HaemophiliaPub Date : 2025-09-11DOI: 10.1111/hae.70124
Eve Justason, Anna M Ishchuk, Benjamin P Ott, M E Sophie Gibson, Paula D James
{"title":"Determining the Impact of Combination Oral Contraceptives on Von Willebrand Factor and Factor VIII in Healthy Patients and Patients With Von Willebrand Disease: A Scoping Review and Meta-Analysis.","authors":"Eve Justason, Anna M Ishchuk, Benjamin P Ott, M E Sophie Gibson, Paula D James","doi":"10.1111/hae.70124","DOIUrl":"https://doi.org/10.1111/hae.70124","url":null,"abstract":"<p><strong>Introduction: </strong>Von Willebrand disease (VWD) is a bleeding disorder characterized by a deficiency or dysfunction of Von Willebrand factor (VWF) and/or Factor VIII (FVIII), critical coagulation proteins. Individuals with VWD often use combination oral contraceptives (COCs) to manage heavy menstrual bleeding. However, the impact of COCs on VWF and FVIII levels and whether COC use affects VWD diagnosis is unclear.</p><p><strong>Aim: </strong>To review the literature and assess the impact of COCs on FVIII and VWF.</p><p><strong>Methods: </strong>This scoping review used the OVID platform in the MEDLINE, EMBASE and Cochrane databases. Keywords \"combination oral contraceptives,\" \"von Willebrand Factor,\" \"Factor VIII\" and \"von Willebrand Disease\" were searched. Primary studies exploring the impact of COCs on VWF and/or FVIII in patients of reproductive age were included. Article titles and abstracts were screened, followed by full-text reviews, data extraction and a meta-analysis.</p><p><strong>Results: </strong>Twenty-seven studies were included. In healthy patients, 11 studies reported no change in VWF levels, while three found changes in VWF levels. Nine studies reported no change in FVIII levels, while 10 studies observed an increase. In patients with VWD, two studies found no significant change in VWF or FVIII levels. Meta-analysis revealed there was no significant difference in VWF% (Estimate: 2.62 (95%CI -0.5905, 5.831); p value: 0.4033) or FVIII% (Estimate: 2.99 (95%CI -4.85, 10.82); p value: 0.4552) with COC use.</p><p><strong>Conclusion: </strong>The meta-analysis revealed no difference in VWD or FVIII levels between participants with and without COCs. The lack of observed differences suggests that COCs do not interfere with accurate VWD diagnosis.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145033191","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}
HaemophiliaPub Date : 2025-09-11DOI: 10.1111/hae.70101
Marie-Anaïs Roques, Natacha Rosso-Delsemme, Amandine Celli, Ngoc Anh Thu Nguyen, Martin Postzich, Sabine Castet, Yoann Huguenin, Annie Harroche, Anne Lienhart, Sandrine Meunier, Christine Biron-Andréani, Florence Rousseau, Roseline d'Oiron, Yohann Repessé, Clémence Tabélé, Any Beltran Anzola, Thomas Sannié, Nicolas Giraud, Pascal Auquier, Hervé Chambost, Noémie Resseguier
{"title":"Navigating the Transition From Adolescence to Adulthood Among Young People With Severe Haemophilia: The Qualitative Phase of the TRANSHEMO Project.","authors":"Marie-Anaïs Roques, Natacha Rosso-Delsemme, Amandine Celli, Ngoc Anh Thu Nguyen, Martin Postzich, Sabine Castet, Yoann Huguenin, Annie Harroche, Anne Lienhart, Sandrine Meunier, Christine Biron-Andréani, Florence Rousseau, Roseline d'Oiron, Yohann Repessé, Clémence Tabélé, Any Beltran Anzola, Thomas Sannié, Nicolas Giraud, Pascal Auquier, Hervé Chambost, Noémie Resseguier","doi":"10.1111/hae.70101","DOIUrl":"https://doi.org/10.1111/hae.70101","url":null,"abstract":"<p><strong>Introduction: </strong>Haemophilia causes spontaneous or prolonged bleeding due to a deficiency in clotting factor VIII (haemophilia A) or IX (haemophilia B). Although substitutive therapies and regular follow-up can prevent severe haemorrhagic events, adherence to treatment remains a challenge. Transitioning from adolescence to adulthood and from paediatric to adult care is particularly complex for young people with severe haemophilia (PwSH), as it involves gaining autonomy in health management.</p><p><strong>Objectives: </strong>This study aimed to explore factors influencing the success of the transition process in young PwSH, with a focus on adherence to healthcare.</p><p><strong>Methods: </strong>This qualitative study was part of the mixed-methods TRANSHEMO project. Participants were selected from the quantitative phase of the TRANSHEMO project based on two criteria: adolescents/young adults and adherent/nonadherent to healthcare. Interviews were conducted via video conferencing, transcribed, and thematically analysed to identify key themes affecting the transition process.