Research and Practice in Thrombosis and Haemostasis最新文献

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G protein–coupled receptor kinase 5 regulates thrombin signaling in platelets G 蛋白偶联受体激酶 5 调节血小板中的凝血酶信号传导
IF 3.4 3区 医学
Research and Practice in Thrombosis and Haemostasis Pub Date : 2024-08-01 DOI: 10.1016/j.rpth.2024.102556
{"title":"G protein–coupled receptor kinase 5 regulates thrombin signaling in platelets","authors":"","doi":"10.1016/j.rpth.2024.102556","DOIUrl":"10.1016/j.rpth.2024.102556","url":null,"abstract":"<div><h3>Background</h3><p>Our prior genome-wide association study of thrombin-induced platelet aggregation identified a G protein–coupled receptor kinase 5 (GRK5) noncoding variant (rs10886430-G) that is strongly associated with increased platelet reactivity to thrombin. This variant predisposes to increased risk of stroke, pulmonary embolism, and venous thromboembolism.</p></div><div><h3>Objectives</h3><p>To determine role of platelet specific GRK5 in platelet responses to agonists and injury.</p></div><div><h3>Methods</h3><p>Platelets from GRK5 mutant mice have been shown to have increased thrombin sensitivity, indicating that GRK5 may be a negative regulator of platelet activation. However, this has not been studied in a platelet-specific manner. We therefore used platelet-specific GRK5 mutant mice and models of thrombosis and pulmonary embolism.</p></div><div><h3>Results</h3><p>We now demonstrate that mice lacking GRK5 specifically in platelets had a mild increase in thrombin responses <em>in vitro</em> and a shortened time to arterial thrombosis <em>in vivo</em>. In addition, platelet GRK5 mutant mice had increased thrombin but not collagen-induced thrombus burden in a mouse model of pulmonary embolism.</p></div><div><h3>Conclusion</h3><p>These data indicate that platelet GRK5 has a significant role in limiting platelet responses to thrombin.</p></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924002516/pdfft?md5=5185fefc966689b08d67786f0a393f41&pid=1-s2.0-S2475037924002516-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142171901","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Erratum to ‘Illustrated State-of-the-Art Capsules of the ISTH 2024 Congress’ [Research and Practice in Thrombosis and Haemostasis Volume 8, Issue 4, May 2024, 102432] ISTH 2024 大会最新进展图解 "的勘误 [《血栓与止血研究与实践》第 8 卷第 4 期,2024 年 5 月,102432]
IF 3.4 3区 医学
Research and Practice in Thrombosis and Haemostasis Pub Date : 2024-07-01 DOI: 10.1016/j.rpth.2024.102498
Chris Ward , Nicola Curry , Magdy El-Ekiaby , Kerstin Jurk , Henri H. Versteeg , Charithani Keragala , Tal Burstyn-Cohen , Silvio Antoniak , Yuko Suzuki , Ross I. Baker , Olivier Christophe , Shoshana Revel-Vilk , Alice Hart , Carsten Deppermann , Huyen Tran , Nicola Pozzi , Walter H.A. Kahr , Steven P. Grover , Philip Wenzel , Ashley C. Brown , Ana Marín-Quilez
{"title":"Erratum to ‘Illustrated State-of-the-Art Capsules of the ISTH 2024 Congress’ [Research and Practice in Thrombosis and Haemostasis Volume 8, Issue 4, May 2024, 102432]","authors":"Chris Ward ,&nbsp;Nicola Curry ,&nbsp;Magdy El-Ekiaby ,&nbsp;Kerstin Jurk ,&nbsp;Henri H. Versteeg ,&nbsp;Charithani Keragala ,&nbsp;Tal Burstyn-Cohen ,&nbsp;Silvio Antoniak ,&nbsp;Yuko Suzuki ,&nbsp;Ross I. Baker ,&nbsp;Olivier Christophe ,&nbsp;Shoshana Revel-Vilk ,&nbsp;Alice Hart ,&nbsp;Carsten Deppermann ,&nbsp;Huyen Tran ,&nbsp;Nicola Pozzi ,&nbsp;Walter H.A. Kahr ,&nbsp;Steven P. Grover ,&nbsp;Philip Wenzel ,&nbsp;Ashley C. Brown ,&nbsp;Ana Marín-Quilez","doi":"10.1016/j.rpth.2024.102498","DOIUrl":"https://doi.org/10.1016/j.rpth.2024.102498","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924001936/pdfft?md5=a798afb3bd6f58ff3b81992af90d1d12&pid=1-s2.0-S2475037924001936-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141605149","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Syndemics in women’s health: poverty, social exclusion, and clustering of thrombotic and hemostasis disorders 妇女健康的综合症:贫困、社会排斥与血栓和止血疾病的聚集
