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Corrigendum to “Efficacy and safety of prophylaxis and treatment of bleeding events with a novel fibrinogen concentrate from human plasma in patients with congenital fibrinogen deficiency” [Thromb. Res. volume 259 (2026) https://doi.org/10.1016/j.thromres.2026.109616] “先天性纤维蛋白原缺乏症患者用一种新型血浆浓缩纤维蛋白原预防和治疗出血事件的有效性和安全性”的更正[Thromb]。第259卷(2026)https://doi.org/10.1016/j.thromres.2026.109616]。
IF 3.4 3区 医学
Thrombosis research Pub Date : 2026-04-01 Epub Date: 2026-03-04 DOI: 10.1016/j.thromres.2026.109633
Claudia Djambas Khayat , Amal El-Beshlawy , Naglaa Omar , Emna Gouider Belhadjali , Abderrahim Khelif , Sonia Adolf , Wolfgang Miesbach , Silke Aigner , Salomon Abraha , Joerg Schuettrumpf , Heike Boehm
{"title":"Corrigendum to “Efficacy and safety of prophylaxis and treatment of bleeding events with a novel fibrinogen concentrate from human plasma in patients with congenital fibrinogen deficiency” [Thromb. Res. volume 259 (2026) https://doi.org/10.1016/j.thromres.2026.109616]","authors":"Claudia Djambas Khayat , Amal El-Beshlawy , Naglaa Omar , Emna Gouider Belhadjali , Abderrahim Khelif , Sonia Adolf , Wolfgang Miesbach , Silke Aigner , Salomon Abraha , Joerg Schuettrumpf , Heike Boehm","doi":"10.1016/j.thromres.2026.109633","DOIUrl":"10.1016/j.thromres.2026.109633","url":null,"abstract":"","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"260 ","pages":"Article 109633"},"PeriodicalIF":3.4,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147366447","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Multi-omics integration reveals molecular heterogeneity and constructs a machine learning survival model for sepsis-induced coagulopathy 多组学整合揭示了脓毒症诱导凝血病的分子异质性,并构建了机器学习生存模型。
IF 3.4 3区 医学
Thrombosis research Pub Date : 2026-03-01 Epub Date: 2026-02-12 DOI: 10.1016/j.thromres.2026.109618
Songzan Qian , Rui Zheng , Yiyi Shi , Misha Lai , Junhao Hu , Mingyue Xu , Danxiao Pang , Hewei Ge , Dingyuan Wang , Jingye Pan
{"title":"Multi-omics integration reveals molecular heterogeneity and constructs a machine learning survival model for sepsis-induced coagulopathy","authors":"Songzan Qian ,&nbsp;Rui Zheng ,&nbsp;Yiyi Shi ,&nbsp;Misha Lai ,&nbsp;Junhao Hu ,&nbsp;Mingyue Xu ,&nbsp;Danxiao Pang ,&nbsp;Hewei Ge ,&nbsp;Dingyuan Wang ,&nbsp;Jingye Pan","doi":"10.1016/j.thromres.2026.109618","DOIUrl":"10.1016/j.thromres.2026.109618","url":null,"abstract":"<div><h3>Background</h3><div>Sepsis-induced coagulopathy (SIC) is a life-threatening complication characterized by high heterogeneity and mortality. Current prognostic models relying solely on clinical indices often fail to capture complex molecular pathophysiology, limiting precise risk stratification. This study aimed to unveil the molecular landscape of SIC via multi-omics integration and develop a robust machine learning (ML) predictive model.</div></div><div><h3>Methods</h3><div>We conducted a comprehensive study of 878 SIC patients. A discovery cohort of 626 patients underwent blood transcriptomic profiling (RNA-seq) and a subset of 214 patients was analyzed for proteomic validation. Weighted Gene Co-expression Network Analysis (WGCNA) and Gene Set Enrichment Analysis (GSEA) were used to identify survival-associated modules and pathways. An ensemble ML framework was developed to integrate the clinical features with transcriptomic signatures for survival prediction.</div></div><div><h3>Results</h3><div>Clinical analysis identified age, lung infection, higher SOFA scores, and lactate levels as significant independent risk factors for mortality. Transcriptomic profiling revealed that elevated expression of <em>GABARAPL1</em>, <em>PHLPP1</em>, and <em>KLF6</em> was strongly associated with an increased risk of death, whereas elevated expression of genes, including TSN, NUP155, and TTC39C, was associated with better outcomes. Functionally, GSEA enrichment analysis revealed the suppression of oxidative phosphorylation and ribosome biogenesis along with the activation of hypoxia, heme metabolism, and inflammatory pathways in non-survivors. Proteomic analyses validated the mechanistic findings. The integrated ensemble machine learning survival model (Clinical + Transcriptomics) achieved a C-index of 0.735, which significantly outperformed the clinical-only model (C-index: 0.694). Stratification based on the model successfully distinguished high-risk patients with significantly lower survival rates (<em>p</em> = 0.00057).