Haemophilia最新文献

{"title":"Congenital Hemophilia-A With High-Titer Inhibitor and Essential Thrombocythemia: A Rare Coexistence.","authors":"Deborah Kasmi, Silvia Sorella, Erminia Baldacci, Mario Biglietto, Bianca Maria Mandelli, Antonio Chistolini, Massimo Breccia, Christian Carulli, Cristina Santoro","doi":"10.1111/hae.70403","DOIUrl":"https://doi.org/10.1111/hae.70403","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2026-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148929244","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Multicentre Retrospective and Prospective Analysis of Data on Emicizumab and Factor VIII Prophylaxis in Children With Severe Haemophilia A: Egypt-Compare Study","authors":"Tamer Hassan, Mohsen Elalfy, Mervat A. M. Youssef, Omar Elalfy, Elzahraa Elmasry, Gehan Lotfy Khalifa, Ahmad Darwish, Eslam E. Elhawary","doi":"10.1111/hae.70310","DOIUrl":"10.1111/hae.70310","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"32 5","pages":"1288-1292"},"PeriodicalIF":3.0,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147928588","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"UK Experience of Children With Inhibitors in Severe Haemophilia B: A Report by the UKHCDO Paediatric Working Party","authors":"Mary Mathias, Jayanthi Alamelu, Neha Bhatnagar, Tina Biss, John Grainger, Simone Green, Anne Kelly M, Jayashree Motwani, Jeanette Payne, Simone Stokley","doi":"10.1111/hae.70315","DOIUrl":"10.1111/hae.70315","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"32 5","pages":"1293-1296"},"PeriodicalIF":3.0,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147941167","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Nonneutralizing Antibodies in Haemophilia A Patients: To Be Ignored or Not to Be!","authors":"Shrimati Shetty,&nbsp;Anam Dhawlarker,&nbsp;Shruti Kharat,&nbsp;Kranti Patil,&nbsp;Gurpreet Kaur Saini,&nbsp;Fiza jivani,&nbsp;Juili Bharankar,&nbsp;Shrinath Kshirsagar,&nbsp;Savita Rangarajan","doi":"10.1111/hae.70322","DOIUrl":"10.1111/hae.70322","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>The existence of nonneutralizing antibodies (NNAs) to factor VIII (FVIII) unrecognized by the clot-based Bethesda assay has been widely accepted. They are reported both in haemophilia A (HA) patients without inhibitors and healthy controls, but are poorly studied.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>The article discusses the contradictory reports on the nature and clinical significance of NNAs.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Discussion and Conclusion</h3>\u0000 \u0000 <p>Initially defined as antibodies to non-functional domains of FVIII, recent studies have provided evidence for multi-domain specificities. Subclass analysis and affinity studies have shown evidence for distinct immune regulatory pathways which lead to antibody responses against FVIII in different groups. Their role in pharmacokinetics of FVIII is the most frequently studied, while few studies also indicate their possible role as biomarkers for future development of neutralizing antibodies (inhibitors). The impact of NNAs on the clinical severity of HA is largely elusive; however, they can cause difficulties in the interpretation of immunological tests. There is a need for longitudinal comprehensive studies in HA patients on the natural history of NNAs and also for clear guidelines for accurate diagnosis. Present review summarizes the data available on the prevalence of NNAs, their clinical impact and discusses directions for future research.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"32 5","pages":"1160-1169"},"PeriodicalIF":3.0,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148101390","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Safety and Efficacy of Damoctocog Alfa Pegol Prophylaxis in Patients With Severe Haemophilia A: Results of an Interventional, Post-Marketing Study","authors":"Pål André Holme,&nbsp;Lone Hvitfeldt Poulsen,&nbsp;Claudia Tueckmantel,&nbsp;Monika Maas Enriquez,&nbsp;María Teresa Alvarez Román,&nbsp;Raimondo De Cristofaro","doi":"10.1111/hae.70099","DOIUrl":"10.1111/hae.70099","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3>  </h3>\u0000 \u0000 <p>This is a plain language summary of the results of a trial of damoctocog alfa pegol (BAY 94–9027, Jivi).</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"32 5","pages":"1206-1207"},"PeriodicalIF":3.0,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70099","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144951609","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Perceived Barriers to Healthcare Access Among Paediatric Patients With Haemophilia in Cambodia: A Cross-Sectional Survey","authors":"Chean Sophâl,&nbsp;Heng Sivmeng,&nbsp;Lean Kimsreng,&nbsp;Ith Ponndara","doi":"10.1111/hae.70343","DOIUrl":"10.1111/hae.70343","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Haemophilia is a rare inherited bleeding disorder, and in low- and middle-income countries, care is often limited by delayed diagnosis and restricted access to specialised services. In Cambodia, care is centralised in only two treatment centres, which may further limit timely diagnosis and treatment.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>To assess perceived barriers to healthcare access among paediatric patients with haemophilia in Cambodia.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A cross-sectional survey was conducted in Cambodia from July to December 2025 among 100 reachable patients aged ≤18 years with Haemophilia A or B from a National Hemophilia Registry of 250 patients. Data were collected through telephone or face-to-face interviews using a structured questionnaire administered primarily to parents or caregivers.