IF 3.4 3区 医学
Research and Practice in Thrombosis and Haemostasis Pub Date : 2024-07-01 DOI: 10.1016/j.rpth.2024.102481
Ellen O’Rourke , Sarah Kelliher , Barry Kevane
{"title":"Syndemics in women’s health: poverty, social exclusion, and clustering of thrombotic and hemostasis disorders","authors":"Ellen O’Rourke ,&nbsp;Sarah Kelliher ,&nbsp;Barry Kevane","doi":"10.1016/j.rpth.2024.102481","DOIUrl":"https://doi.org/10.1016/j.rpth.2024.102481","url":null,"abstract":"<div><p>A State of the Art lecture titled “Syndemics in Women’s Health: Poverty, Social Exclusion and Clustering of Thrombotic and Haemostasis Disorders” was presented at the International Society on Thrombosis and Haemostasis (ISTH) Congress in 2023. Syndemics are characterized by the clustering of specific health conditions in vulnerable populations. These populations become vulnerable as a result of large-scale social, political, and economic factors that influence social determinants of health and increase susceptibility to disease. Vulnerable populations at risk of experiencing a syndemic include those who are subjected to social exclusion and gender- or race-based marginalization. Biological sex (assigned at birth based on physical &amp; genetic differences) and gender identity (the personal sense of ones own gender) have been recognized as important determinants of health outcomes in the context of certain syndemic diseases. Potential examples of syndemic biosocial interactions in the field of thrombosis and hemostasis include the effect of social determinants of health in perpetuating the global maternal mortality crisis and the role of poverty and marginalization in influencing thrombosis risk in socially excluded individuals. Initiatives directed at prevention and treatment of syndemic conditions require multilevel interventions directed at the socio-economic as well as the biological determinants of the disease. In the present article, we describe potential syndemic disease interactions in the field of thrombosis and hemostasis, and we summarize some relevant new data relating to the social determinants of health presented during the 2023 ISTH Congress.</p></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924001705/pdfft?md5=fc674c00a5ee93b88112cb5ebbc04c53&pid=1-s2.0-S2475037924001705-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141604757","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Exploratory rivaroxaban trial for isolated calf deep vein thrombosis with a risk factor of thrombosis extension: an open-label, multicenter, randomized controlled trial 针对具有血栓扩展危险因素的孤立性小腿深静脉血栓的利伐沙班探索性试验:一项开放标签、多中心、随机对照试验
IF 3.4 3区 医学
Research and Practice in Thrombosis and Haemostasis Pub Date : 2024-07-01 DOI: 10.1016/j.rpth.2024.102515
{"title":"Exploratory rivaroxaban trial for isolated calf deep vein thrombosis with a risk factor of thrombosis extension: an open-label, multicenter, randomized controlled trial","authors":"","doi":"10.1016/j.rpth.2024.102515","DOIUrl":"10.1016/j.rpth.2024.102515","url":null,"abstract":"<div><h3>Background</h3><p>Limited evidence exists regarding the incidence of recurrent venous thromboembolism (VTE) in patients diagnosed with isolated distal deep vein thrombosis (DVT) who are at risk of thrombosis extension whether they receive anticoagulation therapy or not.</p></div><div><h3>Objectives</h3><p>The study aimed to investigate the incidence of recurrent VTE and the impact of rivaroxaban in this patient population.