</div></div><div><h3>Conclusion</h3><div>Our multi-omics analysis highlights metabolic reprogramming, hypoxia, and dysregulated heme metabolism as the key molecular features of SIC. The developed ensemble ML model, which integrates molecular and clinical features, offers a superior tool for early risk stratification and precision management of septic coagulopathy.</div></div>","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"259 ","pages":"Article 109618"},"PeriodicalIF":3.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146228400","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Evaluating clinical outcomes for very early versus late venous thromboembolism prophylaxis in patients with isolated severe spinal trauma 评估孤立性严重脊柱创伤患者早期与晚期静脉血栓栓塞预防的临床结果。
IF 3.4 3区 医学
Thrombosis research Pub Date : 2026-03-01 Epub Date: 2026-02-18 DOI: 10.1016/j.thromres.2026.109627
Youssef Nasef , Alexander Brown , Sukriti Prashar , Ian Bundschu , Yusra Othman , Ariel Hus , Adel Elkbuli
{"title":"Evaluating clinical outcomes for very early versus late venous thromboembolism prophylaxis in patients with isolated severe spinal trauma","authors":"Youssef Nasef ,&nbsp;Alexander Brown ,&nbsp;Sukriti Prashar ,&nbsp;Ian Bundschu ,&nbsp;Yusra Othman ,&nbsp;Ariel Hus ,&nbsp;Adel Elkbuli","doi":"10.1016/j.thromres.2026.109627","DOIUrl":"10.1016/j.thromres.2026.109627","url":null,"abstract":"<div><h3>Introduction</h3><div>Patients with traumatic spinal injuries face a high risk of venous thromboembolism (VTE); however, the optimal timing of VTE prophylaxis to most effectively reduce VTE rates remains unclear. This study aims to assess the association between thromboprophylaxis timing and clinical outcomes in patients with traumatic spinal injuries.</div></div><div><h3>Methods</h3><div>This retrospective cohort study used the ACS-TQIP database (2017–2023) to evaluate the timing of VTE prophylaxis in adult trauma patients (≥18 years) with isolated acute spinal cord injuries (SCI). Patients were stratified by prophylaxis type, management strategy, and spinal injury area. The primary outcomes were the odds of deep vein thrombosis (DVT) and pulmonary embolism (PE). Secondary outcomes included in-hospital mortality, complication rates, transfusion requirements, and ICU length of stay (ICU-LOS).</div></div><div><h3>Results</h3><div>Among severe spinal trauma patients, very early prophylaxis was associated with significant reductions in the odds of DVT (aOR: 0.387, <em>p</em> &lt; 0.001, SE = 0.159) and PE (aOR: 0.342, p &lt; 0.001, SE = 0.249) without increasing mortality or other complications. In patients undergoing operative management, only very early prophylaxis was associated with decreased odds of VTE (DVT: aOR: 0.506, <em>p</em> = 0.016, SE: 0.283; PE: aOR: 0.271, <em>p</em> = 0.035, SE: 0.620).</div></div><div><h3>Conclusion</h3><div>Very early VTE prophylaxis resulted in a significant reduction in VTE rates without increasing adverse outcomes in patients with severe spinal trauma, regardless of spinal region, management strategy, and prophylaxis type.</div></div>","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"259 ","pages":"Article 109627"},"PeriodicalIF":3.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146259218","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
In Memory of Dr. Jeffrey Ginsberg 纪念杰弗里·金斯伯格博士
IF 3.4 3区 医学
Thrombosis research Pub Date : 2026-03-01 Epub Date: 2026-02-25 DOI: 10.1016/j.thromres.2026.109617
Jack Hirsh
