Research and Practice in Thrombosis and Haemostasis最新文献

{"title":"HTRS2025.O6C.2 Abstract Travel Award Clinical manifestations, management, and outcomes in patients with triple positive antiphospholipid antibodies","authors":"Madeline Young , Maria Teresa De Sancho","doi":"10.1016/j.rpth.2025.103136","DOIUrl":"10.1016/j.rpth.2025.103136","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 ","pages":"Article 103136"},"PeriodicalIF":3.4,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145712071","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}

{"title":"Rituximab-first versus eltrombopag monotherapy in pediatric persistent/chronic immune refractory thrombocytopenia after first-line therapy: a prospective multicenter cohort study","authors":"Jingyao Ma ,&nbsp;Zhifa Wang ,&nbsp;Juntao Ouyang ,&nbsp;Zheyan Lin ,&nbsp;Lingling Fu ,&nbsp;Zhenping Chen ,&nbsp;Yan Liu ,&nbsp;Lijuan Wang ,&nbsp;Xi Lin ,&nbsp;Xiaoling Cheng ,&nbsp;Yu Hu ,&nbsp;Shuyue Dong ,&nbsp;Jinxi Meng ,&nbsp;Jie Ma ,&nbsp;Runhui Wu","doi":"10.1016/j.rpth.2025.103241","DOIUrl":"10.1016/j.rpth.2025.103241","url":null,"abstract":"<div><h3>Background</h3><div>Immune thrombocytopenia (ITP) in children is an autoimmune disorder in which a significant subset develops persistent or chronic disease requiring second-line therapies, yet the optimal sequencing of agents such as rituximab (RTX) and eltrombopag (ELT) to achieve sustained remission off-treatment remains (SRoT) undefined.</div></div><div><h3>Objectives</h3><div>This study assessed whether RTX-first sequential therapy outperforms upfront ELT monotherapy in achieving durable remission for pediatric persistent/chronic ITP (p/cITP) after first-line failure.</div></div><div><h3>Methods</h3><div>This nonrandomized multicenter prospective cohort study enrolled 55 children (April 2020 to July 2023) with refractory p/cITP until first-line therapy. They were assigned to RTX-first (RfG, <em>n</em> = 35: single RTX dose, then ELT for nonresponders) or ELT monotherapy (ELT-first group [EfG], <em>n</em> = 20) groups, with 12-month follow-up. Primary endpoints were SRoT and treatment duration (TD), and secondary endpoints were response rate and safety. The study is registered at ChiCTR2100048162.</div></div><div><h3>Results</h3><div>Among the 55 enrolled children (RfG = 35, EfG = 20), RfG had superior SRoT (30%; 95% CI, 15.6%-48.7%) than ELT-monotherapy group (0%; 95% CI, 0%-18.7%; <em>P</em> = .016), although 12-month remission rates were comparable (84.8% vs 87.5%; <em>P</em> = .804). RfG had a shorter TD (median 18 vs 34 weeks; <em>P</em> = .009), even in severe cases (baseline platelets 20 ×10⁹/L vs 29 ×10⁹/L; <em>P</em> = .034). Early ELT (≤96 days) in RfG nonresponders improved outcomes (<em>P</em> = .024). Both treatments had favorable safety (grade 1-2 adverse events, &lt;15%).</div></div><div><h3>Conclusions</h3><div>RTX-first treatment yields a better SRoT and shorter TD than ELT monotherapy for pediatric p/cITP, with comparable remission and safety.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 8","pages":"Article 103241"},"PeriodicalIF":3.4,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145621443","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}

