Sylvain Lamoine , Vincent Jury , Virginie Fourneyron , Jonathan Douxfils , Dorian Teissandier , Laurie Talon , Thomas Sinegre , Aurélien Lebreton
{"title":"Thrombin generation to evaluate the complex hemostatic balance of hemophilia A plasma containing direct oral anticoagulant and supplemented by factor VIII","authors":"Sylvain Lamoine , Vincent Jury , Virginie Fourneyron , Jonathan Douxfils , Dorian Teissandier , Laurie Talon , Thomas Sinegre , Aurélien Lebreton","doi":"10.1016/j.rpth.2024.102576","DOIUrl":"10.1016/j.rpth.2024.102576","url":null,"abstract":"<div><h3>Background</h3><div>The incidence of cardiovascular diseases is increasing in persons with hemophilia A (HA). Therefore, anticoagulant therapy based on direct oral anticoagulants (DOACs) may be needed, despite the bleeding risk. In case of surgery or bleeding, such patients may be concomitantly treated with emicizumab (routine prophylaxis), factor (F)VIII products, and DOAC. Their concomitant presence constitutes a hemostatic challenge. Recent international guidelines stated that data are scarce on the hemostatic balance of plasma samples from patients with HA receiving emicizumab and DOAC.</div></div><div><h3>Objectives</h3><div>The aim of this observational study was to assess the coagulation of FVIII-deficient plasma spiked with DOAC and emicizumab and to evaluate the effects of FVIII addition.</div></div><div><h3>Methods</h3><div>Prothrombin time, activated partial thromboplastin time, and thrombin generation (TG) using the calibrated automated thrombogram method were evaluated in aliquots of a commercial severe HA plasma supplemented with emicizumab (0, 12.5, 25, 50, and 100 ng/mL), DOAC (0, 50, 100, 200, and 400 ng/mL of apixaban, rivaroxaban, edoxaban, or dabigatran) and FVIII (0%, 5%, 15%, 50%, and 100%).</div></div><div><h3>Results</h3><div>DOAC rapidly induced a TG decrease. Emicizumab could counter this effect only for the lowest DOAC dose. FVIII addition to the FVIII-deficient plasma containing a DOAC and emicizumab improved TG and countered the anticoagulant effect of DOAC at ≤100 ng/mL.</div></div><div><h3>Conclusion</h3><div>Our findings indicate that FVIII can be safely used with emicizumab to counter the anticoagulant effect of DOAC at ≤100 ng/mL. The TG assay is an efficient tool to monitor plasma containing anti-FXa DOAC, but not dabigatran (anti-FIIa).</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142445224","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}
Nicolas Gendron , Dominique Helley , Philippe Rousselot , Virginie Siguret , Pascale Gaussem , Chloé James , Lina Khider , Nadine Ajzenberg , Elodie Boissier , Nicolas Boissel , David M. Smadja , Benjamin Planquette
{"title":"Evaluation of hemostasis understanding in medical and pharmacy students from a Parisian university","authors":"Nicolas Gendron , Dominique Helley , Philippe Rousselot , Virginie Siguret , Pascale Gaussem , Chloé James , Lina Khider , Nadine Ajzenberg , Elodie Boissier , Nicolas Boissel , David M. Smadja , Benjamin Planquette","doi":"10.1016/j.rpth.2024.102547","DOIUrl":"10.1016/j.rpth.2024.102547","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924002425/pdfft?md5=34202f20a3eeb2182fdfd224e051812c&pid=1-s2.0-S2475037924002425-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142158269","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}
Michael Hardy , Emilie Catry , Marie Pouplard , Thomas Lecompte , François Mullier