</p></div><div><h3>Methods</h3><p>This open-label, exploratory, and randomized controlled trial was conducted at 7 centers in Japan between April 2019 and April 2022. Adult patients with isolated distal DVT at risk of thrombosis extension received either rivaroxaban combined with physical therapy or physical therapy alone for 90 days. Whole-leg ultrasound was performed at 14 and 90 days. We assessed a composite outcome of symptomatic or asymptomatic proximal DVT or symptomatic pulmonary embolism as the primary outcome until the end of the treatment period using an intention-to-treat analysis. Major bleeding was evaluated as a key secondary outcome.</p></div><div><h3>Results</h3><p>Out of 90 enrolled patients, 3 were excluded due to withdrawal of consent; therefore, we analyzed 87 participants. The rivaroxaban group (<em>n</em> = 42) reported no primary outcomes (0%; 95% CI, 0.0%-8.4%), whereas the physical therapy group (<em>n</em> = 45) had 2 cases of symptomatic proximal DVT (4.4%; 95% CI, 0.5%-15.1%). Major bleeding events occurred in 4 patients in the rivaroxaban group (9.5%; 95% CI, 2.7%-22.6%), whereas no events occurred in the physical therapy group (0%; 95% CI, 0%-7.9%).</p></div><div><h3>Conclusion</h3><p>Preliminary data suggest that rivaroxaban may reduce the risk of VTE recurrence among this patient subset, albeit with an increased incidence of bleeding events.</p></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924002103/pdfft?md5=08d54b0330e6ca9b0791899a0db535c7&pid=1-s2.0-S2475037924002103-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141690000","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Thromboembolic events in severe postpartum hemorrhage treated with recombinant activated factor VII: a systematic literature review and meta-analysis 使用重组活化因子 VII 治疗严重产后出血的血栓栓塞事件:系统性文献综述和荟萃分析
IF 3.4 3区 医学
Research and Practice in Thrombosis and Haemostasis Pub Date : 2024-07-01 DOI: 10.1016/j.rpth.2024.102533
{"title":"Thromboembolic events in severe postpartum hemorrhage treated with recombinant activated factor VII: a systematic literature review and meta-analysis","authors":"","doi":"10.1016/j.rpth.2024.102533","DOIUrl":"10.1016/j.rpth.2024.102533","url":null,"abstract":"<div><p>Postpartum hemorrhage (PPH) is an obstetric complication with high associated morbidity. Recombinant activated factor VII (rFVIIa) is used to treat severe PPH when uterotonics fail to stop bleeding. However, data on the safety of rFVIIa treatment of severe PPH from adequately powered trials are lacking. We systematically reviewed published data on the incidence of thromboembolic events (TEs) in women with PPH treated or not treated with rFVIIa (PROSPERO CRD42022360736). Databases (Embase, MEDLINE, BIOSIS, Current Contents, and the Cochrane Library) were searched for peer-reviewed publications published between January 1996 and August 2022 and conference abstracts published between January 2017 and August 2022 using search terms related to thromboembolism or infarction and PPH. Data were extracted from all publications reporting on a general population of women with PPH with information on TEs. Descriptive summary statistics and the estimated proportion of TEs were analyzed using a generalized linear mixed model based on the binomial distribution. Quality assessments were based on the checklist by Downs and Black. From 1637 potentially eligible studies, 55 publications were included reporting on 611 women treated and 32,488 women not treated with rFVIIa. The global estimated proportion of TEs was 1.82% (prediction interval [PI], 0.30-10.23) and 0.72% (PI, 0.03-16.47) in women with severe PPH treated and those not treated with rFVIIa, respectively. The estimated proportions of TEs were similarly small, with wide and largely overlapping PIs. Additional well-designed trials are needed to improve understanding of TE incidence in PPH.