</p><p><strong>Results: </strong>Twenty-two interviews were conducted. Four major themes emerged as critical to transition success: (1) Care factors [continuity of care, treatment rituals, and evolving therapies]; (2) Family and social factors [support from family, friends, peers, and overprotection]; (3) Personal factors [understanding haemophilia, risk management, optimism, and coping strategies]; and (4) Autonomy [secondary benefits, independence, proactivity in disease management, and accompaniment by caregivers].</p><p><strong>Conclusion: </strong>Based on the enlightened determinants, supportive strategies and patient education programs should focus on the development of autonomy, personal factors such as acquisition and application of health literacy in haemophilia care, and family factors such as support from family.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145032699","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}
HaemophiliaPub Date : 2025-09-11DOI: 10.1111/hae.70114
Mathieu Fiore, Andrea Artoni, Robert Klamroth, Mary Mathias, Roger Schutgens, Roseline d'Oiron
{"title":"European Management of Glanzmann's Thrombasthenia: A Survey of Current Clinical Practice.","authors":"Mathieu Fiore, Andrea Artoni, Robert Klamroth, Mary Mathias, Roger Schutgens, Roseline d'Oiron","doi":"10.1111/hae.70114","DOIUrl":"https://doi.org/10.1111/hae.70114","url":null,"abstract":"<p><strong>Introduction: </strong>Glanzmann's thrombasthenia is a rare inherited platelet disorder characterized by a lack of platelet aggregation. Patients tend to be diagnosed in early childhood with treatment strategies involving a multifaceted approach to prevent and manage bleeding episodes. Unfortunately, there is currently no European consensus regarding the management of GT.</p><p><strong>Aim: </strong>This initiative aimed to gain an understanding of current clinical management of GT across Europe, with the aim of aligning best practice and improving patient outcomes.</p><p><strong>Methods: </strong>The authors, on behalf of the EAHAD Glanzmann Working Group, administered an online survey of 57 questions to European haematologists currently involved in the management of patients with GT. The survey covered topics related to diagnosis, treatment access and selection, immunization, peri-operative management and use of second-line therapies.</p><p><strong>Results: </strong>Responses reflected physician consensus around some topics, including peri-operative treatment, use of recombinant factor VIIa, and concerns around antibody development. However, more varied responses were received on topics such as antibody screening (anti-αIIbβ3 antibodies screening conducted by ≤53% of respondents in all countries of interest except France), access to HLA-matched platelet concentrates (none or limited for 55% of respondents) and duration of platelet transfusions for major surgery (13%-31% for 1, 2, 3 and 4 or more days of transfusions).</p><p><strong>Conclusion: </strong>Establishing comprehensive guidelines to manage GT will enhance patient outcomes by ensuring patients receive high-quality and effective care as well as standardize care across different healthcare settings.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145033180","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}
HaemophiliaPub Date : 2025-09-02DOI: 10.1111/hae.70120
Martijn R Brands, Lieke Baas, Mariette H Driessens, Samantha C Gouw, Rieke van der Graaf, Karina Meijer
{"title":"People With Haemophilia as Data Coordinators: An Analysis of the Ethics and Feasibility of Self-Management With Personal Health Records.","authors":"Martijn R Brands, Lieke Baas, Mariette H Driessens, Samantha C Gouw, Rieke van der Graaf, Karina Meijer","doi":"10.1111/hae.70120","DOIUrl":"10.1111/hae.70120","url":null,"abstract":"<p><strong>Background: </strong>People with haemophilia perform various self-management tasks, supported by multiple health apps. Personal health records will enable individuals to access and add health information from different institutions in a single digital tool, providing an integrated overview of data. Later, individuals will also be able to share their data with health care providers and relatives. This creates a new role for users: Coordinator of data exchange.