{"title":"Is lupus anticoagulant testing with dilute Russell’s viper venom clotting times reliable in the presence of inflammation?","authors":"Michael Hardy , Emilie Catry , Marie Pouplard , Thomas Lecompte , François Mullier","doi":"10.1016/j.rpth.2024.102536","DOIUrl":"10.1016/j.rpth.2024.102536","url":null,"abstract":"<div><h3>Background</h3><p>Testing for lupus anticoagulant (LA) is not recommended in case of inflammation as C-reactive protein (CRP) can interfere <em>in vitro</em> with the phospholipids present in the activated partial thromboplastin time test used to detect an LA. However, the potential interference of an acute phase protein (ie, CRP) in LA testing using the dilute Russell’s viper venom (DRVV) test is poorly studied.</p></div><div><h3>Objectives</h3><p>To study the effect of inflammation, as evidenced by increased CRP levels, on DRVV tests.</p></div><div><h3>Methods</h3><p>First, a retrospective analysis (2013-2023) was performed: data on all LA workups were retrieved, and the association between CRP levels and DRVV screen, mix, and confirm clotting times was studied. Second, data on DRVV panels and CRP levels were extracted from 2 prospective studies involving intensive care unit patients to study the association between both variables. Third, CRP was added to normal pooled plasma at 6 relevant concentrations (up to 416 mg/L) to study the association between CRP itself and DRVV coagulation times.</p></div><div><h3>Results</h3><p>In the retrospective analysis, DRVV screen and confirm clotting times significantly increased as CRP increased (increase of 0.11 seconds and 0.03 seconds per 1 mg/L increase of CRP level, respectively). In the prospective analysis, only DRVV screen was prolonged with high CRP levels (increase of 0.06 seconds for a 1 mg/L increase in CRP level); DRVV screen/confirm ratio was also increased with high CRP levels. <em>In vitro</em>, the addition of CRP did not significantly increase any DRVV clotting times.</p></div><div><h3>Conclusion</h3><p>LA testing should be performed with much caution in the presence of inflammation as it may be associated with prolongation of both activated partial thromboplastin time and DRVV clotting times.</p></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924002310/pdfft?md5=74e746b83b6a5631d7903e0ed7aa6f1b&pid=1-s2.0-S2475037924002310-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141842116","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}
S.F.B. van der Horst , Y. de Jong , N. van Rein , J.W. Jukema , M. Palmen , E. Janssen , E.F. Bonneville , F.A. Klok , M.V. Huisman , L.F. Tops , P.L. den Exter
{"title":"Erratum to ‘Performance of Risk Scores in Predicting Major Bleeding in Left Ventricular Assist Device (LVAD) Recipients: a Comparative External Validation. ’[Res Pract Thromb Haemost. 2024;8:e102437]","authors":"S.F.B. van der Horst , Y. de Jong , N. van Rein , J.W. Jukema , M. Palmen , E. Janssen , E.F. Bonneville , F.A. Klok , M.V. Huisman , L.F. Tops , P.L. den Exter","doi":"10.1016/j.rpth.2024.102558","DOIUrl":"10.1016/j.rpth.2024.102558","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S247503792400253X/pdfft?md5=f935f34e505b825f1063b17253406f03&pid=1-s2.0-S247503792400253X-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142098440","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}
Julia S. Gauer , Abigail Ajanel , Lutale M. Kaselampao , Isabel Candir , Amanda D.V. MacCannell , Lee D. Roberts , Robert A. Campbell , Robert A.S. Ariëns
{"title":"Plant-derived compounds normalize platelet bioenergetics and function in hyperglycemia","authors":"Julia S. Gauer , Abigail Ajanel , Lutale M. Kaselampao , Isabel Candir , Amanda D.V. MacCannell , Lee D. Roberts , Robert A. Campbell , Robert A.S. Ariëns","doi":"10.1016/j.rpth.2024.102548","DOIUrl":"10.1016/j.rpth.2024.102548","url":null,"abstract":"<div><h3>Background</h3><p>Polyphenols have been shown to decrease oxidative stress and modulate glycemic response. Nevertheless, their effect on platelet bioenergetics and clot structure in diabetes and hyperglycemia is unknown.</p></div><div><h3>Objectives</h3><p>To investigate the effect of polyphenols on human platelet bioenergetics and its subsequent effect on clot structure in normoglycemia vs acute hyperglycemia <em>in vitro</em>.