Verónica Soto-Arellano , Agustina María Petracco , Carlos Cruz-Montecinos
{"title":"Joint health in Chilean children with haemophilia on primary prophylaxis","authors":"Verónica Soto-Arellano , Agustina María Petracco , Carlos Cruz-Montecinos","doi":"10.1016/j.thromres.2025.109401","DOIUrl":"10.1016/j.thromres.2025.109401","url":null,"abstract":"<div><h3>Introduction</h3><div>Primary prophylaxis is the gold standard in severe haemophilia A and B. Proper monitoring and follow-up of this treatment are essential for achieving good outcomes. Haemophilia Early Arthropathy Ultrasound (HEAD-US) helps assess joint status and detect initial damage.</div><div>Our study aimed to evaluate the effectiveness of primary prophylaxis on joint health through HEAD-US in the paediatric population in Chile.</div></div><div><h3>Method</h3><div>We selected all patients under 15 years old in the country with severe haemophilia A or B who had received primary prophylaxis with plasma-derived FVIII/FIX products. Variables included in the analysis were joint health, treatment adherence (Veritas-Pro), morning prophylaxis treatment, home and hospital administration of treatment, and residence in the metropolitan region.</div></div><div><h3>Results</h3><div>Eighty-six children from 23 public centres were assessed (covers 85 % of the total population in Chile). Half of the children receiving standard half-life FVIII/IX prophylaxis still present joint damage, with the ankle the most affected joint. Among protective factors for joint health, adherence and living in the metropolitan region showed a significant odds ratio (<em>p</em> < 0.05).</div></div><div><h3>Conclusion</h3><div>Joint damage was present in 50% of the children, with the ankle being the most affected. Cartilage damage is a key marker in children over eight, while synovitis is predominant in younger ones. Adherence to prophylaxis and living in the metropolitan region are protective factors. These results highlight the importance of monitoring joint health and using HEAD-US for early screening in children on primary prophylaxis.</div></div>","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"253 ","pages":"Article 109401"},"PeriodicalIF":3.7,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144634121","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}
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":"Real-world safety and effectiveness of rurioctocog alfa pegol in 338 patients with hemophilia A in South Korea: A postmarketing surveillance study","authors":"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","doi":"10.1016/j.thromres.2025.109402","DOIUrl":"10.1016/j.thromres.2025.109402","url":null,"abstract":"<div><h3>Introduction</h3><div>Rurioctocog alfa pegol is a recombinant coagulation factor VIII (FVIII) with an extended half-life versus the parent product. Limited real-world data exist on the treatment of hemophilia A with rurioctocog alfa pegol in South Korea. This postmarketing surveillance study assessed its safety and effectiveness in a real-world setting, according to Korean regulatory requirements.</div></div><div><h3>Methods</h3><div>In this prospective, multicenter study (NCT03824522), data were collected from medical records of patients, including children, with hemophilia A receiving rurioctocog alfa pegol as standard clinical practice in South Korea. Safety (adverse events [AEs], adverse drug reactions [ADRs], and unexpected AEs/ADRs) and hemostatic effectiveness were analyzed.</div></div><div><h3>Results</h3><div>In total, 338 patients were included (mean age [range], 25.0 [1.0–61.0] years; children <12 years, <em>n</em> = 54 [16 %]; all had a history of FVIII treatment). AEs occurred in 20 (5.9 %) patients (unexpected AEs, <em>n</em> = 18 [5.3 %]; ADRs, <em>n</em> = 4 [1.2 %]; unexpected ADRs, <em>n</em> = 2 [0.6 %]; serious AEs, n = 4 [1.2 %]; serious ADRs, <em>n</em> = 0 [0.0 %]). Among children, AEs occurred in 1 (50 %) and 6 (11.5 %) patients aged <2 and ≥2 to <12 years, respectively. No new safety signals were observed. No bleeding events occurred in 239 (76.1 %) patients on prophylaxis. Hemostatic effectiveness was rated by physicians as excellent/good in most patients. Bleeding events were managed with 1–2 infusions. Treatment effectiveness was maintained in children during the study period. No patients developed inhibitor antibodies against rurioctocog alfa pegol.</div></div><div><h3>Conclusion</h3><div>Rurioctocog alfa pegol was effective in children and adults, with no new safety signals; most patients on prophylaxis experienced no bleeds during the study.</div></div>","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"253 ","pages":"Article 109402"},"PeriodicalIF":3.7,"publicationDate":"2025-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144655397","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}
