Alexander Couzens, Frédéric Masclaux, Baptiste Micheli, Michel Guipponi, Alexandre Butelet, Colette Ballan-Legin, Alessandro Casini, Marguerite Neerman-Arbez
{"title":"Targeted long-read sequencing identifies a novel 1.3 kb FGG deletion associated with variable bleeding and thrombotic phenotypes in hypofibrinogenemia","authors":"Alexander Couzens, Frédéric Masclaux, Baptiste Micheli, Michel Guipponi, Alexandre Butelet, Colette Ballan-Legin, Alessandro Casini, Marguerite Neerman-Arbez","doi":"10.1016/j.rpth.2026.107001","DOIUrl":"10.1016/j.rpth.2026.107001","url":null,"abstract":"<div><h3>Background</h3><div>Congenital fibrinogen disorders are rare inherited disorders of fibrinogen quantity or function and are associated with variable bleeding and thrombotic phenotypes. They are caused by variants in <em>FGA</em>, <em>FGB</em>, or <em>FGG</em>. Most reported pathogenic variants are single-nucleotide variants or small insertions/deletions, whereas structural variants likely remain under-reported because short-read sequencing often cannot detect or fully resolve them.</div></div><div><h3>Objectives</h3><div>We aimed to identify and characterize the genetic cause of hypofibrinogenemia in three probands and their families and to investigate additional genetic variation potentially contributing to clinical variability.</div></div><div><h3>Methods</h3><div>Whole-exome sequencing (WES) identified variants in fibrinogen and other coagulation and fibrinolysis genes. Targeted Oxford Nanopore long-read sequencing resolved a suspected structural variant, defined its breakpoints, and enabled haplotype phasing across families. Kinship analysis assessed relatedness between probands.</div></div><div><h3>Results</h3><div>WES showed reduced read depth across <em>FGG</em> in three apparently unrelated probands. Long-read sequencing resolved a novel 1.3 kb deletion spanning the 5′ end of <em>FGG</em>, with breakpoints in A/T-rich low-complexity sequences. All three probands shared an identical 18 kb haplotype carrying the deletion, supporting inheritance from a common founder. Phenotypes ranged from mild bleeding to recurrent thrombosis. Compound heterozygosity for the deletion and <em>FGG</em> p.Ala108Gly was associated with a more marked reduction in fibrinogen levels than heterozygosity for the deletion alone. Additional rare variants in hemostasis-related genes were identified, although their contribution to phenotypic variability remains uncertain.</div></div><div><h3>Conclusion</h3><div>Long-read sequencing enables precise characterization and phasing of structural variants in congenital fibrinogen disorders, whereas WES facilitates investigation of additional genetic variation that may contribute to disease expression.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"10 7","pages":"Article 107001"},"PeriodicalIF":3.5,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149253983","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}
Kristina Lindsley, Arianna Nevo, Carmine Colavecchia, Lisa J. Wilcox, Priya Patel, Carlos Estevez, Cynthia Khanji, Jennifer Uyei, Steven W. Pipe, Guy Young
{"title":"Bleeding events in people with hemophilia B on prophylactic factor IX treatment: a systematic review and meta-analysis","authors":"Kristina Lindsley, Arianna Nevo, Carmine Colavecchia, Lisa J. Wilcox, Priya Patel, Carlos Estevez, Cynthia Khanji, Jennifer Uyei, Steven W. Pipe, Guy Young","doi":"10.1016/j.rpth.2026.106954","DOIUrl":"10.1016/j.rpth.2026.106954","url":null,"abstract":"<div><h3>Background</h3><div>Hemophilia B is a rare, X-linked disorder leading to deficient or dysfunctional clotting factor IX and bleeding complications.</div></div><div><h3>Objectives</h3><div>The objective of this systematic review and meta-analysis was to summarize bleeding events in people with hemophilia B of any severity without inhibitors receiving prophylactic factor IX replacement therapies to better understand disease burden and unmet needs.</div></div><div><h3>Methods</h3><div>A systematic search was conducted to identify clinical trials and observational studies reporting annualized bleeding rate (ABR), annualized joint bleeding rate, spontaneous bleeding events, traumatic bleeding events, and percentage of patients with zero-bleeding events. Bleeding outcomes were specified as total, treated, or unspecified and analyzed using the random-effects model. Subgroup meta-analyses assessed differences by age, treatment type, follow-up duration, and publication date, when feasible.