</p></div><div><h3>Methods</h3><p>Four polyphenols (resveratrol, hesperetin, epigallocatechin gallate [EGCG], and quercetin) were selected for initial analysis. Healthy volunteers’ isolated platelets/platelet-rich plasma were treated with 5 or 25 mM glucose to represent normoglycemia and acute hyperglycemia, respectively. Platelet-derived reactive oxygen species (ROS), citrate synthase activity (mitochondrial density), mitochondrial calcium flux, and mitochondrial respiration were performed following exposure to polyphenols (20 µM, 1 hour) to determine their effects on platelet bioenergetics. Procoagulant platelets (annexin V) and fibrin fiber density (Alexa Fluor-488 fibrinogen; Invitrogen) were analyzed by laser scanning confocal microscopy, while clot porosity was determined using platelet-rich plasma following exposure to polyphenols (20 µM, 20 minutes).</p></div><div><h3>Results</h3><p>Acute hyperglycemia increased ROS, mitochondrial calcium flux, maximal respiration, and procoagulant platelet number. Resveratrol, quercetin, and EGCG reduced platelet ROS in normoglycemic and acute hyperglycemic conditions. Mitochondrial density was decreased by quercetin and EGCG in normoglycemia. Resveratrol and EGCG reduced mitochondrial calcium flux in acute hyperglycemia. Resveratrol also decreased procoagulant platelet number and attenuated oxygen consumption rate in normoglycemia and acute hyperglycemia. No effect of hyperglycemia or polyphenols was observed on fibrin fiber density or clot pore size.</p></div><div><h3>Conclusion</h3><p>Our results suggest polyphenols attenuate increased platelet activity stemming from hyperglycemia and may benefit thrombosis-preventative strategies in patients with diabetes.</p></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924002437/pdfft?md5=da81283ebae9bbdd6adf35c823a86f11&pid=1-s2.0-S2475037924002437-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142167785","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":"Can hemophilia be cured? It depends on the definition","authors":"Lieke Baas , Rieke van der Graaf , Karina Meijer","doi":"10.1016/j.rpth.2024.102559","DOIUrl":"10.1016/j.rpth.2024.102559","url":null,"abstract":"<div><div>Over the years, the palette of treatment options for hemophilia has grown extensively, leading to an increased life expectancy and quality of life for people living with hemophilia. Nonetheless, it is frequently emphasized that none of the current treatment modalities provides a “cure.” It is therefore hoped that innovative treatments such as gene therapy may bridge this void. However, the precise definition of a “cure” for hemophilia remains unclear. In this review, we show how the concept of cure is currently used in the field of hemophilia. We then relate the discussion on cure to debates surrounding the classification of hemophilia and philosophical debates on the concepts of health and disease.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142319149","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}
Bilgimol Chumappumkal Joseph , Tro Sekayan , Nicca Falah , Richard F.W. Barnes , Veronica Flood , Juan A. De Pablo-Moreno , Annette von Drygalski
{"title":"Traumatic bleeding and mortality in mice are intensified by iron deficiency anemia and can be rescued with tranexamic acid","authors":"Bilgimol Chumappumkal Joseph , Tro Sekayan , Nicca Falah , Richard F.W. Barnes , Veronica Flood , Juan A. De Pablo-Moreno , Annette von Drygalski","doi":"10.1016/j.rpth.2024.102543","DOIUrl":"10.1016/j.rpth.2024.102543","url":null,"abstract":"<div><h3>Background</h3><p>Clinical evidence suggests that anemia exacerbates traumatic bleeding and worsens outcomes.</p></div><div><h3>Objectives</h3><p>To study the influence of iron deficiency anemia on traumatic bleeding, coagulopathy, and mortality.</p></div><div><h3>Methods</h3><p>C57BL/6J mice received an iron-deficient diet (8 weeks; ±1 mg intraperitoneal iron dextran 2 weeks before trauma). Control mice received a normal diet. Iron deficiency anemia was confirmed by hematocrit, red cell indices, and liver iron. Mice received saline or tranexamic acid (TXA; 10 mg/kg) just before liver laceration. Blood loss, coagulopathy (activated partial thromboplastin time, factor [F]II, FV, FVIII, FX, and fibrinogen), D-dimer, thrombin-antithrombin complexes, and plasmin-alpha-2-antiplasmin complexes were analyzed at 15 and 60 minutes, and a cytokine panel was performed at 60 minutes and 6 hours after trauma. Survival was monitored for 7 days.