Elin Baddeley , Carme Font , Isabelle Mahé , Michelle Edwards , Stephanie Sivell , Kate J. Lifford , Victoria Mailen Arfuch , Nuri Coma-Auli , Mette Søgaard , Helle Enggaard , Hélène Helfer , Nassima Si Mohammed , Kathy Seddon , Mark Pearson , Simone P. Mooijaart , Sebastian Szmit , F.A. Klok , Simon Noble , Anette Arbjerg Højen , SERENITY consortium
{"title":"“Who am I to say that I'm not going to take it”: patient perspectives on decisions about antithrombotic therapy in the context of advanced cancer","authors":"Elin Baddeley , Carme Font , Isabelle Mahé , Michelle Edwards , Stephanie Sivell , Kate J. Lifford , Victoria Mailen Arfuch , Nuri Coma-Auli , Mette Søgaard , Helle Enggaard , Hélène Helfer , Nassima Si Mohammed , Kathy Seddon , Mark Pearson , Simone P. Mooijaart , Sebastian Szmit , F.A. Klok , Simon Noble , Anette Arbjerg Højen , SERENITY consortium","doi":"10.1016/j.thromres.2025.109399","DOIUrl":"10.1016/j.thromres.2025.109399","url":null,"abstract":"<div><h3>Introduction</h3><div>The decision to reconsider antithrombotic therapy (ATT) in cancer patients nearing the end of life is complex given the increasing risk of haemorrhage and thrombosis. A decision support tool (DST) is being developed to facilitate this process. Understanding patients' experiences, values, and perspectives are an essential component, yet remain largely unexplored.</div></div><div><h3>Aim</h3><div>To explore these patients' experiences, values and perspectives regarding ATT use.</div></div><div><h3>Methods</h3><div>Qualitative study using semi-structured interviews with patients with advanced cancer receiving ATT, across Denmark, France, Spain, and the United Kingdom. Data were analysed using Framework Analysis.</div></div><div><h3>Results</h3><div>Sixty patients and 13 relatives participated. Three major themes were generated:<ul><li><span>1.</span><span><div>ATT is important and lifelong: Deprescription was perceived as counterintuitive; continuation was preferred, providing a sense of security.</div></span></li><li><span>2.</span><span><div>Varying perspectives regarding roles and responsibilities in ATT decision-making: Patients' views regarding their role varied. When a good relationship existed with their clinician, patients trusted them to lead on the decision. Relatives played a key supportive role.</div></span></li><li><span>3.</span><span><div>Challenges in navigating ATT management in the context of advanced cancer and multiple comorbidities: Decisions relating to ATT were rarely made in isolation. Patients prioritised cancer management and described difficulties navigating multiple health concerns.</div></span></li></ul></div></div><div><h3>Conclusion</h3><div>Patients found decision-making around ATT near the end of life multifaceted, occurring amid a myriad of competing priorities. While patients reported a reticence to discontinuing, ultimately many deferred such decisions to a clinician, whose role was highly valued. These findings support a need for a DST, to support informed and shared choices in ATT decisions.</div></div>","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"253 ","pages":"Article 109399"},"PeriodicalIF":3.7,"publicationDate":"2025-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144596057","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}
David E. González-Mendoza , Francisco Fernández-Nogueira , Misael Uribe , Norberto C. Chávez-Tapia , Natalia Nuño-Lámbarri
{"title":"Hemostatic alterations in metabolic dysfunction-associated steatotic liver disease (MASLD) and their link to venous thromboembolism (VTE)","authors":"David E. González-Mendoza , Francisco Fernández-Nogueira , Misael Uribe , Norberto C. Chávez-Tapia , Natalia Nuño-Lámbarri","doi":"10.1016/j.thromres.2025.109395","DOIUrl":"10.1016/j.thromres.2025.109395","url":null,"abstract":"<div><div>Metabolic dysfunction-associated steatotic liver disease (MASLD) is the most prevalent chronic liver condition worldwide. It is associated with obesity, insulin resistance, type 2 diabetes, hypertension, and dyslipidemia, contributing to increased cardiovascular risk. As MASLD progresses, it may evolve into more advanced liver damage, namely metabolic dysfunction-associated steatohepatitis (MASH), fibrosis, cirrhosis, and, in advanced stages, hepatocellular carcinoma. The liver plays a central role in hemostasis by synthesizing procoagulant, anticoagulant, and fibrinolytic factors. In MASLD patients, alterations in these hemostatic pathways may contribute to a prothrombotic state. Insulin resistance and chronic inflammation further exacerbate these disturbances, increasing the risk of thromboembolism. In conclusion, MASLD may represent not only a hepatic disorder but also an emerging cardiovascular and thromboembolic risk factor. This review aims to provide an updated overview of MASLD and its relationship with venous thromboembolism (VTE), focusing on physiological mechanisms, molecular alterations, and current clinical evidence on both conditions.