</div></div><div><h3>Results</h3><div>The search yielded 5684 citations; 24 clinical trials and 53 observational studies were included in the meta-analysis. Treated ABR was 2.23 (95% CI, 1.52-3.27) in 13 trials; treated or unspecified ABR was 2.25 (95% CI, 1.67-3.03) in 22 trials. In 48 observational studies, treated or unspecified ABR was 2.08 (95% CI, 1.62-2.66). ABR was generally lower for patients <12 years of age (vs ≥12 years of age) and patients treated with extended half-life (vs standard half-life).</div></div><div><h3>Conclusion</h3><div>Results from this meta-analysis reflect advances in hemophilia B treatment, but also highlight unmet needs, with many patients experiencing ≥2bleeding events per year. Inconsistent ABR definitions and variability in follow-up times may introduce heterogeneity in the combined estimates.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"10 7","pages":"Article 106954"},"PeriodicalIF":3.5,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149213334","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}
Jérémy Datchanamourty, Antonio Morabito, Raphael Lhote, Siwar Hammami, Nanthara Sritharan, Matthieu Plichart, Sylvain Le Jeune
{"title":"Impact of an integrated hospital-community care pathway on the management of venous thromboembolism: the venous thromboembolic disease care pathway 93 study","authors":"Jérémy Datchanamourty, Antonio Morabito, Raphael Lhote, Siwar Hammami, Nanthara Sritharan, Matthieu Plichart, Sylvain Le Jeune","doi":"10.1016/j.rpth.2026.106956","DOIUrl":"10.1016/j.rpth.2026.106956","url":null,"abstract":"<div><h3>Background</h3><div>Venous thromboembolism (VTE) carries a high risk of recurrence and bleeding complications. Care transitions represent a vulnerable period, particularly in medically underserved areas where loss to follow-up is common. Integrated hospital-community care pathways may improve outcomes, but data are limited.</div></div><div><h3>Objectives</h3><div>To evaluate whether an integrated hospital–community care pathway for patients with venous thromboembolism was associated with improved clinical outcomes and continuity of care compared with standard care.</div></div><div><h3>Methods</h3><div>We conducted a single-center, retrospective, observational study of 161 consecutive patients managed for VTE in the day hospital at Avicenne Hospital (Bobigny, France) from December 2022 to December 2024. Patients enrolled in a structured city hospital care pathway (<em>n</em> = 64) were compared with those receiving standard care (<em>n</em> = 97). The primary endpoint was a composite of thrombotic recurrence, bleeding, hospitalization, or death. Inverse probability of treatment weighting was used to address confounding.</div></div><div><h3>Results</h3><div>The structured pathway was associated with a lower risk of the primary composite endpoint (hazard ratio [HR], 0.44; 95% CI, 0.21-0.93; <em>P</em> = .03), consistent with the inverse probability of treatment weighting analysis (HR, 0.44; 95% CI, 0.21-0.92; <em>P</em> = .03). Loss to follow-up was markedly lower in the structured pathway group (7.8% vs 30%; <em>P</em> = .002), and patients lost to follow-up had a 3.5-fold higher risk of complications (HR, 3.50; 95% CI, 1.71-7.18; <em>P</em> < .001). Enrollment in the pathway was associated with lower odds of loss to follow-up (odds ratio, 0.21; 95% CI, 0.07-0.53; <em>P</em> = .002).</div></div><div><h3>Conclusion</h3><div>In this single-center, retrospective cohort study, a structured city hospital care pathway was associated with a lower risk of composite complications and a lower loss-to-follow-up rate among patients managed for VTE. These findings support the potential value of integrated care coordination pathways, particularly in medically underserved areas.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"10 7","pages":"Article 106956"},"PeriodicalIF":3.5,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149162529","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}