</p></div><div><h3>Results</h3><p>Compared with nonanemic mice, anemic mice had lower hematocrit and hepatic iron content. Anemic mice experienced higher blood loss compared with nonanemic mice, which was reduced by TXA. Both groups developed traumatic coagulopathy characterized by activated partial thromboplastin time prolongation, thrombin-antithrombin complex formation, and depletion of FV, FVIII, and fibrinogen. TXA corrected the coagulopathy. However, plasmin-alpha-2-antiplasmin complex formation and D-dimers, markers of fibrinolysis, were higher in anemic mice and were not corrected by TXA. Seven-day survival was low in anemic mice, and rescued by TXA, but high in nonanemic mice without additional improvement by TXA. Among cytokines, only interleukin-6 increased, which was prevented by TXA most notably in anemic mice.</p></div><div><h3>Conclusion</h3><p>These observations provide first and critical proof-of-principle evidence that anemia accelerates traumatic bleeding and increases mortality, which could be rescued by anemia correction (parenteral iron) or periprocedural TXA.</p></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924002383/pdfft?md5=8c627950af83952d283afc85b92939ac&pid=1-s2.0-S2475037924002383-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142122799","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}
Marie-Astrid van Dievoet , Karim Zouaoui Boudjeltia , Madeleine Rousseaux , Jonathan Douxfils , Ton Lisman , Xavier Stephenne
{"title":"Fibrinolytic profile depends on disease severity in pediatric patients with cirrhosis: illustration by 2 different plasma-based fibrinolysis assays","authors":"Marie-Astrid van Dievoet , Karim Zouaoui Boudjeltia , Madeleine Rousseaux , Jonathan Douxfils , Ton Lisman , Xavier Stephenne","doi":"10.1016/j.rpth.2024.102551","DOIUrl":"10.1016/j.rpth.2024.102551","url":null,"abstract":"","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924002462/pdfft?md5=6d10eb640300c8d1535d98f889f2a52a&pid=1-s2.0-S2475037924002462-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142151133","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}
David Mutschlechner , Maximilian Tscharre , Franziska Wittmann , Daniela Kitzmantl , Thomas Schlöglhofer , Patricia Pia Wadowski , Günther Laufer , Beate Eichelberger , Silvia Lee , Dominik Wiedemann , Simon Panzer , Daniel Zimpfer , Thomas Gremmel
{"title":"Platelet reactivity is associated with pump thrombosis in patients with left ventricular assist devices","authors":"David Mutschlechner , Maximilian Tscharre , Franziska Wittmann , Daniela Kitzmantl , Thomas Schlöglhofer , Patricia Pia Wadowski , Günther Laufer , Beate Eichelberger , Silvia Lee , Dominik Wiedemann , Simon Panzer , Daniel Zimpfer , Thomas Gremmel","doi":"10.1016/j.rpth.2024.102564","DOIUrl":"10.1016/j.rpth.2024.102564","url":null,"abstract":"<div><h3>Background</h3><div>Patients with left ventricular assist devices (LVADs) are treated with a potent antithrombotic regimen to prevent pump thrombosis and thromboembolism. High on-treatment residual platelet reactivity (HRPR) is associated with ischemic outcomes in cardiovascular disease.</div></div><div><h3>Objectives</h3><div>In the current study, we investigated the prevalence and clinical impact of HRPR in stable LVAD patients.</div></div><div><h3>Methods</h3><div>Pump thrombosis, bleeding events, and death were assessed in 62 LVAD patients (19 HeartWare HVAD [Medtronic] and 43 HeartMate 3 [Abbott]) during a 2-year follow-up. Platelet aggregation was measured by multiple electrode aggregometry, and HRPR was defined as arachidonic acid (AA)–inducible platelet aggregation of ≥21 aggregation units. Soluble P-selectin was determined by enzyme-linked immunosorbent assay.