{"title":"In Memory of Dr. Jeffrey Ginsberg","authors":"Jack Hirsh","doi":"10.1016/j.thromres.2026.109617","DOIUrl":"10.1016/j.thromres.2026.109617","url":null,"abstract":"","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"259 ","pages":"Article 109617"},"PeriodicalIF":3.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147400839","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Monocyte depletion reduces late experimental post-thrombotic fibrotic injury in a stasis mouse model 单核细胞耗竭减少实验性晚期血栓后纤维化损伤在停滞小鼠模型
IF 3.4 3区 医学
Thrombosis research Pub Date : 2026-03-01 Epub Date: 2026-01-22 DOI: 10.1016/j.thromres.2026.109595
Mattea Pellerito , Abigail R. Dowling , Catherine E. Luke , Qing Cai , Antonio M. Pellerito , Andrew Quang , Lonnie Shea , Farouc Jaffer , Andrea Obi , Peter K. Henke
{"title":"Monocyte depletion reduces late experimental post-thrombotic fibrotic injury in a stasis mouse model","authors":"Mattea Pellerito ,&nbsp;Abigail R. Dowling ,&nbsp;Catherine E. Luke ,&nbsp;Qing Cai ,&nbsp;Antonio M. Pellerito ,&nbsp;Andrew Quang ,&nbsp;Lonnie Shea ,&nbsp;Farouc Jaffer ,&nbsp;Andrea Obi ,&nbsp;Peter K. Henke","doi":"10.1016/j.thromres.2026.109595","DOIUrl":"10.1016/j.thromres.2026.109595","url":null,"abstract":"<div><h3>Background</h3><div>Post thrombotic syndrome is a fibrotic disease related to inflammation resolution. There are no direct therapies that can ameliorate this disease. Monocyte/macrophages (Mo/MØ) are the primary leukocyte involved with later venous resolution, and likely direct vein wall responses and healing. Herein, we explored the vein wall response with Mo/MØ depletion by two methods.</div></div><div><h3>Methods</h3><div>Using two mouse models of venous thrombosis (VT), complete stasis and a flow restricted model, Mo/MØ depletion was accomplished using CD11b-DTR mice administered diphtheria toxin, and clodronate micelle administration in wild type mice. Tissue assays for structural histology, immunohistochemistry and western blotting were performed.</div></div><div><h3>Results</h3><div>Mo/MØ depletion resulted in significantly less vein wall fibrotic thickness, in the stasis model at day 14 in both the CD11b-DTR mice and those receiving clodronate micelles. No significant effect of Mo/MØ depletion was observed in the flow restricted VT model. The decrease in vein wall fibrosis was associated with fewer DDR2+ vein wall cells in the CD11b-DTR mice, but no difference in endothelial luminal coverage. Decreased cytokine and growth factor expression of IL-6, FSP-1, and VEGFa were associated with Mo/MØ depletion. Lastly, PMN depletion was associated with increased proinflammatory Mo/MØ, and a trend towards increased vein wall fibrosis as compared with controls.</div></div><div><h3>Conclusion</h3><div>Mo/MØ direct the post VT late fibrotic response, possibly by affecting fibroblasts and inflammatory cytokine expression. This effect was only found with the complete stasis model and is consistent with worsened PTS in humans with complete venous obstruction.</div></div>","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"259 ","pages":"Article 109595"},"PeriodicalIF":3.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146057467","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Monitoring the Hemostatic Balance: measuring thrombin generation in a patient with acquired hemophilia A on combined pro- and anticoagulant therapy 监测止血平衡:测量获得性血友病a患者在抗凝和促凝联合治疗中的凝血酶生成。
IF 3.4 3区 医学
Thrombosis research Pub Date : 2026-03-01 Epub Date: 2026-01-31 DOI: 10.1016/j.thromres.2026.109604
Marieke J.A. Verhagen , Saskia E.M. Schols , Sanna R. Rijpma , An K Stroobants
{"title":"Monitoring the Hemostatic Balance: measuring thrombin generation in a patient with acquired hemophilia A on combined pro- and anticoagulant therapy","authors":"Marieke J.A. Verhagen ,&nbsp;Saskia E.M. Schols ,&nbsp;Sanna R. Rijpma ,&nbsp;An K Stroobants","doi":"10.1016/j.thromres.2026.109604","DOIUrl":"10.1016/j.thromres.2026.109604","url":null,"abstract":"","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"259 ","pages":"Article 109604"},"PeriodicalIF":3.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146133027","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Simplifying the next-generation of anticoagulants: Elexians – Why a unified nomenclature for FXI/XIa inhibitors is needed 简化下一代抗凝血剂:Elexians -为什么需要FXI/Xia抑制剂的统一命名
