Research and Practice in Thrombosis and Haemostasis最新文献

{"title":"Changes in body mass index following venous thromboembolism in children: a prospective cohort study","authors":"Mary P. Dang ,&nbsp;Maria Hanna ,&nbsp;Zhuo Tina Yang ,&nbsp;Kendra Malone ,&nbsp;Elliot S. Rinzler ,&nbsp;Song Zhang ,&nbsp;Ayesha Zia","doi":"10.1016/j.rpth.2025.102728","DOIUrl":"10.1016/j.rpth.2025.102728","url":null,"abstract":"<div><h3>Background</h3><div>We hypothesized that acute pediatric venous thromboembolism (VTE) is associated with an increase in body mass index (BMI) over time. We posited that if children gain weight following VTE, targeted interventions may be advised clinically or tested in future studies.</div></div><div><h3>Objectives</h3><div>The objectives of our study were to investigate BMI changes from VTE diagnosis to 3 and 6 months after diagnosis, identify predictors, and calculate the prevalence of overweight and obesity.</div></div><div><h3>Methods</h3><div>: In this prospective cohort study, we followed 63 participants (mean age, 12.8 years [SD, 5]) for 6 months following first episode of acute VTE. We chose percentage of BMI₉₅ (%-of-BMI₉₅) instead of absolute BMI as a measure of weight to standardize across sex and age and used change in %-of-BMI₉₅ as a measure of weight change. Δ%-of-BMI₉₅ was the primary outcome measure documenting change over time, categorized as increased if Δ%-of-BMI₉₅ was &gt;0, unchanged if Δ%-of-BMI₉₅ was 0, and decreased if Δ%-of-BMI₉₅ was &lt;0. To assess BMI changes, we created a prespecified subgroup of participants who required intensive care unit (ICU) vs those who did not.</div></div><div><h3>Results</h3><div>Sixty-two percent of participants were overweight/obese at diagnosis. Mean %-of-BMI₉₅ was 102.5% (95% CI, 95-109). The Δ%-of-BMI₉₅ at 3 and 6 months after diagnosis was, 1.5 (95% CI, −0.8 to 3.6) and 2.2 (95% CI, −0.6 to 5.2), respectively. We identified 3 predictors of weight change: non-ICU stay and longer length of stay predicted weight gain, whereas a higher %-of-BMI₉₅ at diagnosis in the ICU cohort correlated with decreased BMI.</div></div><div><h3>Conclusion</h3><div>BMI increases following pediatric VTE except those in the ICU. Weight-based counseling and lifestyle changes represent potential targeted interventions after VTE.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 3","pages":"Article 102728"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143874513","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":"Residual pulmonary vascular obstruction computed with ventilation/perfusion single photon emission computed tomography/computed tomography to predict the risk of venous thromboembolism recurrence in patients with pulmonary embolism: protocol for a cohort study (PRONOSPECT)","authors":"Pierre–Yves Le Roux ,&nbsp;Philippe Robin ,&nbsp;Romain Le Pennec ,&nbsp;Cécile Tromeur ,&nbsp;Francis Couturaud ,&nbsp;Julien Asselineau ,&nbsp;Grégoire Le Gal ,&nbsp;Pierre–Yves Salaun","doi":"10.1016/j.rpth.2025.102867","DOIUrl":"10.1016/j.rpth.2025.102867","url":null,"abstract":"<div><h3>Background</h3><div>In patients with pulmonary embolism (PE), identifying predictors of recurrence is important to risk-stratify patients and tailor anticoagulation duration. After PE, a significant proportion of patients demonstrate residual pulmonary vascular obstruction (RPVO) on lung imaging. However, the exact prognostic significance of RPVO for venous thromboembolism recurrence remains unclear.</div></div><div><h3>Objectives</h3><div>The primary objective is to assess whether RPVO on ventilation/perfusion (V/Q) single photon emission computed tomography (SPECT)/CT imaging after completion of 3 to 6 months of anticoagulation is an independent predictor of venous thromboembolism recurrence in patients with PE.