</div></div>","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"253 ","pages":"Article 109395"},"PeriodicalIF":3.7,"publicationDate":"2025-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144570012","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}
Deborah Cappelletti , Francesco Bianco , Valentina Bucciarelli , Elena Raffaelli , Linda Bordignon , Gianluca Di Cesare , Benedetta Bucciarelli , Alessandro Bacchiocchi , Camilla Manini , Ettore Merlino , Sergio Filippelli , Maria Elena Lionetti
{"title":"Comparison of rivaroxaban with warfarin for intracardiac thrombosis in the pediatric population at different cardiac sites: Early experience of anticoagulation approach and treatment outcomes","authors":"Deborah Cappelletti , Francesco Bianco , Valentina Bucciarelli , Elena Raffaelli , Linda Bordignon , Gianluca Di Cesare , Benedetta Bucciarelli , Alessandro Bacchiocchi , Camilla Manini , Ettore Merlino , Sergio Filippelli , Maria Elena Lionetti","doi":"10.1016/j.thromres.2025.109397","DOIUrl":"10.1016/j.thromres.2025.109397","url":null,"abstract":"<div><h3>Aims</h3><div>To compare the rivaroxaban treatment with warfarin for intracardiac thrombi resolution in pediatric patients diagnosed with intracardiac thrombosis (ICT).</div></div><div><h3>Methods</h3><div>In 2020–24, we enrolled 18 [0.7 (0.3–12, Q1-Q3) years-old, 67 % males] consecutive ICT pediatric patients (< 16 years old) treated in 2020–22 with warfarin (<em>n</em> = 9, 0.1 mg/Kg/day) and in 2023–24 with rivaroxaban (n = 9, dosage based on patient's body weight). ICT was suspected by echocardiography and confirmed with cardiac computed tomography (CCT) or magnetic resonance (CMR) imaging. All the participants were treated with enoxaparin for 7 days before the rivaroxaban implementation. CCT/CMR was repeated to confirm ICT resolution. <em>N</em> = 9/18 (50 %) patients presented with right atrial thrombosis, <em>n</em> = 5/18 (28 %) and <em>n</em> = 3/18 (17 %) with right/left ventricular thrombi, respectively, and <em>n</em> = 1/18 (5 %) with left atrial appendage thrombus.</div></div><div><h3>Results</h3><div>Compared with warfarin, treatment with rivaroxaban had a shorter duration (Log-rank <em>P</em> = 0.020); warfarin vs. rivaroxaban: 9.67 ± 3.6 vs. 6.6 ± 2.2 weeks (<em>P</em> = 0.0040). After rivaroxaban, all the thromboses were resolved and confirmed at the CCT/CMR imaging; no major bleeding event was documented during the treatment based on the BASIC bleeding assessment scale. Only minor bleedings were documented. In details: warfarin experienced <em>n</em> = 6/9 (67 %) gastrointestinal bleedings and <em>n</em> = 2/9 macroscopic hematuria, while 3 gingival bleedings were documented in the rivaroxaban group. After a follow-up period of 6 months, no recurrences were detected/suspected at the echocardiography evaluation.</div></div><div><h3>Conclusions</h3><div>Among pediatric patients with ICT, irrespective of the thrombosis site, rivaroxaban seemed effective in thrombus resolution.</div></div><div><h3>ClinicalTrials.gov ID</h3><div><span><span>NCT06371170</span><svg><path></path></svg></span></div></div>","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"253 ","pages":"Article 109397"},"PeriodicalIF":3.7,"publicationDate":"2025-07-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144570011","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}
Hilary Whitworth , Kristina Wagner , Allison Zelinski , Elana DiCocco , Amy J. Goodwin Davies , Rochelle Jordan , Miranda Higginbotham , Michael L. O'Byrne , L. Charles Bailey , Leslie Raffini , Madhvi Rajpurkar