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Among 100 participants, 88% reported at least one diagnostic barrier, mainly limited caregiver knowledge, difficulty accessing experienced providers, and delayed referral. Barriers to daily home-based bleeding management were reported by 83%, largely related to inadequate caregiver education and limited healthcare support. Barriers to accessing haemophilia treatment centres affected 73%, mainly geographic distance, financial constraints and transportation difficulties. Delays in receiving treatment were reported by 69% and were significantly associated with clotting factor shortages, financial constraints and delayed referral. Clotting factor shortage was not significantly associated with home-based management barriers but contributed to treatment delays. Most participants reported satisfaction with care (82%) and perceived care as equitable (85%).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Paediatric haemophilia care in Cambodia is limited by geographic, financial, educational and health-system barriers. Improving education, referral pathways, home-care support, clotting factor supply and financial protection may strengthen care access.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"32 5","pages":"1200-1205"},"PeriodicalIF":3.0,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70343","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148251296","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Monitoring of Coagulation Potential of Efanesoctocog Alfa in Haemophilia A: Insights From Conventional and Global Hemostasis Assays","authors":"Masahiro Takeyama,&nbsp;Yasuharu Nishida,&nbsp;Shoko Furukawa,&nbsp;Kenichi Ogiwara,&nbsp;Keiji Nogami","doi":"10.1111/hae.70341","DOIUrl":"10.1111/hae.70341","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Efanesoctocog alfa (Efa) is a high-sustained factor (F)VIII engineered to overcome von Willebrand factor (VWF)-dependent clearance, enabling once-weekly dosing. However, FVIII activity (FVIII:C) differs between the one-stage assay (OSA) and chromogenic substrate assay (CSA), and its relationship with global assays remains unclear.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>To assess the procoagulant potential in people with haemophilia A (PwHA) treated with Efa using conventional and global assays.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Plasma samples were collected from five PwHA treated with Efa. FVIII:C was assayed using OSA (RevohemAPTT, PathromtinSL at two institutions) and CSA. Clot waveform analysis (CWA) using RevohemAPTT SLA and ThromboCheck APTT SLA, thrombin generation assay (TGA), and rotational thromboelastometry were performed. Apparent equivalent FVIII:C (app-eq-FVIII:C) was derived from reference curves for cross-assay comparison.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>OSA-FVIII:C showed reagent- and site-dependent variability. Pathromtin-based FVIII:C aligned with Revohem-based values at one site but was 1.6–1.8-fold higher at the other. CSA values were higher; CSA/2.5 variably approximated OSA in this dataset. CWA increased consistently after administration, with Revohem-based assays higher than ThromboCheck-based assays, indicating reagent-dependent variation within CWA. TGA showed increased thrombin generation with interpatient variability, ranging from minimal to relatively marked increases; conversion to app-eq-FVIII:C was strongly parameter-dependent (time-to-Peak vs Peak-Th). Rotational thromboelastometry revealed heterogeneous responses, and app-eq-FVIII:C was frequently lower than OSA and CSA/2.5-derived values.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Efa demonstrated assay-detected post-infusion increases; however, FVIII:C measurement varied across platforms and parameters. In this small exploratory cohort, these findings support assay-specific validation and cautious interpretation of inter-platform differences, rather than definitive conclusions regarding optimal monitoring strategy or direct clinical decision-making.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"32 5","pages":"1272-1281"},"PeriodicalIF":3.0,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148251246","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical Factors for Predicting Synovial Hypertrophy in Early Haemophilic Arthropathy on Prophylaxis: A Cross-Sectional Study of Patient- and Joint-Level Analyses Using Musculoskeletal Ultrasound","authors":"Takeo Abe,&nbsp;Yusuke Inagaki,&nbsp;Chie Ogita,&nbsp;Yuko Minagawa,&nbsp;Taduko Tokugawa,&nbsp;Akihiro Sawada,&nbsp;Satoshi Higasa,&nbsp;Yoshitoshi Ohtsuka,&nbsp;Teppei Hashimoto,&nbsp;Naoto Azuma","doi":"10.1111/hae.70345","DOIUrl":"10.1111/hae.70345","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Early identification of synovial hypertrophy is critical for preventing irreversible changes in haemophilic arthropathy (HA). Although prophylaxis has improved joint outcomes, it remains unclear which patients and joints in early HA should be prioritised for musculoskeletal ultrasound (MSKUS) screening.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>To identify the predictors of synovial hypertrophy at the patient and joint levels in early HA.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We enrolled patients with haemophilia aged 6–49 years with mild joint damage (Arnold–Hilgartner grades I–III). Elbows, knees and ankles were examined using MSKUS. Patient-level factors included age, body mass index, haemophilia characteristics, adherence and activity. Joint-level factors included symptoms, recent bleeding, physical findings and radiographic scores. Logistic regression was applied to patient-level predictors and mixed-effects models to joint-level predictors.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>In total, 203 joints from 40 patients were evaluated; synovial hypertrophy was identified in 82 joints (34 patients), most frequently in the knees (54.5%), followed by the elbows (38.9%) and ankles (22.2%). Adult age was the only significant patient-level predictor of synovial hypertrophy (odds ratio, 5.2; <i>p </i>= 0.001). At the joint level, no examined factors were significantly associated with synovial hypertrophy. Among asymptomatic joints, 37.9% demonstrated synovial hypertrophy (23.7% in paediatric patients vs. 56.9% in adults), with elbow involvement showing no significant age-related differences (34.4% vs. 40.7%, <i>p </i>= 0.79).