Adeline Pontis, Alexandre Mansour, Axelle Maurice, Chloé Rousseau, Fabienne Nédelec-Gac, Pierre Guéret, Louis Soulat, Laure Maucorps, Anne Godier, Isabelle Gouin-Thibault
{"title":"Management of bleeding and nonelective invasive procedures in patients receiving direct oral anticoagulants (DOACs): a 5-year single-center observational study","authors":"Adeline Pontis, Alexandre Mansour, Axelle Maurice, Chloé Rousseau, Fabienne Nédelec-Gac, Pierre Guéret, Louis Soulat, Laure Maucorps, Anne Godier, Isabelle Gouin-Thibault","doi":"10.1016/j.rpth.2026.107003","DOIUrl":"10.1016/j.rpth.2026.107003","url":null,"abstract":"<div><h3>Background</h3><div>Guidelines exist for managing bleeding or nonelective invasive procedures in patients on direct oral anticoagulants (DOACs), but their clinical impact remains unclear.</div></div><div><h3>Objectives</h3><div>To describe the management of patients who had DOAC concentrations measured for bleeding or nonelective invasive procedures and to assess associations between guideline compliance, outcomes, and DOAC concentrations.</div></div><div><h3>Methods</h3><div>This retrospective, single-center study (2014-2018) included adult patients receiving dabigatran etexilate, rivaroxaban, or apixaban who required hospital care for bleeding or nonelective invasive procedures and had DOAC concentrations measured at the time of the event. Compliance with French and European guidelines was assessed. Adverse outcomes—a composite of all-cause mortality, worsening or new bleeding, and thrombotic events—were recorded until discharge.</div></div><div><h3>Results</h3><div>A total of 584 events (432 procedures and 152 bleeding episodes) in 547 patients were analyzed. Guideline compliance was 79% and showed no association with outcomes. Among 95 events treated with prothrombin complex concentrates, one thrombotic event occurred. DOAC concentrations were higher in the bleeding group than in the procedural group (122 [IQR, 57-219] vs 55 [IQR, 25-127] ng/mL), but bleeding severity was not associated with drug levels. In bleeding patients, rivaroxaban concentrations were higher in those with adverse outcomes compared with those without (243 [IQR, 74-367] vs 88 [IQR, 35-199] ng/mL).</div></div><div><h3>Conclusion</h3><div>In a large cohort of patients who had DOAC concentrations measured at the time of a bleeding or a nonelective invasive procedure, compliance with guidelines was high and not associated with outcomes. In bleeding patients, rivaroxaban concentrations were higher in those with vs without adverse events.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"10 7","pages":"Article 107003"},"PeriodicalIF":3.5,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149283034","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}
Marius Brühl, Fabian Tomschi, Andreas Christian Strauss, Joschua Wiese, Pia Möllers, Alexander Schmidt, Thomas Hilberg
{"title":"Biomechanical viscoelastic properties in leg muscles of people with hemophilia: a case control study","authors":"Marius Brühl, Fabian Tomschi, Andreas Christian Strauss, Joschua Wiese, Pia Möllers, Alexander Schmidt, Thomas Hilberg","doi":"10.1016/j.rpth.2026.107000","DOIUrl":"10.1016/j.rpth.2026.107000","url":null,"abstract":"<div><h3>Background</h3><div>People with hemophilia (PwH) are prone to joint bleeds, resulting in hemophilic arthropathy, potentially altering muscle tissue. Although functional and morphologic impairments are documented, little is known about biomechanical muscle properties in PwH.</div></div><div><h3>Objectives</h3><div>This study investigates muscle stiffness in relaxed and contracted states in the lower limbs of adult PwH and its relationship to muscle strength and joint health.</div></div><div><h3>Methods</h3><div>In this cross-sectional case-control study, 40 PwH and 20 healthy controls were assessed. Using myotonometry, stiffness of 8 lower limb muscles was measured under relaxed (<em>S</em><sub><em>rel</em></sub>) and maximum voluntarily contracted conditions (<em>S</em><sub><em>con</em></sub>). Stiffness increase rate (<em>SIR</em>) was calculated. Joint health was assessed via the Hemophilia Joint Health Score and sonography (Hemophilia Early Arthropathy Detection with Ultrasound), and muscle strength was measured using a mobile isometric force gauge. PwH were stratified by lower limb joint impairment (<em>PwH</em><sub><em>minor</em></sub> vs <em>PwH</em><sub><em>major</em></sub>).