{"title":"A novel Computable phENotype To Identify Pulmonary Embolism in chilDrEn: The CENTIPEDE Study","authors":"Hilary Whitworth , Kristina Wagner , Allison Zelinski , Elana DiCocco , Amy J. Goodwin Davies , Rochelle Jordan , Miranda Higginbotham , Michael L. O'Byrne , L. Charles Bailey , Leslie Raffini , Madhvi Rajpurkar","doi":"10.1016/j.thromres.2025.109385","DOIUrl":"10.1016/j.thromres.2025.109385","url":null,"abstract":"<div><h3>Background</h3><div>Pediatric pulmonary embolism (PE) is rare and requires multicenter research to generate meaningful results and improve care. PEDSnet is a multicenter learning health system including 10 pediatric institutions and a valuable tool to study rare diseases.</div></div><div><h3>Objectives</h3><div>We aimed to develop a computable phenotype (CP) to accurately identify pediatric PE in PEDSnet.</div></div><div><h3>Methods</h3><div>We developed an algorithm for identification of pediatric PE in Children's Hospital of Philadelphia data within PEDSnet and compared it to blinded chart review. We identified a cohort of patients from 1/1/2012–12/31/2022 who were predicted to have PE based on the CP and an equal number predicted to not have a PE. Manual chart review identified true positive and negative and false positive and negative PE. Subsequent adjustments were made to improve CP performance. In patients with a confirmed PE, we identified select outcomes in PEDSnet and compared them to chart review to evaluate the ability to assess outcomes in PEDSnet.</div></div><div><h3>Results</h3><div>There were 152 PE patients identified by the best performing CP which included: 1) Anticoagulant prescribed, dispensed, or administered in an inpatient or emergency department encounter, 2) SNOMED-CT code for PE, 3) Chest CT or perfusion study in the encounter. CP performance characteristics were as follows: sensitivity 96 %, specificity 88 %, positive predictive value 90 %, and negative predictive value 95 %. Preliminary outcome validation highlighted areas for improvement in identifying transfusions and thrombolysis.</div></div><div><h3>Conclusions</h3><div>We report the first pediatric PE CP. Future research will include multicenter CP validation and improved identification of interventions and outcomes.</div></div>","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"253 ","pages":"Article 109385"},"PeriodicalIF":3.7,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144556946","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":"Long-term recurrence, bleeding, and mortality after first-time subsegmental pulmonary embolism compared to more proximal pulmonary embolism: Findings from the TROLL registry","authors":"Camilla Tøvik Jørgensen , Mazdak Tavoly , Heidi Hassel Pettersen , Synne Frønæs , Waleed Ghanima , Jostein Gleditsch","doi":"10.1016/j.thromres.2025.109386","DOIUrl":"10.1016/j.thromres.2025.109386","url":null,"abstract":"<div><h3>Background</h3><div>Subsegmental pulmonary embolism (SSPE) refers to clots that exclusively obstruct the subsegmental arteries. Data on long-term recurrence and bleeding risks following SSPE remain limited.</div></div><div><h3>Objectives</h3><div>To determine the long-term incidence of venous thromboembolism (VTE) recurrence after cessation of anticoagulation in patients with SSPE compared to those with more proximal PE (non-SSPE), to assess major and clinically relevant non-major bleeding (CRNMB), and to evaluate 30-day and 31–365-day all-cause mortality.</div></div><div><h3>Methods</h3><div>Between January 2005 and May 2020, 1135 cancer-free patients with a first-time computed tomography-verified PE were identified from The Venous Thrombosis Registry in ØstfOLd HospitaL (TROLL), Norway.</div></div><div><h3>Results</h3><div>Among the 1135 patients, 72 (6.3 %) were diagnosed with SSPE, while 1063 (93.7 %) had more proximal PE. Median age was 70 years (IQR: 58–80), and 51.4 % were women. The 10-year cumulative incidence of VTE recurrence after discontinuation of anticoagulation was 16.5 % (95 % CI: 4.5–35.1) in the SSPE group compared to 28.4 % (95 % CI: 24.5–32.4) in the non-SSPE group (<em>p</em> = 0.08). The 6-month cumulative incidence of major bleeding was 1.4 % (95 % CI, 0.1–6.6) in SSPE cases and 4.6 % (95 % CI: 3.4–5.9) in non-SSPE cases (<em>p</em> = 0.23). The 30-day all-cause mortality rate was 9.7 % (95 % CI: 4.8–19.3) for SSPE and 4.4 % (95 % CI: 3.3–5.8) for non-SSPE (<em>p</em> = 0.04).</div></div><div><h3>Conclusion</h3><div>While the VTE recurrence rate following SSPE was lower than in non-SSPE cases, it remained high, with 16.5 % recurrence within 10 years. Major bleeding rate was lower for SSPE than non-SSPE (1.4 % vs. 4.6 %, <em>p</em> = 0.23). Unexpectedly, the 30-days mortality rate following SSPE was significantly higher compared to non-SSPE cases.</div></div>","PeriodicalId":23064,"journal":{"name":"Thrombosis research","volume":"252 ","pages":"Article 109386"},"PeriodicalIF":3.7,"publicationDate":"2025-06-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144470851","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}