</p></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924002280/pdfft?md5=be0da4bab78466826c5464518f53f215&pid=1-s2.0-S2475037924002280-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141849860","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Spontaneous bleeding in chronic kidney disease: global coagulation assays may predict bleeding risk 慢性肾病自发性出血--全球凝血测定可预测出血风险
IF 3.4 3区 医学
Research and Practice in Thrombosis and Haemostasis Pub Date : 2024-07-01 DOI: 10.1016/j.rpth.2024.102520
{"title":"Spontaneous bleeding in chronic kidney disease: global coagulation assays may predict bleeding risk","authors":"","doi":"10.1016/j.rpth.2024.102520","DOIUrl":"10.1016/j.rpth.2024.102520","url":null,"abstract":"<div><h3>Background</h3><p>Chronic kidney disease (CKD) is associated with increased bleeding and thrombotic risks. Standard blood tests do not sufficiently quantify these risks. Global coagulation assays (GCAs) provide a more comprehensive assessment of coagulation.</p></div><div><h3>Objectives</h3><p>We aimed to evaluate if GCAs are predictive of spontaneous major bleeding (sMB) in CKD.</p></div><div><h3>Methods</h3><p>Adult patients with CKD (estimated glomerular filtration rate, &lt;30 mL/min/1.73m<sup>2</sup>) were recruited to this pilot prospective observational study. Testing with GCAs (thromboelastography, overall hemostatic potential, calibrated automated thrombogram, and plasminogen activator inhibitor-1) was performed, and the results were correlated to sMB events.</p></div><div><h3>Results</h3><p>Eighty-seven CKD patients (median age, 67 years; 67.8% male) were included, with median follow-up of 3.1 years. CKD patients demonstrated elevated fibrinogen, factor VIII, and von Willebrand factor antigen levels, while other conventional coagulation test results were within reference intervals. Ten episodes of sMB (11.5%) were captured (3.0/100 person-years), with no significant association demonstrated between sMB and antiplatelet use (<em>P</em> = .36), platelet count (<em>P</em> = .14), or renal function (urea, <em>P</em> = .27; estimated glomerular filtration rate, <em>P</em> = .09). CKD patients with sMB had more hypocoagulable GCA parameters compared with those without sMB. The lowest quartiles of endogenous thrombin potential (subhazard ratio [sHR], 7.11; 95% CI, 1.84-27.45), overall hemostatic potential (sHR, 6.81; 95% CI, 1.77-26.16), and plasminogen activator inhibitor-1 (sHR, 5.26; 95% CI, 1.55-17.91) were associated with sMB.</p></div><div><h3>Conclusion</h3><p>This pilot study demonstrates that GCAs such as thrombin and fibrin generation may predict sMB risk in patients with CKD, which has potential to be practice-changing. Larger studies are required to validate these findings.</p></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924002152/pdfft?md5=ff2aea45562ef749471de4163fc80ab0&pid=1-s2.0-S2475037924002152-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141851634","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Managing surgical relapse risk in acquired thrombotic thrombocytopenic purpura: a systematic review 后天性血栓性血小板减少性紫癜手术复发风险的管理:系统性综述
IF 3.4 3区 医学
Research and Practice in Thrombosis and Haemostasis Pub Date : 2024-07-01 DOI: 10.1016/j.rpth.2024.102478