{"title":"AOZORA study: 3-year interim analysis of safety and joint health in pediatric people with hemophilia A receiving emicizumab prophylaxis","authors":"Midori Shima ,&nbsp;Hideyuki Takedani ,&nbsp;Kaoru Kitsukawa ,&nbsp;Masashi Taki ,&nbsp;Akira Ishiguro ,&nbsp;Chiai Nagae ,&nbsp;Azusa Nagao ,&nbsp;Daisuke Nosaka ,&nbsp;Yui Kyogoku ,&nbsp;Hiroki Oki ,&nbsp;Keisuke Iwasaki ,&nbsp;Keiji Nogami","doi":"10.1016/j.rpth.2025.103228","DOIUrl":"10.1016/j.rpth.2025.103228","url":null,"abstract":"<div><h3>Background</h3><div>Recurrent joint bleeding in people with hemophilia A (PwHA) can cause hemophilic arthropathy, resulting in limited movement and chronic pain. Emicizumab is a bispecific monoclonal antibody bridging activated factor (F)IX and FX to substitute for deficient activated FVIII in PwHA, thereby improving hemostasis.</div></div><div><h3>Objectives</h3><div>This 3-year interim analysis of the ongoing, open-label, phase IV AOZORA study (jRCT1080224629) analyzes medium-term safety and joint health in pediatric PwHA without FVIII inhibitors receiving emicizumab.</div></div><div><h3>Methods</h3><div>PwHA aged &lt;12 years with severe hemophilia A without FVIII inhibitors were eligible. Participants entered AOZORA as emicizumab-naïve or having previously initiated emicizumab during the HOHOEMI study. Endpoints included safety, and joint health, as assessed by magnetic resonance imaging and Hemophilia Joint Health Score (HJHS). Participants will receive emicizumab for 6 years.</div></div><div><h3>Results</h3><div>A total of 30 male PwHA were enrolled. Data cutoff was the last day of week 145 for each participant. Median (range) age was 4.2 (0.7-11.1) years, and 27 of the 30 (90.0%) had received prior FVIII prophylaxis. The emicizumab safety profile was confirmed. No thrombotic events/microangiopathies occurred. All joints with synovial hypertrophy and hemosiderin resolved or improved by week 145. HJHS remained at 0 from week 1 to week 145 for 18 (66.7%) participants; overall, there was no worsening trend in HJHS over time. Model-based annualized bleeding rate (95% CI) for treated bleeds was 3.6 (2.04-6.46) prior to emicizumab and 0.8 (0.47-1.22) after receiving emicizumab.</div></div><div><h3>Conclusion</h3><div>Emicizumab is well tolerated and appears to maintain or improve joint health in pediatric PwHA.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 8","pages":"Article 103228"},"PeriodicalIF":3.4,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145578113","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}

{"title":"Prognostic value of the pan-immune-inflammation value for mortality in sepsis-induced coagulopathy: a Medical Information Mart for Intensive Care study","authors":"Jian-Yue Yang,&nbsp;Li-Li Li,&nbsp;Su-Zhen Fu","doi":"10.1016/j.rpth.2025.103257","DOIUrl":"10.1016/j.rpth.2025.103257","url":null,"abstract":"<div><h3>Background</h3><div>Sepsis is a life-threatening condition characterized by organ dysfunction caused by a dysregulated host response to infection. Its associated coagulopathy, known as sepsis-induced coagulopathy (SIC), significantly increases mortality risk. The pan-immune-inflammation value (PIV), a composite biomarker reflecting systemic immune and inflammatory status, has been linked to prognosis in various diseases.</div></div><div><h3>Objectives</h3><div>This study aimed to evaluate the prognostic significance of PIV in patients with SIC and to develop predictive models accordingly.</div></div><div><h3>Methods</h3><div>This retrospective study utilized data from the Medical Information Mart for Intensive Care IV database and included 4554 patients diagnosed with sepsis. Patients were stratified into high- and low-PIV groups based on the median PIV, and clinical characteristics were compared between groups. Kaplan–Meier survival analysis and Cox regression were employed to assess the association between PIV and patient outcomes. Least absolute shrinkage and selection operator regression was used to identify key variables for constructing a nomogram model. Additionally, machine learning algorithms, including random forest, were applied to build and validate predictive models.</div></div><div><h3>Results</h3><div>Patients in the high-PIV group had significantly higher 30-day and 90-day mortality rates. Kaplan–Meier analysis showed that patients with lower PIVs had markedly better survival, and a nonlinear positive correlation was observed between PIV and mortality risk. Least absolute shrinkage and selection operator regression identified 8 key variables, including Acute Physiology Score III, lactate, red cell distribution width, mean corpuscular volume, acute kidney injury, and continuous renal replacement therapy. The nomogram based on these variables achieved areas under the receiver operating characteristic curve of 0.84 and 0.87 in the training and validation cohorts, respectively. Among machine learning models, the random forest algorithm exhibited the best predictive performance, with areas under the curve of 0.837 and 0.947 in the training and validation sets, respectively. External validation using a real-world cohort from Xingtai People’s Hospital further confirmed the association between elevated PIV and increased mortality and SIC, with consistent survival trends and nonlinear patterns observed in both Kaplan–Meier and restricted cubic spline analyses.</div></div><div><h3>Conclusion</h3><div>To our knowledge, this study is the first to incorporate PIV into the prognostic assessment of patients with SIC. The development of a visual nomogram and machine learning-based models provides clinicians with practical tools for early identification of patients at high risk for SIC, potentially aiding in the optimization of treatment strategies.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 8","pages":"Article 103257"},"PeriodicalIF":3.4,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145691043","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}