</p><p><strong>Objective: </strong>To analyze if and how personal health records contribute to self-management, with a particular emphasis on the role of coordinating data exchange.</p><p><strong>Methods: </strong>We applied various interpretations of self-management to the promises of personal health records to identify what goals it intends to achieve. We then assessed various skills and responsibilities that are required from users to work with personal health records. Last, we analyzed potential scenarios of the coordination of data exchange.</p><p><strong>Results: </strong>Personal health records promise to support both compliant self-management (i.e., managing care according to medical regimens) and concordant self-management (i.e., managing care according to personal values and goals). Which of these forms is promoted depends on the goal of data coordinating tasks. The chosen design of the data sharing feature may impact the usability and accessibility of personal health records for a wide group of users.</p><p><strong>Conclusion: </strong>What form of self-management is promoted by personal health records needs to be more clearly defined. A participatory design strategy can ensure that the design of coordinating data exchange matches individuals' and health care providers' needs.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144951665","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}
HaemophiliaPub Date : 2025-09-02DOI: 10.1111/hae.70121
Jan Astermark, Susanna Ranta, Linda Myrin-Westesson, Marianne Hoffmann, Sandrine Quere, Amparo Yovanna Castro Sanchez, Susan Robson, Tünde Czirok, Riitta Lassila, Pål André Holme
{"title":"Health-Related Quality of Life, Physical Activity and Joint Health in People With Severe Haemophilia A Receiving Emicizumab: Results From the Phase IV HemiNorth 2 Study.","authors":"Jan Astermark, Susanna Ranta, Linda Myrin-Westesson, Marianne Hoffmann, Sandrine Quere, Amparo Yovanna Castro Sanchez, Susan Robson, Tünde Czirok, Riitta Lassila, Pål André Holme","doi":"10.1111/hae.70121","DOIUrl":"10.1111/hae.70121","url":null,"abstract":"<p><strong>Introduction: </strong>Despite factor (F)VIII prophylaxis, a perceived increased risk of bleeding for some people with severe haemophilia A (PwSHA) exists, limiting physical activity (PA) and restricting quality of life (QoL).</p><p><strong>Aim: </strong>HemiNorth 2 (EudraCT# 2020-003256-32) is an interventional study evaluating the impact of switching from FVIII prophylaxis to emicizumab in PwSHA without FVIII inhibitors who have a need for improved prophylaxis in the Nordic countries.</p><p><strong>Methods: </strong>Following completion of the HemiNorth non-interventional study (NIS), eligible participants (aged ≥ 12-61 years) were enrolled in HemiNorth 2. The primary endpoint was health-related QoL via the Comprehensive Assessment Tool for Challenges in Hemophilia (CATCH). Secondary endpoints included PA (International Physical Activity Questionnaire-Short Form [IPAQ-SF]), treatment preference (Emicizumab Preference [EmiPref] survey), joint health, model-based annualised bleeding rates (ABRs) and adverse events.</p><p><strong>Results: </strong>Overall, 28 physically active male PwSHA were enrolled. Most baseline CATCH domains were ≤ 25 and remained consistent; mean treatment burden considerably improved from baseline for adults (-17.8) and adolescents (+16.7). IPAQ-SF scores were consistent throughout the study. Overall, 23 of 25 (92.0%) EmiPref respondents preferred emicizumab over FVIII prophylaxis. Model-based ABRs for treated bleeds decreased from 5.9 (95% confidence interval [CI]: 3.8-9.1) to 1.6 (95% CI: 0.9-3.0) from the NIS to HemiNorth 2, and participants with zero treated bleeds increased from 8 (28.6%) to 16 (57.1%). No new safety signals were reported.</p><p><strong>Conclusions: </strong>Emicizumab improved treatment burden and was preferred by most participants over FVIII prophylaxis. PA levels were consistently high, and bleeding rates improved with emicizumab versus prior FVIII prophylaxis.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144951648","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}
HaemophiliaPub Date : 2025-09-02DOI: 10.1111/hae.70123
Kelsey Uminski, Paula James, Nathan Connell, Roy Khalife