</div></div><div><h3>Results</h3><div><em>PwH</em><sub><em>major</em></sub> exhibited significantly increased <em>S</em><sub><em>rel</em></sub> in anterior thigh and calf muscles compared with controls and <em>PwH</em><sub><em>minor</em></sub> (all <em>P</em> ≤ .05, <em>r</em> = 0.397-1.010). Under contraction, <em>PwH</em><sub><em>major</em></sub> showed reduced <em>S</em><sub><em>con</em></sub> in the tibialis anterior (<em>P</em> ≤ .05, <em>r</em> = 0.511-0.683) and biceps femoris (<em>P</em> = .04, <em>r</em> = 0.390). The <em>SIR</em> was significantly lower in <em>PwH</em><sub><em>major</em></sub> across most muscles (all <em>P</em> ≤ .05, <em>r</em> = 0.380-0.573), indicating impaired muscle contractility. Furthermore, muscle strength correlated negatively with <em>S</em><sub><em>rel</em></sub> (<em>r</em><sub><em>s</em></sub> = –0.220 to –0.430) and positively with both <em>S</em><sub><em>con</em></sub> and <em>SIR</em> (<em>r</em><sub><em>s</em></sub> = 0.273-0.449). Hemophilia Joint Health Score correlated with myotonometric parameters (<em>r</em><sub><em>s</em></sub> = 0.248-0.683), linking joint damage to altered muscle properties.</div></div><div><h3>Conclusion</h3><div>Advanced hemophilic arthropathy is associated with increased passive stiffness and reduced active muscle response, particularly in stabilizing muscle groups. Myotonometry may serve as a potential supplementary tool for the exploratory assessment of biomechanical muscle status in PwH.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"10 7","pages":"Article 107000"},"PeriodicalIF":3.5,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149283036","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}
Davide Sacchet, Corrado Lodigiani, Clara Sacco, Stefano Barco, Simon Wolf, Sandrine Zweifel, Marco Paolo Donadini, Luca Librè, Lorenza Bertù, Walter Ageno
{"title":"Long-term outcomes of antithrombotic treatment for retinal vein occlusion","authors":"Davide Sacchet, Corrado Lodigiani, Clara Sacco, Stefano Barco, Simon Wolf, Sandrine Zweifel, Marco Paolo Donadini, Luca Librè, Lorenza Bertù, Walter Ageno","doi":"10.1016/j.rpth.2026.106976","DOIUrl":"10.1016/j.rpth.2026.106976","url":null,"abstract":"<div><h3>Background</h3><div>Retinal vein occlusion (RVO) is a leading cause of visual impairment. Several cardiovascular and thrombotic predisposing factors have been linked to RVO. Antithrombotic agents are commonly prescribed, but their effectiveness for visual recovery and long-term outcomes remains uncertain.</div></div><div><h3>Objectives</h3><div>To study the course of long-term clinical outcomes in RVO patients.</div></div><div><h3>Methods</h3><div>In this multicenter, retrospective, observational study of patients with an index RVO at 3 academic hospitals, we estimated the risk of recurrent RVO, cardiovascular events, venous thromboembolic events, bleeding complications, and death, reported as incidence rates per 100 person-years, and compared these rates between patients treated with antithrombotic drugs and those untreated.</div></div><div><h3>Results</h3><div>Between 2004 and 2023, 198 patients were enrolled (median age, 65 years; IQR, 54-75; 105 females, 53.0%) and followed for a median of 2.6 years (IQR, 0.9-4.5); of these, 39.4% received antiplatelet therapy, 16.6% received anticoagulant therapy, and 44.0% received no antithrombotic treatment. The median duration of anticoagulant therapy was 46 days (IQR, 30-158), while that of antiplatelet therapy was 296 days (IQR, 124-947). The incidence rate ratios (IRRs) for cardiovascular events and mortality were higher in the untreated group (IRR, 6.04; 95% CI, 1.17-38.9 for cardiovascular events; IRR, 3.89; 95% CI, 1.29-12.3 for mortality) than in patients receiving antithrombotic treatment. For RVO recurrence and hemorrhagic complications, we found no differences between treated and untreated patients (IRR, 1.02; 95% CI, 0.18-3.97 for recurrent RVO; IRR, 1.12; 95% CI, 0.4-2.76 for bleeding complications).</div></div><div><h3>Conclusion</h3><div>In this real-world cohort study of RVO, our data suggest that patients with RVO should undergo careful cardiovascular risk assessment. Antithrombotic therapy should not be used routinely after RVO, only when indicated by established cardiovascular prevention principles or specific clinical circumstances. Notably, rates of recurrent RVO and bleeding complications were similar between treated and untreated patients.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"10 7","pages":"Article 106976"},"PeriodicalIF":3.5,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149253993","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}