{"title":"Disparities and trends in pulmonary embolism mortality with and without obesity: a nationwide US analysis","authors":"Aman Goyal ,&nbsp;Humza Saeed ,&nbsp;Samia Aziz Sulaiman ,&nbsp;Muhammad Khubaib Arshad ,&nbsp;Kevin Michael Alexander ,&nbsp;Sripal Bangalore ,&nbsp;Liana K. Billings ,&nbsp;Alfonso J. Tafur ,&nbsp;Manan Pareek ,&nbsp;Gregory Piazza ,&nbsp;Arman Qamar","doi":"10.1016/j.rpth.2025.103240","DOIUrl":"10.1016/j.rpth.2025.103240","url":null,"abstract":"<div><h3>Background</h3><div>Obesity increases the risk of pulmonary embolism (PE) through multiple mechanisms.</div></div><div><h3>Objectives</h3><div>This study examined mortality trends in patients with coexisting PE and obesity.</div></div><div><h3>Methods</h3><div>We analyzed the Centers for Disease Control and Prevention Wide-ranging Online Data for Epidemiologic Research database for individuals aged 25 years and older who died between 1999 and 2020 from concurrent PE and obesity. Temporal trends and age-adjusted mortality rates (AAMRs) were assessed using Joinpoint regression software. Annual percent changes (APCs), average annual percentage change differences, and 95% CIs were calculated, with statistical significance set at <em>P</em> &lt; .05.</div></div><div><h3>Results</h3><div>From 1999 to 2020, the AAMR for PE with obesity increased from 5.1 (95% CI, 4.7-5.4) to 13.9 (95% CI, 13.4-14.4) per 1,000,000. The AAMR rose significantly from 1999 to 2018 (APC, 3.45; 95% CI, 2.65-4.01; <em>P</em> = .005), followed by a sharper increase from 2018 to 2020 (APC, 16.28; 95% CI, 6.24-21.22; <em>P</em> &lt; .001). Women consistently had higher AAMRs than men (8.8; 95% CI, 8.6-8.9; vs 6.5; 95% CI, 6.4-6.6). Among age groups, middle-aged adults had the highest AAMR (10; 95% CI, 9.8-10.1), while among the ethnoracial groups, non-Hispanic Black individuals had the highest AAMR (16.8; 95% CI, 16.5-17.2). Residents of nonmetropolitan areas had higher AAMRs than those in metropolitan areas (8.9; 95% CI, 8.7-9.1; vs 7.5; 95% CI, 7.4-7.6). The increase in AAMR was significantly greater for PE with obesity compared with PE alone (average annual percentage change difference, 3.61; 95% CI, 2.91-4.32; <em>P</em> &lt; .001).</div></div><div><h3>Conclusions</h3><div>The analysis reveals a significant rise in mortality from concurrent PE and obesity, with higher rates observed in women, middle-aged adults, non-Hispanic Black individuals, and residents of nonmetropolitan areas. These findings highlight the need for targeted interventions in these high-risk groups.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 8","pages":"Article 103240"},"PeriodicalIF":3.4,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145621442","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}