IF 3.4 3区 医学
Thrombosis research Pub Date : 2026-03-01 Epub Date: 2026-02-01 DOI: 10.1016/j.thromres.2026.109608
Andaleb Kholmukhamedov
{"title":"Simplifying the next-generation of anticoagulants: Elexians – Why a unified nomenclature for FXI/XIa inhibitors is needed","authors":"Andaleb Kholmukhamedov","doi":"10.1016/j.thromres.2026.109608","DOIUrl":"10.1016/j.thromres.2026.109608","url":null,"abstract":"","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"259 ","pages":"Article 109608"},"PeriodicalIF":3.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146114125","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Obesity and type 2 diabetes mellitus add up to induce platelet hyperreactivity and platelet activation 肥胖和2型糖尿病共同诱发血小板高反应性和血小板活化。
IF 3.4 3区 医学
Thrombosis research Pub Date : 2026-03-01 Epub Date: 2026-02-19 DOI: 10.1016/j.thromres.2026.109626
Giuseppe Guglielmini , Emanuela Falcinelli , Michelantonio De Fano , Anna Maria Mezzasoma , Loredana Bury , Gabriele Perriello , Pietro Minuz , Carmine Giuseppe Fanelli , Paolo Gresele
{"title":"Obesity and type 2 diabetes mellitus add up to induce platelet hyperreactivity and platelet activation","authors":"Giuseppe Guglielmini ,&nbsp;Emanuela Falcinelli ,&nbsp;Michelantonio De Fano ,&nbsp;Anna Maria Mezzasoma ,&nbsp;Loredana Bury ,&nbsp;Gabriele Perriello ,&nbsp;Pietro Minuz ,&nbsp;Carmine Giuseppe Fanelli ,&nbsp;Paolo Gresele","doi":"10.1016/j.thromres.2026.109626","DOIUrl":"10.1016/j.thromres.2026.109626","url":null,"abstract":"<div><h3>Background</h3><div>Platelet hyperreactivity and in vivo platelet activation, effectors of increased cardiovascular risk, have been associated with both type 2 diabetes (T2DM) and obesity, however, whether T2DM and obesity when combined further enhance platelet activation has not been explored.</div></div><div><h3>Materials and methods</h3><div>We assessed several parameters of platelet reactivity and in vivo platelet activation, VWF activity and adipokine levels in 40 well characterized obese subjects without T2DM (metabolically healthy obese, MHO), non obese T2DM patients (metabolically unhealthy normal weight, MUNW), obese and T2DM patients (metabolically unhealthy obese, MUO) and age and sex-matched healthy controls (metabolically healthy normal weight, MHNW).</div></div><div><h3>Results</h3><div>Both MUNW and MHO subjects showed enhanced platelet thrombus formation, increased response to collagen and ADP at light transmission aggregometry, raised urinary 11-dehydro-TxB<sub>2</sub>, higher production of platelet reactive oxygen species and impaired platelet nitric oxide formation compared to MHNW, which were further significantly worsened in MUO. VWF activity and resistin levels were significantly higher in MUO patients compared to the other groups.</div></div><div><h3>Conclusions</h3><div>Our data show that the concomitant presence of T2DM and obesity exert a significant additive effect on platelet hyperreactivity and in vivo platelet activation compared with only one of these risk factors, and suggest that enhanced VWF and resistin levels in MUO subjects play a pathogenic role in the modulation of the platelet response to stimuli leading to a strong platelet hyperreactivity and in vivo platelet activation, in turn favoring enhanced cardiovascular risk.</div></div>","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"259 ","pages":"Article 109626"},"PeriodicalIF":3.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147310489","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Corrigendum to “Real-world safety and effectiveness of rurioctocog alfa pegol in 338 patients with hemophilia A in South Korea: A postmarketing surveillance study” [Thromb. Res. 253 (2025) 109402] 对韩国338例A型血友病患者ruurioctocog alfa pegol的实际安全性和有效性的更正:一项上市后监测研究[Thromb]。第253(2025)条[9402]。
IF 3.4 3区 医学
Thrombosis research Pub Date : 2026-03-01 Epub Date: 2026-01-30 DOI: 10.1016/j.thromres.2026.109586
Ji Yoon Kim , Taiju Hwang , Sang Kyu Park , Ki-Young Yoo , Eun Jin Choi , Soyon Kim , Chur Woo You , Eungsun Kim , Aeran Jung , Young-Shil Park