Hyung-Jun Lim, Kunye Kwak, Changgon Kim, Yoon Seok Choi, Yong Park, Byung Soo Kim, Ki Hoon Ahn, Ka-Won Kang
{"title":"Treatment patterns and safety outcomes after splenectomy or thrombopoietin receptor agonists as second-line therapy for immune thrombocytopenia: Korean claims-based study","authors":"Hyung-Jun Lim, Kunye Kwak, Changgon Kim, Yoon Seok Choi, Yong Park, Byung Soo Kim, Ki Hoon Ahn, Ka-Won Kang","doi":"10.1016/j.rpth.2026.106992","DOIUrl":"10.1016/j.rpth.2026.106992","url":null,"abstract":"<div><h3>Background</h3><div>Splenectomy and thrombopoietin receptor agonists (TPO-RAs) are recommended second-line therapies for immune thrombocytopenia. Although guidelines recommend tailoring second-line treatments to patient characteristics, this approach is often unclear in real-world practice.</div></div><div><h3>Objectives</h3><div>We examined claims-based time to next treatment (TTNT) and safety outcomes after splenectomy or TPO-RA as a second-line therapy using nationally representative Korean claims data.</div></div><div><h3>Methods</h3><div>Patients diagnosed with immune thrombocytopenia between 2002 and 2021 were included. From the eligible cohort, we identified patients whose index second-line treatment was splenectomy (group 1) or TPO-RA without prior splenectomy (group 2). Propensity score matching was performed between patients in both groups who had received corticosteroids as first-line treatment.</div></div><div><h3>Results</h3><div>After matching, 447 group 1 and 57 group 2 patients were analyzed. Subsequent treatment was administered to 82.3% and 61.4% of patients in groups 1 and 2, respectively. Median TTNTs were 5.80 and 3.03 months in groups 1 and 2, respectively. Estimated TTNT curves diverged after 24 months, with a longer TTNT observed in the TPO-RA group. Pneumococcal infections occurred in 3.1% of patients in group 1 and none in group 2. Venous thromboembolism (VTE) occurred in 10.5% and 15.7% of patients in groups 1 and 2, respectively. The observed time to VTE was shorter in group 2 than in group 1.</div></div><div><h3>Conclusion</h3><div>Patients treated with TPO-RAs as a second-line therapy showed a longer TTNT after 24 months, whereas VTE occurred earlier in the TPO-RA group. However, these findings should be interpreted with caution because of residual confounding.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"10 7","pages":"Article 106992"},"PeriodicalIF":3.5,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149213331","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}
Jian Xiao, Jie Ji, Hai Yi, Lei Ma, Xiaodong Liu, Lan Li, Shan Luo, Fang Hua, Kun Chen
{"title":"Hetrombopag in promoting platelet engraftment after hematopoietic stem cell transplantation: a multicenter, single-arm, phase 2 study","authors":"Jian Xiao, Jie Ji, Hai Yi, Lei Ma, Xiaodong Liu, Lan Li, Shan Luo, Fang Hua, Kun Chen","doi":"10.1016/j.rpth.2026.106967","DOIUrl":"10.1016/j.rpth.2026.106967","url":null,"abstract":"<div><h3>Background</h3><div>Delayed platelet engraftment remains a frequent and clinically significant complication following hematopoietic stem cell transplantation (HSCT), contributing to increased morbidity and mortality.</div></div><div><h3>Objective</h3><div>This study aimed to evaluate the efficacy and safety of hetrombopag, a novel oral thrombopoietin receptor agonist, in promoting platelet engraftment after HSCT.</div></div><div><h3>Methods</h3><div>This multicenter, single-arm, phase 2 trial enrolled patients aged ≥14 years who underwent allogeneic or autologous HSCT. Hetrombopag was administered orally at 5 mg/d from day +3 to +7 posttransplant until platelet engraftment was achieved. The primary endpoint was the time to platelet engraftment, defined as a platelet count >20 × 10<sup>9</sup>/L for at least 3 consecutive days without transfusion support.