{"title":"Bridging the Evidence Gap in von Willebrand Disease: A Call to Action for Equitable, Evidence-Based Care","authors":"Kelsey Uminski, Paula James, Nathan Connell, Roy Khalife","doi":"10.1111/hae.70123","DOIUrl":"10.1111/hae.70123","url":null,"abstract":"<p>Von Willebrand disease (VWD) is the most common inherited bleeding disorder, affecting up to 1% of the population [<span>1</span>]. Over the past two decades, there has been significant progress in understanding its pathophysiology, classification and clinical management. However, research in VWD continues to lag behind that of haemophilia—a rarer condition that has benefited from earlier and more sustained investment. Today, haemophilia care is supported by robust national and global registries, individualized pharmacokinetic (PK) dosing strategies and a rich therapeutic pipeline, including gene and non-factor therapies [<span>2, 3</span>].</p><p>VWD spans a spectrum of biologically distinct subtypes, each with unique genetic drivers, bleeding patterns and variable severity. This diversity complicates every stage of research—patient selection, endpoint harmonization and treatment stratification—prompting industry partners and investigators to call for innovative, subtype-sensitive trial designs. Scientific complexity is only part of the challenge: limited access to specialized diagnostics, sparse clinical trial infrastructure and historical underinvestment in patient-relevant outcome measures have further slowed progress. Diagnostic variability—including frequent misclassification of subtypes and uneven availability of advanced assays—adds another layer of fragmentation to both clinical care and research. Table 1 summarizes interventional trials completed or underway in the past 20 years, highlighting areas of progress while revealing persistent gaps in scope and scale. For comparison, a search for interventional studies completed or underway in haemophilia over the same 20-year period yielded 442 studies.</p><p>Although VWD research has not progressed as rapidly as haemophilia, recent efforts signal meaningful momentum. A World Federation of Hemophilia (WFH) Congress Plenary by Peter Lenting—later published in Blood [<span>4</span>]—highlighted the imbalance between haemophilia and VWD, helping galvanize the community around a renewed research agenda. Since then, initiatives such as the ASH/ISTH/NHF/WFH 2021 guidelines [<span>5, 6</span>] and the development of CoreVWD [<span>7</span>] have established a foundation for standardized diagnosis, management and outcome measurements. These initiatives are evidence of a maturing research foundation with potential for further growth.</p><p>Haemophilia trials routinely enrol hundreds of participants across continents [<span>8, 9</span>]. Most VWD studies, by comparison, remain small and geographically limited. Even the landmark prophylaxis trial by Peyvandi et al. [<span>10</span>] enrolled only 19 participants, with 12 participants completing the study. Recent studies, including WIL-31 [<span>11</span>] and VWDMin [<span>12</span>], signal a growing interest in VWD research, with their scale and scope offering a springboard for broader, coordinated programmes such as seen in haemophilia. Moreover, ","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 5","pages":"815-822"},"PeriodicalIF":3.0,"publicationDate":"2025-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70123","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144951680","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}
HaemophiliaPub Date : 2025-09-02DOI: 10.1111/hae.70115
François Grand, Florence Blanc-Jouvan, Guillaume Mourey, Anne Ryman, Valérie Eschwege, Marie Brionne-François, Dominique Lasne, Sophie Voisin, Véronique Le Cam-Duchez, Isabelle Diaz-Cau, Claire Flaujac, Pauline Noyel, Cecile Lavenu-Bombled, Emmanuel Demaistre, Emmanuelle Jeanpierre, Magali Donnard, Claire Pouplard, Nathalie Hezard, Laurent Sattler, Christophe Nougier