</div></div><div><h3>Methods</h3><div>The PRONOSPECT trial is a prospective multicenter cohort study. Participants are patients who experienced an objectively proven PE, provoked by a minor transient risk factor or unprovoked; who have been treated with anticoagulant therapy for 3 to 6 uninterrupted months; and for whom anticoagulation will not be prolonged. A standardized baseline patient assessment will be conducted including V/Q SPECT/CT imaging, collection of other potential predictor variables, and a functional evaluation. Anticoagulants will be withdrawn at the 3- or 6-month points from diagnosis and patients will be followed up for up to 2 years.</div></div><div><h3>Conclusion</h3><div>The PRONOSPECT cohort study has the potential to determine whether the presence of RPVO on V/Q SPECT/CT imaging predicts the risk of recurrence in patients with PE in whom there remains a doubt on duration of anticoagulation.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 3","pages":"Article 102867"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144070309","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":"Risk factors and prognosis of symptomatic postpartum ovarian vein thrombosis in 2 French prospective cohort studies","authors":"Eloïse Laouenan ,&nbsp;Claire de Moreuil ,&nbsp;Sara Robin ,&nbsp;Karine Morcel ,&nbsp;François Anouilh ,&nbsp;Cécile Tromeur ,&nbsp;Francis Couturaud ,&nbsp;Emmanuelle Le Moigne","doi":"10.1016/j.rpth.2025.102849","DOIUrl":"10.1016/j.rpth.2025.102849","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 3","pages":"Article 102849"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143879141","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":"Reply to “Reevaluating thromboprophylaxis in COVID-19 outpatients: the case for a targeted, risk-based approach”","authors":"Davide Di Vece ,&nbsp;Jean M. Connors ,&nbsp;Stefano Barco","doi":"10.1016/j.rpth.2025.102861","DOIUrl":"10.1016/j.rpth.2025.102861","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 3","pages":"Article 102861"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143937216","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":"A systematic review of direct oral anticoagulants for thromboprophylaxis in multiple myeloma","authors":"Cătălina Codreanu ,&nbsp;Tessa Elling ,&nbsp;Nic J.G. M. Veeger ,&nbsp;Wilfried W.H. Roeloffzen ,&nbsp;Karina Meijer","doi":"10.1016/j.rpth.2025.102865","DOIUrl":"10.1016/j.rpth.2025.102865","url":null,"abstract":"<div><div>Multiple myeloma (MM) is associated with increased venous thromboembolism (VTE) risk. Current guidelines recommend aspirin or low-molecular-weight heparin for thromboprophylaxis depending on VTE risk. Nevertheless, VTE risks remain high: a recent meta-analysis reported an incidence of 6.2% during the entire MM course. Direct oral anticoagulants (DOACs) showed promising results in other malignancies. This systematic review provides an overview of evidence on DOAC thromboprophylaxis in MM. PubMed and Embase were searched up to November 21, 2023, for studies evaluating MM and DOAC thromboprophylaxis (PROSPERO: CRD42022376152). Two authors independently screened titles, abstracts, and texts, assessed bias using a modified version of the Newcastle Ottawa Scale and certainty of evidence with the Grading of Recommendations Assessment, Development and Evaluation approach, and performed data extraction and analysis. Seven articles comprising 416 patients with DOAC thromboprophylaxis were included, primarily involving newly diagnosed patients with MM (56.3%) receiving lenalidomide-based regimens (69.1%). Overall Newcastle Ottawa Scale study quality was moderate. Four studies reported follow-up duration ranging from 90 days after induction to 7 months. VTE proportions ranged from 0% to 23.5%, with 4 studies reporting 0%. The proportions of minor, clinically relevant nonmajor, and major bleeding ranged from 0% to 18.2%, 0% to 7.7%, and 0% to 4.5%, respectively. Arterial thrombosis proportions ranged from 0% to 2.9%. Only 2 studies reported on mortality (2% and 7.1%). Overall Grading of Recommendations Assessment, Development and Evaluation certainty of evidence was very low for all outcomes. Current evidence regarding routine DOACs in MM is insufficient, warranting further research to establish the DOAC thromboprophylaxis risk-to-benefit ratio in MM.