</div></div><div><h3>Results</h3><div>A total of 48 patients were included, comprising 28 autologous and 19 allogeneic HSCT recipients, with 1 umbilical cord blood transplant. All patients achieved both platelet and neutrophil engraftment. The median time to platelet engraftment was 13 days (range, 8-40), and the median time to neutrophil engraftment was 11 days (range, 8-20). Thirty-eight patients (79.2%) achieved a platelet count of ≥50 × 10<sup>9</sup>/L within a median of 16 days (range, 9-56), and 25 (52.1%) patients achieved a platelet count of ≥100 × 10<sup>9</sup>/L within 20 days (range, 11-89). The median cumulative platelet transfusion volume was 3 units (range, 0-24). Hetrombopag was well tolerated; no thrombotic events or treatment-related deaths occurred. The most common adverse events were mild elevations in transaminase (33.3%) and bilirubin (37.5%) levels.</div></div><div><h3>Conclusion</h3><div>Hetrombopag was associated with favorable platelet engraftment and low transfusion requirements following HSCT and demonstrated a favorable safety and tolerability profile.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"10 7","pages":"Article 106967"},"PeriodicalIF":3.5,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149078503","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":"A crowded podium in hemophilia A: when a low annualized bleeding rate is no longer enough to tell the contenders apart","authors":"Laurent Frenzel","doi":"10.1016/j.rpth.2026.106942","DOIUrl":"10.1016/j.rpth.2026.106942","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"10 7","pages":"Article 106942"},"PeriodicalIF":3.5,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149162530","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 of dual-pathway antithrombotic therapy with primary patency after iliofemoral venous stenting: a multicenter cohort study","authors":"Can Chen, Zhenyu Guo, Jian Tang, Zhiqing Wei, Guowei Liu, Zhenyu Shi, Daqiao Guo, Weiguo Fu, Qianzhou Lv, Baolei Guo","doi":"10.1016/j.rpth.2026.106965","DOIUrl":"10.1016/j.rpth.2026.106965","url":null,"abstract":"<div><h3>Background</h3><div>The optimal antithrombotic strategy after iliofemoral venous stenting remains uncertain.</div></div><div><h3>Objectives</h3><div>This study aimed to evaluate the association of dual-pathway antithrombotic therapy with primary patency after iliofemoral venous stenting.</div></div><div><h3>Methods</h3><div>This ambispective, multicenter observational study included consecutive patients who underwent successful iliofemoral venous stent implantation between 2019 and 2024. Patients were categorized according to their postoperative antithrombotic regimen into dual-pathway antithrombotic therapy (anticoagulation plus antiplatelet therapy [AC+APT]) and anticoagulation (AC) alone groups. The primary outcome was primary stent patency. Cox regression was used to identify independent predictors of loss of patency.</div></div><div><h3>Results</h3><div>A total of 247 patients were included, with 145 receiving AC+APT and 102 receiving AC alone. During a median follow-up of 24 months (IQR, 15-38 months), the overall primary patency rate was 91.1%. Primary patency did not differ significantly between the AC+APT and AC groups in the overall cohort (log-rank <em>P</em> = 1.00). However, in the post-thrombotic syndrome subgroup, AC alone was associated with a higher risk of patency loss compared with AC+APT (hazard ratio, 3.31; 95% CI, 1.02-10.74; <em>P</em> = .047).</div></div><div><h3>Conclusion</h3><div>Primary patency after iliofemoral venous stenting was similarly high with dual-pathway antithrombotic therapy and AC alone. An exploratory analysis suggested a possible association between AC+APT and improved patency in patients with post-thrombotic syndrome. Adequately powered randomized trials with longer follow-up are needed to confirm this finding and assess long-term safety.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":"10 7","pages":"Article 106965"},"PeriodicalIF":3.5,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149162564","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}