{"title":"Managing surgical relapse risk in acquired thrombotic thrombocytopenic purpura: a systematic review","authors":"","doi":"10.1016/j.rpth.2024.102478","DOIUrl":"10.1016/j.rpth.2024.102478","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924001675/pdfft?md5=3920f44ea26e27f3ca81846026c365f0&pid=1-s2.0-S2475037924001675-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141630778","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Interference of lupus anticoagulant causing antiprothrombin and anti–beta-2-glycoprotein I antibodies on international normalized ratio measurements: comparative analysis of international normalized ratio methods 狼疮抗凝剂引起的抗凝血酶和抗β-2-糖蛋白 I 抗体对 INR 测量的干扰:INR 方法的比较分析
IF 3.4 3区 医学
Research and Practice in Thrombosis and Haemostasis Pub Date : 2024-07-01 DOI: 10.1016/j.rpth.2024.102470
Rachel Gehlen , Roxanne G. Moesbergen , CuiCui Bai , Philip G. de Groot , A.J.Gerard Jansen , Joost C.M. Meijers , Bas de Laat , Jasper A. Remijn
{"title":"Interference of lupus anticoagulant causing antiprothrombin and anti–beta-2-glycoprotein I antibodies on international normalized ratio measurements: comparative analysis of international normalized ratio methods","authors":"Rachel Gehlen ,&nbsp;Roxanne G. Moesbergen ,&nbsp;CuiCui Bai ,&nbsp;Philip G. de Groot ,&nbsp;A.J.Gerard Jansen ,&nbsp;Joost C.M. Meijers ,&nbsp;Bas de Laat ,&nbsp;Jasper A. Remijn","doi":"10.1016/j.rpth.2024.102470","DOIUrl":"10.1016/j.rpth.2024.102470","url":null,"abstract":"<div><h3>Background</h3><p>Life-long vitamin K antagonist (VKA) therapy is recommended as a standard of care in antiphospholipid syndrome (APS) patients with thrombosis. Concerns have been raised about the validity of international normalized ratio (INR) measurements in lupus anticoagulant (LA)-positive APS patients because LA may interfere with phospholipid-dependent coagulation tests and could elevate INR measurements.</p></div><div><h3>Objectives</h3><p>Here, we aimed to determine the interference of antigen-specific monoclonal and isolated patient antibodies with LA activity on INR measurements.</p></div><div><h3>Methods</h3><p>Pooled normal plasma and control plasma from patients on VKA (without LA) were incubated with monoclonal and isolated patient immunoglobulin G antiprothrombin and anti–beta-2-glycoprotein I antibodies that express LA activity. INR was determined before and after addition using 3 laboratory assays (Owren STA-Hepato Prest, Quick STA-NeoPTimal, and Quick STA-Neoplastine R) and 1 point-of-care test device (CoaguChek Pro II).</p></div><div><h3>Results</h3><p>Antiprothrombin and anti–beta-2-glycoprotein I antibodies with LA activity interfered with recombinant human thromboplastin reagents (Quick STA-Neoplastine R and CoaguChek Pro II), particularly when added to plasma of VKA-treated controls. This effect was most evident on point-of-care test INR measurements, while the recombinant Quick reagent exhibited a lesser degree of interference. In contrast, tissue-derived thromboplastin reagents (Owren STA-Hepato Prest and Quick STA-NeoPTimal) remained largely unaffected by these antibodies, both in pooled normal plasma and VKA anticoagulated control plasma. Among these reagents, the Owren INR reagent exhibited the lowest sensitivity to the influence of LA antibodies. This observed difference in sensitivity is independent of the plasma dilution factor or the presence of factor V or fibrinogen in Owren reagent.</p></div><div><h3>Conclusion</h3><p>INR reagents that utilize recombinant human thromboplastin are more sensitive to the presence of monoclonal and patient-derived antibodies with LA activity. Consequently, APS patients positive for LA should be monitored using tissue-derived thromboplastin reagents, given its reduced susceptibility to interference by LA-causing antibodies.</p></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924001596/pdfft?md5=907440664fb64fd45bb59c757e8a972f&pid=1-s2.0-S2475037924001596-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141409482","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Real-world usage and effectiveness of recombinant factor VIII/factor IX Fc in hemophilia A/B: final data from the 24-month, prospective, noninterventional PREVENT study in Germany 重组因子 VIII/ 因子 IX Fc 在 A/B 型血友病中的实际使用情况和效果:德国为期 24 个月的前瞻性非干预性 PREVENT 研究的最终数据