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 3","pages":"Article 102865"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143937525","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":"Reevaluating thromboprophylaxis in COVID-19 outpatients: the case for a targeted, risk-based approach","authors":"Chia Siang Kow ,&nbsp;Dinesh Sangarran Ramachandram ,&nbsp;Syed Shahzad Hasan ,&nbsp;Kaeshaelya Thiruchelvam","doi":"10.1016/j.rpth.2025.102862","DOIUrl":"10.1016/j.rpth.2025.102862","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 3","pages":"Article 102862"},"PeriodicalIF":3.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143906384","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":"Thiol isomerase ERp18 enhances platelet activation and arterial thrombosis","authors":"Chao He ,&nbsp;Aizhen Yang ,&nbsp;Keyu Lv ,&nbsp;Yuxin Zhang ,&nbsp;Zhenzhen Zhao ,&nbsp;Yi Lu ,&nbsp;Chao Fang ,&nbsp;Yue Han ,&nbsp;Depei Wu ,&nbsp;Miao Jiang ,&nbsp;Jingyu Zhang ,&nbsp;Yi Wu","doi":"10.1016/j.rpth.2025.102706","DOIUrl":"10.1016/j.rpth.2025.102706","url":null,"abstract":"<div><h3>Background</h3><div>Thiol isomerases regulate the thiol-disulfide exchange of functional proteins in cells. Using genetically modified mouse models and inhibitors, we and others demonstrated that 7 thiol isomerases (ERp57, protein diisulfide isomerase, ERp72, ERp46, ERp5, TMX4, and TMX1) participate in thrombosis. There are 21 thiol isomerases in mammals, but whether other enzymes of this family also contribute to thrombosis remains unknown.</div></div><div><h3>Objectives</h3><div>Investigate whether and how ERp18 participates in arterial thrombosis.</div></div><div><h3>Methods</h3><div>ERp18 knockout mice and arterial thrombosis models were used to determine the role of ERp18 in thrombosis. Platelets from ERp18 knockout mice were used to detect aggregation, activation, spreading, and clot retraction. Finally, flow cytometry and immunoprecipitation were used to detect the binding between ERp18 and α_IIbβ₃.</div></div><div><h3>Results</h3><div>The mice lacking ERp18 exhibited a prolonged tail bleeding time and decreased platelet thrombus formation in FeCl₃-induced carotid arterial injury and laser-induced cremaster artery injury models. ERp18 deficiency inhibited platelet aggregation, adenosine triphosphate release, integrin α_IIbβ₃ activation, P-selectin expression, platelet adhesion, as well as clot retraction. Flow cytometry and coimmunoprecipitation analyses revealed that ERp18 binds to the platelet surface via interaction with integrin α_IIbβ₃. Moreover, the ERp18 protein promoted the binding of integrin α_IIbβ₃ to fibrinogen and platelet aggregation. Furthermore, the recombinant ERp18 protein exhibited reductase activity and cleaved integrin α_IIbβ₃ disulfides.</div></div><div><h3>Conclusion</h3><div>ERp18 participates in platelet activation and thrombosis. Its function is, at least in part, through the regulation of integrin α_IIbβ₃ function. This finding expands our understanding of the role of thiol isomerases in the redox regulation of thrombosis and platelet function.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 2","pages":"Article 102706"},"PeriodicalIF":3.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143683442","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}