</div></div><div><h3>Results</h3><div>Three patients (4.8%) had pump thrombosis and 10 patients (16.1%) suffered a bleeding complication. AA-inducible platelet aggregation was significantly higher in patients with pump thrombosis (<em>P</em> = .01), whereas platelet aggregation in response to adenosine diphosphate (ADP) and thrombin receptor–activating peptide (TRAP) was comparable between patients without and those with pump thrombosis (both <em>P</em> > .05). Platelet aggregation in response to AA, ADP, and TRAP was similar in patients without and with a bleeding event (all <em>P</em> > .05). HRPR was detected in 29 patients (46.8%) and was associated with significantly higher platelet aggregation in response to AA, ADP, and TRAP as well as higher levels of soluble P-selectin compared with patients without HRPR (all <em>P</em> < .05). All pump thromboses occurred in patients with HRPR (3 vs 0; <em>P</em> = .06) and HVAD.</div></div><div><h3>Conclusion</h3><div>Platelet reactivity is associated with pump thrombosis in LVAD patients. HRPR may represent a risk marker for pump thrombosis, particularly in HVAD patients.</div></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142327541","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}
Csaba Siffel , Abhinav Wadhwa , Vanita Tongbram , Margaret Katana Ogongo , Henrik Sliwka , Hanna T. Gazda , Peter L. Turecek
{"title":"Comprehensive literature review of protein C concentrate use in patients with severe congenital protein C deficiency","authors":"Csaba Siffel , Abhinav Wadhwa , Vanita Tongbram , Margaret Katana Ogongo , Henrik Sliwka , Hanna T. Gazda , Peter L. Turecek","doi":"10.1016/j.rpth.2024.102542","DOIUrl":"10.1016/j.rpth.2024.102542","url":null,"abstract":"<div><p>Severe congenital protein C deficiency (SCPCD) is a rare disorder associated with life-threatening purpura fulminans and disseminated intravascular coagulation that typically present within hours after birth. Treatment options for patients with SCPCD include replacement therapy with a plasma-derived protein C concentrate. In this targeted literature review, we summarize information on the use of protein C concentrate as long-term prophylaxis (>1 week of treatment) for patients with SCPCD. In total, 18 publications were included in the review, of which 15 were case studies. Treatment with protein C concentrate (Ceprotin; Baxalta US Inc, a Takeda company; Takeda Manufacturing Austria AG) was reported in 11 publications, and treatment with protein C concentrate (Protexel; LFB Biomedicaments) was reported in 2 publications. One publication reported on both Ceprotin and Protexel. Details of protein C concentrate treatment regimens, including the dose, administration frequency, and route of administration, were reported in 11 publications. Dosing regimens varied across all 11 publications, possibly due to different protein C trough levels among patients or the administration of concomitant medications. Seven of the 11 publications reported on patients who initially received intravenous protein C concentrate and subsequently switched to subcutaneous administration. Treatment outcomes with protein C concentrate were generally favorable, including the prevention of coagulopathy and thrombosis and the healing of cutaneous lesions. Three adverse events in 1 publication were identified as being possibly related to Ceprotin administration. Although published data are limited, this review provides valuable insights into the treatment of patients with SCPCD in clinical practice, including protein C concentrate dosing regimens, administration routes, and associated clinical outcomes.</p></div>","PeriodicalId":20893,"journal":{"name":"Research and Practice in Thrombosis and Haemostasis","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2475037924002371/pdfft?md5=043e05369302daf2abcd9801e3978b6b&pid=1-s2.0-S2475037924002371-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142122797","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}