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Patient- and joint-specific findings alone are insufficient to predict synovial hypertrophy. MSKUS may be used routinely to enhance early detection in patients with HA on prophylaxis. Elbow synovial hypertrophy warrants attention from paediatric patients as one of the earliest stages of the disease.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"32 5","pages":"1255-1263"},"PeriodicalIF":3.0,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148277179","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Accuracy of Factor VIII Assays for Measuring Damoctocog Alfa Pegol: A Comparative Analysis","authors":"Judith C. A. Cluitmans,&nbsp;Piet Meijer,&nbsp;Sanna R. Rijpma,&nbsp;An K. Stroobants","doi":"10.1111/hae.70350","DOIUrl":"10.1111/hae.70350","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Accurate monitoring of treatment with modified factor VIII products remains challenging, due to known variability in assay performance across different laboratory methods and reagents. For the extended half-life product Damoctocog alfa pegol discrepancies have been reported.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aims</h3>\u0000 \u0000 <p>This multicentre field study was designed to evaluate the analytical accuracy of Damoctocog alfa pegol using various methods for one-stage clotting and chromogenic assays.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The study was conducted under conditions closely mimicking routine laboratory settings. Five plasma samples were spiked with varying concentrations of Damoctocog alfa pegol (0.05 to 1.3 IU/ml) and distributed to 118 participating laboratories. FVIII activity of the samples was analysed using 12 one-stage and 7 chromogenic methods. Accuracy results were evaluated per specific reagent with a maximal acceptable mean bias of 20% from the target. Interlaboratory variation was established for reagents with ≥3 participants.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The reliability of factor VIII measurements differ between the tested reagents. Usage of SynthasIL and Pathromtin (one stage) and Biophen (chromogenic) is in most cases reliable, although inter-lab variation contributes to inaccuracy. The reagents STA PTT A and Triniclot (one stage), as well as Siemens (chromogenic) (strongly) underestimate FVIII activity and are therefore unsuitable for accurate measurement of Damoctocog alfa pegol.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>We have evaluated accuracy results for 19 Factor VIII assays in order to provide evidence-based guidance for clinical laboratories in selecting appropriate assay methodologies for reliable quantification of the activity of Damoctocog alfa pegol. Assay performance should be locally validated as large interlaboratory variation is observed.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"32 5","pages":"1282-1287"},"PeriodicalIF":3.0,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70350","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148390732","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Optimizing Perioperative Management of Haemophilia B With rFIX-FP: Pharmacokinetic Validation of the Hemoptidose Tool","authors":"Xavier Delavenne,&nbsp;Célia Vollard,&nbsp;Nicolas Drillaud,&nbsp;Sabine Castet,&nbsp;Nicolas Luyck,&nbsp;Emilie Hénin,&nbsp;Julien Denis-Le Seve,&nbsp;Fabienne Volot,&nbsp;Brigitte Tardy,&nbsp;Stéphanie Désage,&nbsp;Benoît Guillet","doi":"10.1111/hae.70326","DOIUrl":"10.1111/hae.70326","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Surgical management of haemophilia B requires precise factor IX replacement to ensure adequate haemostasis while optimizing factor consumption. Extended half-life rFIX-FP simplifies perioperative management but exhibits substantial pharmacokinetic variability. PK-guided dosing is recommended by WFH guidelines, yet validated tools for perioperative rFIX-FP individualization are lacking.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>To validate a population PK model under perioperative conditions and evaluate Hemoptidose, a model-informed precision dosing (MIPD) platform, for individualizing rFIX-FP therapy during surgery.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We retrospectively analyzed 24 surgeries in haemophilia B patients treated with rFIX-FP. Predictive performance was assessed at population level and after Bayesian individualization with varying numbers of FIX samples. Actual perioperative consumption was retrospectively compared with Hemoptidose-recommended doses.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The population PK model demonstrated robust perioperative performance. Population predictions yielded a mean absolute error (MAE) of 20.6%; after Bayesian updating, accuracy improved with MAE of 10.0%. Prediction error decreased from 20.6% without monitoring to 14% with three samples. Retrospective analysis indicated a potential 49% reduction in factor consumption with model-guided dosing compared to empirical regimens (p &lt; 0.001).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>The population PK model retains valid predictive performance in surgical settings. Two to three FIX measurements enable reliable individualization. The substantial discrepancy between empirical and model-guided dosing highlights major optimization potential. Hemoptidose provides accessible MIPD implementation for perioperative rFIX-FP management.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"32 5","pages":"1179-1187"},"PeriodicalIF":3.0,"publicationDate":"2026-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70326","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148143146","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}