{"title":"Association between genetic risk and adherence to the Dietary Approaches to Stop Hypertension diet for developing venous thromboembolism","authors":"Si Li ,&nbsp;Minghui Jiang ,&nbsp;Yunlong Guan ,&nbsp;Xi Cao ,&nbsp;Zhonghe Shao ,&nbsp;Jun Deng ,&nbsp;Xingjie Hao","doi":"10.1016/j.rpth.2025.102731","DOIUrl":"10.1016/j.rpth.2025.102731","url":null,"abstract":"<div><h3>Background</h3><div>The relationship between diet and venous thromboembolism (VTE) remains unclear, and the joint effects of diet patterns and genetic susceptibility on VTE risk are unknown.</div></div><div><h3>Objectives</h3><div>Investigate the independent and joint effects of Dietary Approaches to Stop Hypertension (DASH) diet adherence and polygenic risk scores (PRS) on VTE risk.</div></div><div><h3>Methods</h3><div>A total of 411,539 UK Biobank participants were included. DASH scores were calculated using Food Frequency Questionnaires, and PRS quantified genetic risk. Cox proportional hazard models estimated hazard ratios (HRs) for VTE, assessing interactions between the DASH diet and genetic susceptibility.</div></div><div><h3>Results</h3><div>During a median follow-up of 13.4 years, 10,543 participants were diagnosed with VTE. Higher DASH scores were associated with a lower VTE risk (HR, 0.87; 95% CI, 0.82-0.92). A low-adherent DASH diet combined with high-genetic risk had the highest VTE risk (HR, 2.78; 95% CI, 2.47-3.14). High DASH scores reduced VTE risk in high-genetic-risk individuals (HR, 0.84; 95% CI, 0.76-0.92). Sex-specific associations were detected in the joint effect and interaction of DASH scores and PRS. Notably, high DASH scores can offset moderate genetic risk among men (HR, 0.79; 95% CI, 0.67-0.94). There were additive interactions between DASH scores and high genetic risk in total subjects and men, while not observed in women.</div></div><div><h3>Conclusion</h3><div>The DASH diet is associated with reduced VTE risk and can partially offset genetic predisposition. Low adherence to the DASH diet increases VTE risk, particularly in high-genetic-risk individuals. The protective effect of high DASH scores against genetic risks for VTE is more pronounced in males. Precision medicine should consider both diet and genetics for VTE prevention.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 2","pages":"Article 102731"},"PeriodicalIF":3.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143759589","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}

{"title":"Development of a novel gene editing lexicon for hemophilia: methodology and results","authors":"Craig M. Kessler ,&nbsp;Leonard A. Valentino ,&nbsp;Courtney D. Thornburg ,&nbsp;Carmen Unzu ,&nbsp;Mark A. Kay ,&nbsp;Flora Peyvandi ,&nbsp;Penni Smith ,&nbsp;Wolfgang Miesbach ,&nbsp;William McKeown ,&nbsp;Glenn F. Pierce ,&nbsp;Kate Khair ,&nbsp;Katarina Starcevic ,&nbsp;Monisha Pillai ,&nbsp;Micheala Jones ,&nbsp;Anil Sindhurakar ,&nbsp;Lauren Whyte ,&nbsp;Virginie Delwart ,&nbsp;Megan Chiao ,&nbsp;David E. Gutstein ,&nbsp;Ilia Antonino ,&nbsp;Steven W. Pipe","doi":"10.1016/j.rpth.2025.102710","DOIUrl":"10.1016/j.rpth.2025.102710","url":null,"abstract":"<div><h3>Background</h3><div>Clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9)-based targeted gene editing platforms are being developed to treat genetic diseases like hemophilia. Such novel therapy involves complex concepts and terminology that require aligned language to engage key stakeholders in the hemophilia community. Thus, a globally aligned gene editing lexicon – a consistent language to communicate the fundamentals of gene editing in hemophilia, designed to be credible and accessible for people with hemophilia and caregivers while avoiding unnecessary complexity – is required to address this need.</div></div><div><h3>Objectives</h3><div>To establish an aligned language and communications framework that facilitates informed consent and shared decision-making regarding gene editing and treatment considerations in hemophilia.