{"title":"High bleeding rates in δ-storage pool disease during surgeries and deliveries","authors":"Elleke van Heerwaarde ,&nbsp;Annick de Moor ,&nbsp;Albert Huisman ,&nbsp;Rolf Urbanus ,&nbsp;Idske Kremer Hovinga ,&nbsp;Roger Schutgens ,&nbsp;Thrombocytopathy in the Netherlands (TiN) study group","doi":"10.1016/j.rpth.2025.103239","DOIUrl":"10.1016/j.rpth.2025.103239","url":null,"abstract":"<div><h3>Background</h3><div>δ-storage pool disease (δ-SPD) involves various platelet disorders due to deficient or reduced secretion of dense granules, causing increased bleeding tendency, particularly during surgery and delivery. The optimal treatment to prevent bleeding in δ-SPD is unknown.</div></div><div><h3>Objectives</h3><div>To evaluate the efficacy of preventive treatment in a cohort of patients with δ-SPD.</div></div><div><h3>Methods</h3><div>In a single-center study, we retrospectively reviewed the medical files of δ-SPD patients diagnosed between 2016 and 2024. Data included invasive diagnostic procedures, surgeries (minor/major), and deliveries (vaginal/cesarean section) before and after diagnosis. Diagnosis was confirmed by platelet adenosine diphosphate levels (&lt;1.4 μmol/10¹¹ platelets). Bleeding complications were defined according to the International Society on Thrombosis and Haemostasis bleeding scale. Data on treatment and platelet transfusions were documented.</div></div><div><h3>Results</h3><div>Thirty-eight patients (mean age, 35.6 years; range, 0-67; 63.2% female) were included. The average adenosine diphosphate level was 0.82 μmol/10¹¹ platelets (range, 0.2-1.4). A total of 161 interventions and deliveries were analyzed. The bleeding rate was dependent on the timing of diagnosis (51.5% before δ-SPD diagnosis and 16.8% after; <em>P</em> &lt; .001) and on the use of preventive treatment (45.8% without and 17.9% with; <em>P</em> &lt; .001). A total of 145 interventions were analyzed, including 63 minor and 82 major. Of these, 43/145 (29.7%) were complicated by bleeding (39 minor bleeds and 4 major bleeds). Sixteen deliveries were analyzed. Among 10 vaginal deliveries, 9 had postpartum hemorrhage, of whom 6 had no prophylaxis. All 6 cesarean sections received prophylactic platelet transfusion; no bleeding occurred.</div></div><div><h3>Conclusion</h3><div>We observed a high perioperative/peripartum bleeding rate in patients with δ-SPD. This decreased significantly after a correct diagnosis. In addition, bleeding rates were significantly lower among patients who received preventive treatment before the procedure.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 8","pages":"Article 103239"},"PeriodicalIF":3.4,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145577683","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}

{"title":"Do combined oral contraceptives induce formation of tissue factor?","authors":"Søren Risom Kristensen ,&nbsp;Jette Nybo ,&nbsp;Jesper Strandberg","doi":"10.1016/j.rpth.2025.103260","DOIUrl":"10.1016/j.rpth.2025.103260","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 8","pages":"Article 103260"},"PeriodicalIF":3.4,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145691045","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}

{"title":"HTRS2025.P1.51 SARS-CoV-2 infection in a mouse model of Long COVID shows enhanced thrombotic susceptibility, endotheliopathy, and abnormal cognitive behavior for up to 5 months post-infections","authors":"Roberto Aiolfi ,&nbsp;Antonella Zampolli ,&nbsp;Miriam Hauer-Jensen ,&nbsp;Jose A. Fernandez ,&nbsp;Angel Gandarilla ,&nbsp;Julia C. Mosnier ,&nbsp;Amanda J. Roberts ,&nbsp;Juan Carlos de la Torre ,&nbsp;Laurent O. Mosnier ,&nbsp;Xiao Xu","doi":"10.1016/j.rpth.2025.103071","DOIUrl":"10.1016/j.rpth.2025.103071","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 ","pages":"Article 103071"},"PeriodicalIF":3.4,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145711785","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}

{"title":"HTRS2025.P2.54 Corticosteroid Experience With Fidanacogene Elaparvovec in Adults With Moderately Severe or Severe Hemophilia B: Results From the Phase 3 BENEGENE-2 Gene Therapy Trial","authors":"Adam Cuker ,&nbsp;Madison Snyder ,&nbsp;Kaan Kavakli ,&nbsp;Robert Klamroth ,&nbsp;Lisa J. Wilcox ,&nbsp;Delphine Agathon ,&nbsp;Pascal Klaus ,&nbsp;Pengling Sun ,&nbsp;Frank Plonski","doi":"10.1016/j.rpth.2025.103074","DOIUrl":"10.1016/j.rpth.2025.103074","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 ","pages":"Article 103074"},"PeriodicalIF":3.4,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145711788","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}

{"title":"HTRS2025.P2.61 Post-operative outcomes in adult patients with Hemophilia A using efanesoctocog alfa","authors":"Anya Parekh ,&nbsp;Cody Angerman ,&nbsp;Marvin Sanchez ,&nbsp;Anjlee Mahajan ,&nbsp;Adam Giermasz","doi":"10.1016/j.rpth.2025.103081","DOIUrl":"10.1016/j.rpth.2025.103081","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 ","pages":"Article 103081"},"PeriodicalIF":3.4,"publicationDate":"2025-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145711795","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}