{"title":"Corrigendum to “Real-world safety and effectiveness of rurioctocog alfa pegol in 338 patients with hemophilia A in South Korea: A postmarketing surveillance study” [Thromb. Res. 253 (2025) 109402]","authors":"Ji Yoon Kim ,&nbsp;Taiju Hwang ,&nbsp;Sang Kyu Park ,&nbsp;Ki-Young Yoo ,&nbsp;Eun Jin Choi ,&nbsp;Soyon Kim ,&nbsp;Chur Woo You ,&nbsp;Eungsun Kim ,&nbsp;Aeran Jung ,&nbsp;Young-Shil Park","doi":"10.1016/j.thromres.2026.109586","DOIUrl":"10.1016/j.thromres.2026.109586","url":null,"abstract":"","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"259 ","pages":"Article 109586"},"PeriodicalIF":3.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146097499","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Efficacy and safety of prophylaxis and treatment of bleeding events with a novel fibrinogen concentrate from human plasma in patients with congenital fibrinogen deficiency 先天性纤维蛋白原缺乏症患者血浆中新型浓缩纤维蛋白原预防和治疗出血事件的有效性和安全性。
IF 3.4 3区 医学
Thrombosis research Pub Date : 2026-03-01 Epub Date: 2026-02-10 DOI: 10.1016/j.thromres.2026.109616
Claudia Djambas Khayat , Amal El-Beshlawy , Naglaa Omar , Emna Gouider Belhadjali , Abderrahim Khelif , Sonia Adolf , Wolfgang Miesbach , Silke Aigner , Salomon Abraha , Joerg Schuettrumpf , Heike Boehm
{"title":"Efficacy and safety of prophylaxis and treatment of bleeding events with a novel fibrinogen concentrate from human plasma in patients with congenital fibrinogen deficiency","authors":"Claudia Djambas Khayat ,&nbsp;Amal El-Beshlawy ,&nbsp;Naglaa Omar ,&nbsp;Emna Gouider Belhadjali ,&nbsp;Abderrahim Khelif ,&nbsp;Sonia Adolf ,&nbsp;Wolfgang Miesbach ,&nbsp;Silke Aigner ,&nbsp;Salomon Abraha ,&nbsp;Joerg Schuettrumpf ,&nbsp;Heike Boehm","doi":"10.1016/j.thromres.2026.109616","DOIUrl":"10.1016/j.thromres.2026.109616","url":null,"abstract":"<div><h3>Background</h3><div>Congenital fibrinogen deficiency is a rare inherited coagulation disorder characterized by partial or total lack of plasma fibrinogen (hypo- or afibrinogenemia), or expression of dysfunctional fibrinogen (dysfibrinogenemia), which may cause plasma level reduction (hypodysfibrinogenemia). Loss of functional fibrinogen causes bleeding disposition. In case of trauma or surgical intervention, supplementation of human fibrinogen is required to stop or prevent extensive bleeding events.</div></div><div><h3>Study design</h3><div>A prospective, open-label, uncontrolled, multi-center phase I/III trial was performed to investigate pharmacokinetics, efficacy and safety of single and/or repetitive intravenous infusions of a human fibrinogen concentrate (BT524) for on-demand prophylaxis (ODP) and/or on-demand treatment (ODT) of bleeding events in patients with congenital fibrinogen deficiency.</div></div><div><h3>Patients</h3><div>In the phase III part of the trial reported here, 36 patients (3 children &lt;6 years, 9 children 6–12 years, 4 adolescents and 20 adults) were treated with BT524 for 175 bleeding events, either for ODP or ODT.</div></div><div><h3>Results</h3><div>Fibrinogen concentrate infusion substantially improved maximum clot firmness (MCF). The overall hemostatic response was rated as good or excellent in 98.9% of bleedings. Loss of blood was considered normal in the majority of surgical interventions, and wound healing was also positively assessed. Additional evaluations, including blood product consumption and adverse events, further supported the efficacy and confirmed the favorable safety profile of the novel fibrinogen product.</div></div><div><h3>Conclusion</h3><div>BT524 represents a novel human fibrinogen concentrate provenly efficacious and safe both for the treatment of bleeding and for peri-operative bleeding prophylaxis in patients with congenital fibrinogen deficiency of all ages.</div></div>","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"259 ","pages":"Article 109616"},"PeriodicalIF":3.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146214278","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
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