</div></div><div><h3>Methods</h3><div>Through an innovative partnership with global experts in hemophilia, gene editing, and biotechnology, initial insights were gathered via interviews, workshops, and analysis of existing language within the hemophilia community. Qualitative research involving lived experience experts (people with hemophilia and caregivers; <em>n</em> = 43) and hematologists (<em>n</em> = 24) informed the lexicon development, which was further validated by a steering committee of global experts in the hemophilia and gene editing fields. Finally, optimized language recommendations were developed for a clear, consistent gene editing lexicon.</div></div><div><h3>Results</h3><div>Key themes included insights into audience mindsets, guiding language principles, and optimized terminology for key topics like gene editing concepts and posttreatment considerations. Audience mindsets revealed cautious optimism around gene therapy, with more skepticism around gene editing. Guiding language principles indicated a preference for plainspoken over technical language, definitions that link to patient benefits, and explanations that highlight the precise nature of gene editing.</div></div><div><h3>Conclusion</h3><div>This collaborative approach ensures broad adoption of the lexicon within the hemophilia community and readiness for beta testing.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 2","pages":"Article 102710"},"PeriodicalIF":3.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143747392","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}

{"title":"Global patterns of hemophilia drug trials, hemophilia care, and health care measures","authors":"Stacey A. Fedewa ,&nbsp;Leonard A. Valentino ,&nbsp;Andee Koo ,&nbsp;Lorraine Cafuir ,&nbsp;Theresa W. Gillespie ,&nbsp;Tyler W. Buckner ,&nbsp;Duc Q. Tran ,&nbsp;Ana Antun ,&nbsp;Christine L. Kempton","doi":"10.1016/j.rpth.2025.102714","DOIUrl":"10.1016/j.rpth.2025.102714","url":null,"abstract":"<div><h3>Background</h3><div>Drug trials are vital to establish safe and effective treatments for congenital hemophilia, a bleeding disorder that affects about 800,000 males worldwide. The global distribution of hemophilia drug trials (HDTs) and their alignment with hemophilia care is unknown.</div></div><div><h3>Objectives</h3><div>This study aimed to evaluate the global distribution of HDTs and its association with hemophilia care.</div></div><div><h3>Methods</h3><div>In this cross-sectional study, HDTs conducted between 2007 and 2022 were selected from the <span><span>clinicaltrials.gov</span><svg><path></path></svg></span> database. The density of trials per 1000 expected males with hemophilia (eMwH) was assessed according to hemophilia care measures (factor VIII and IX utilization per 1000 eMwH) derived from World Federation of Hemophilia data.</div></div><div><h3>Results</h3><div>Among 124 trials, 55 countries were represented, with an average of 7.9 countries per trial. Most HDT sites were in high-income (74.4%) or upper middle (20.1%)–income countries. The number of sites in lower-middle–income countries doubled, from 12 in 2007-2011 to 30 in 2017-2022—a nonsignificant increase from 5.8% to 7.0% (<em>P</em> = .53). Factor utilization was substantially reduced in lower-middle (0.4 international units [IUs] per 1000 eMwH) and upper middle (2.8 IUs per 1000 eMwH) compared with high (6.8 IUs per 1000 eMwH) income countries. HDT density was moderately correlated with factor usage (<em>r</em> = 0.436; <em>P</em> ≤ .001).</div></div><div><h3>Conclusion</h3><div>Most HDT sites were located in high-income countries, although a substantial proportion were in upper middle–income countries. A small but increasing number of trials were conducted in lower-middle–income countries, where factor usage is relatively low. This study provides evidence on the global distribution of HDT and raises questions regarding the generalizability, barriers, opportunities, and ethics of trials for a rare bleeding disorder.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"9 2","pages":"Article 102714"},"PeriodicalIF":3.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143705392","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}