IF 3.4 3区 医学
Research and Practice in Thrombosis and Haemostasis Pub Date : 2024-07-01 DOI: 10.1016/j.rpth.2024.102482
{"title":"Real-world usage and effectiveness of recombinant factor VIII/factor IX Fc in hemophilia A/B: final data from the 24-month, prospective, noninterventional PREVENT study in Germany","authors":"","doi":"10.1016/j.rpth.2024.102482","DOIUrl":"10.1016/j.rpth.2024.102482","url":null,"abstract":"<div><h3>Background</h3><p>Real-world experience with efmoroctocog alfa (a recombinant factor [F]VIII Fc fusion protein [rFVIIIFc]) and eftrenonacog alfa (a recombinant factor IX Fc fusion protein [rFIXFc]) is needed to bridge evidence gaps.</p></div><div><h3>Objectives</h3><p>To describe rFVIIIFc/rFIXFc usage and effectiveness over a 24-month prospective period.</p></div><div><h3>Methods</h3><p>PREVENT (NCT03055611), a noninterventional study across 25 German hemophilia treatment centers, enrolled previously treated persons with hemophilia A and B (all ages/severities) on individualized rFVIIIFc/rFIXFc prophylaxis before/at enrollment. Primary endpoints included annualized bleeding rate (ABR), injection frequency (IF), and factor consumption (FC). Additionally, up to 12 months of retrospective FVIII/FIX data were collected. Physician and patient satisfaction, and safety outcomes were also assessed.</p></div><div><h3>Results</h3><p>Overall, 150 patients received ≥1 rFVIIIFc dose and 47 patients received ≥1 rFIXFc dose, with median prospective follow-up of 20.6 and 21.0 months, respectively. rFVIIIFc/rFIXFc demonstrated low median ABR (0.5/1.7), annualized IF (121.8/52.2 injections/y), and FC (4611.7/2423.9 IU/kg) in line with product labels. Compared with previous FVIII/FIX, there was a 56.0% reduction in ABR for rFVIIIFc (rate ratio, 0.44; 95% CI, 0.31-0.64), with no change for rFIXFc (rate ratio, 0.93; 95% CI, 0.66-1.31); rFVIIIFc/rFIXFc reduced annualized IF (rFVIIIFc, mean difference, −31.7; 95% CI, −40.3 to −23.1; rFIXFc, mean difference, −37.3; 95% CI, −46.9 to −27.8), while FC remained stable (rFVIIIFc, +374.1; 95% CI, +46.8 to +701.3; rFIXFc, +503.9; 95% CI, +95.4 to +912.4). Most physicians and patients were satisfied or highly satisfied with rFVIIIFc/rFIXFc. rFVIIIFc/rFIXFc were well tolerated, with no inhibitor development or treatment-related serious adverse events.</p></div><div><h3>Conclusion</h3><p>Real-world PREVENT data complement phase 3 trials and show that individualized rFVIIIFc/rFIXFc prophylaxis provided stable bleed protection with low IF and maintained FC. Compared with previous FVIII, ABR was considerably reduced with rFVIIIFc, with stable annualized FC. For rFIXFc, bleed protection was maintained vs previous FIX while reducing annualized IF.</p></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924001717/pdfft?md5=d34012183ee4054591d75ab52ad9d95b&pid=1-s2.0-S2475037924001717-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141638879","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Bleeding in valvular heart disease: is von Willebrand factor the culprit? 瓣膜性心脏病出血:von Willebrand因子是罪魁祸首吗?
IF 3.4 3区 医学
Research and Practice in Thrombosis and Haemostasis Pub Date : 2024-07-01 DOI: 10.1016/j.rpth.2024.102506
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引用次数: 0
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