{"title":"Drug-Specific Calibration: A Solution to Reagent Variability in Extended Half-Life Factor IX Activity Measurements.","authors":"François Grand, Florence Blanc-Jouvan, Guillaume Mourey, Anne Ryman, Valérie Eschwege, Marie Brionne-François, Dominique Lasne, Sophie Voisin, Véronique Le Cam-Duchez, Isabelle Diaz-Cau, Claire Flaujac, Pauline Noyel, Cecile Lavenu-Bombled, Emmanuel Demaistre, Emmanuelle Jeanpierre, Magali Donnard, Claire Pouplard, Nathalie Hezard, Laurent Sattler, Christophe Nougier","doi":"10.1111/hae.70115","DOIUrl":"10.1111/hae.70115","url":null,"abstract":"<p><strong>Introduction: </strong>Discrepancies in factor IX activity (FIX:C) measurements between one-stage clotting assays (OSAs) have been observed following infusion with recombinant factor IX extended half-life concentrates (EHL-rFIX) in the treatment of haemophilia B. These variations, primarily due to differences in activated partial thromboplastin time (APTT) reagents, complicate clinical decision-making.</p><p><strong>Objectives: </strong>The aim of this study was to evaluate whether drug-specific calibrations for albumin-fused recombinant FIX (rFIX-FP) and Fc-fused recombinant FIX (rFIX-Fc) could reduce inter-reagent discrepancies.</p><p><strong>Methods: </strong>In a multicentre field study involving 20 laboratories, plasma samples spiked with rFIX-FP, rFIX-Fc or standard rFIX were tested using different APTT reagents. FIX:C was measured with usual and drug-specific calibration. Data were analysed for compliance (target range: 80%-120% of expected values), interlaboratory variability and intralaboratory reproducibility.</p><p><strong>Results: </strong>Usual calibration resulted in significant discrepancies among reagents, with compliance rates varying widely. Drug-specific calibration significantly improved compliance for all reagents tested, except for one concentration with rFIX-Fc. Interlaboratory variability decreased markedly, with coefficients of variation dropping from 19.4%-36.0% (usual calibration) to 6.0%-12.4% (specific calibration). Intralaboratory reproducibility was consistent whatever the type of calibration.</p><p><strong>Conclusion: </strong>Drug-specific calibration for EHL-rFIX reduces reagent-related variability in OSA FIX assays, ensuring reliable and standardised results. This approach facilitates monitoring with a single reagent system, improving laboratory efficiency. Wider availability of validated calibrators remains crucial for broader adoption and standardisation.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144951613","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}
HaemophiliaPub Date : 2025-08-28DOI: 10.1111/hae.70117
Federico Germini, Carlo Cossa, Elisabetta Trinari, Emma Iserman, Quazi Ibrahim, Drashti Pete, Arun Keepanasseril, David Page, Mark W Skinner, Alfonso Iorio
{"title":"Feasibility of Administering the Patient Reported Outcomes, Burdens and Experiences (PROBE) Questionnaire Through the Canadian Bleeding Disorders Registry (CBDR) and Comparison of Data From the Two Sources.","authors":"Federico Germini, Carlo Cossa, Elisabetta Trinari, Emma Iserman, Quazi Ibrahim, Drashti Pete, Arun Keepanasseril, David Page, Mark W Skinner, Alfonso Iorio","doi":"10.1111/hae.70117","DOIUrl":"https://doi.org/10.1111/hae.70117","url":null,"abstract":"<p><strong>Introduction: </strong>The Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire can be used to measure quality of life in persons with haemophilia (PWH) and is integrated in the Canadian Bleeding Disorders Registry (CBDR). This offers the opportunity to compare the same data inputted by patients in PROBE and their treating team in CBDR.</p><p><strong>Aim: </strong>Our objectives were to assess the feasibility of collecting PROBE data through CBDR and to compare the data collected from these two sources.</p><p><strong>Methods: </strong>We conducted a prospective observational study among PWH using MyCBDR. Participants were invited to digitally complete the PROBE questionnaire at baseline and to repeat it at 6 and 12 months. Additional data were passively collected through CBDR. Data from PROBE and CBDR were compared using Kappa agreement, intraclass correlation (ICC) and Pearson correlation.</p><p><strong>Results: </strong>A total of 142 PWH participated. Recruitment ratios were 21.1% and 12.0% for the two phases. Retention rates were 40.8% at 6 months and 32.4% at 12 months. Three hundred thirteen subjects were involved in the comparison between PROBE and CBDR data. The agreement was good to very good (κ > 0.75) or the correlation very strong, with the exception of the history of inhibitor (κ = 0.57), recent bleeds (κ = 0.48) and current treatment regimen (κ = 0.57).</p><p><strong>Conclusion: </strong>The integration of PROBE with CBDR is feasible and PROBE is a reliable tool for routine PRO data collection. Its use in clinical practice may improve data quality and personalized and patient-centred care.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144951657","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}