comprehensive analysis of the regulatory role of the PDGF/PDGFR axis in hematopoiesis, with a particular focus on platelet production, by summarizing all studies on PDGF/PDGFR from our group and globally.","PeriodicalId":21673,"journal":{"name":"Seminars in thrombosis and hemostasis","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142751496","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

The Role of Platelets in Atherosclerosis: A Historical Review. 血小板在动脉粥样硬化中的作用：历史回顾

IF 3.6 2区医学

Seminars in thrombosis and hemostasis Pub Date : 2024-11-19 DOI: 10.1055/s-0044-1795097

Stefania Momi, Paolo Gresele

{"title":"The Role of Platelets in Atherosclerosis: A Historical Review.","authors":"Stefania Momi, Paolo Gresele","doi":"10.1055/s-0044-1795097","DOIUrl":"https://doi.org/10.1055/s-0044-1795097","url":null,"abstract":"Atherosclerosis is a chronic, multifactorial inflammatory disorder of large and medium-size arteries, which is the leading cause of cardiovascular mortality and morbidity worldwide. Although platelets in cardiovascular disease have mainly been studied for their crucial role in the thrombotic event triggered by atherosclerotic plaque rupture, over the last two decades it has become clear that platelets participate also in the development of atherosclerosis, owing to their ability to interact with the damaged arterial wall and with leukocytes. Platelets participate in all phases of atherogenesis, from the initial functional damage to endothelial cells to plaque unstabilization. Platelets deposit at atherosclerosis predilection sites before the appearance of manifest lesions to the endothelium and contribute to induce endothelial dysfunction, thus supporting leukocyte adhesion to the vessel wall. In particular, platelets release matrix metalloproteinases, which interact with protease-activated receptor 1 on endothelial cells triggering adhesion molecule expression. Moreover, P-selectin and glycoprotein Ibα expressed on the surface of vessel wall-adhering platelets bind PSGL-1 and β2 integrins on leukocytes, favoring their arrest and transendothelial migration. Platelet-leukocyte interactions promote the formation of radical oxygen species which are strongly involved in the lipid peroxidation associated with atherosclerosis. Platelets themselves actively migrate through the endothelium toward the plaque core where they release chemokines that modify the microenvironment by modulating the function of other inflammatory cells, such as macrophages. While current antiplatelet agents seem unable to prevent the contribution of platelets to atherogenesis, the inhibition of platelet secretion, of the release of MMPs, and of some specific pathways of platelet adhesion to the vessel wall may represent promising future strategies for the prevention of atheroprogression.","PeriodicalId":21673,"journal":{"name":"Seminars in thrombosis and hemostasis","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142676221","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Acquired Hemophilia A after Tislelizumab Treatment in a Patient with Right Lung Squamous Cell Carcinoma. 右肺鳞状细胞癌患者接受替斯利珠单抗治疗后获得性血友病 A。

IF 3.6 2区医学

Seminars in thrombosis and hemostasis Pub Date : 2024-11-07 DOI: 10.1055/s-0044-1792106

Fengfei Liu, Ying Wang, Naiqi Pang, Juan Xie, Peizhang Li

引用次数: 0