Haemophilia最新文献

{"title":"Outcomes of Dental Treatment Procedures in People With Haemophilia Treated Under a Comprehensive Care Model: An Analysis of Cases From a Tertiary Care Centre.","authors":"Mathangi Kumar, Sulochana Badagabettu, Keerthilatha M Pai, Annamma Kurien, Archana Mv, Shreemathi S Mayya, Sreejith Govindan","doi":"10.1111/hae.70055","DOIUrl":"https://doi.org/10.1111/hae.70055","url":null,"abstract":"<p><strong>Background: </strong>Haemophilia is a chronic, lifelong bleeding disorder that requires interdisciplinary care to manage and mitigate the disease burden throughout a patient's life. There is no universally accepted protocol for managing haemophilia patients during dental care.</p><p><strong>Objective: </strong>To evaluate the state of practice employed for people with haemophilia undergoing various dental treatment procedures under a comprehensive care model in a tertiary care setting.</p><p><strong>Methods: </strong>Patient records from the archives of Haemophilia Society of Kasturba Hospital, Manipal were retrieved, and the demographic data, disease status and dental treatment records of each patient were systematically reviewed. The various dental procedures and the outcomes were correlated with the severity of haemophilia and the quantity of prophylactic factors concentrate utilised for the dental treatment procedures.</p><p><strong>Results: </strong>A total of 73 patients attended the comprehensive Haemophilia clinic during the study period. Sixty nine of them underwent 84 dental treatment procedures. The various dental procedures that were performed on these patients were oral prophylaxis (scaling), extraction of grossly decayed teeth, restoration (fillings) of carious teeth, endodontic treatment and other minor oral surgical procedures. The amount of clotting factor concentrates given prior to dental treatment ranged from 250 to 1500 IU. None of the patients had excessive haemorrhage following dental procedures. Statistical analyses were performed with SPSS software, and statistical significance was set at p < 0.05. Chi-square and Fisher's exact test were used for the analysis of quantitative variables.</p><p><strong>Conclusion: </strong>Standardisation of prophylactic clotting factors for various dental treatment procedures is essential to prevent haemorrhagic complications. Trial Registration Clinical Trials Registry - India (CTRI) number: REF/2024/01/077096.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144063619","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":"European Principles of Nursing Care for Persons With Inherited Bleeding Disorders.","authors":"Nanda Uitslager, Mary Kavanagh, Maj Birkedal, Linda Myrin-Westesson","doi":"10.1111/hae.70054","DOIUrl":"https://doi.org/10.1111/hae.70054","url":null,"abstract":"<p><strong>Introduction: </strong>A nurse is one of the core members of the multidisciplinary haemophilia team. However, what people with inherited bleeding disorders (PwBD) can expect from them has not yet been formally defined.</p><p><strong>Aim: </strong>This project, initiated by the EAHAD Nurses Committee, aimed to establish Principles of Nursing Care (PoNC) for PwBD to promote high-quality, consistent nursing care throughout the European Haemophilia Comprehensive Care Centres (EHCCC) and European Haemophilia Treatment Centres (EHTC).</p><p><strong>Methods/results: </strong>The PoNC were developed through a multi-phase process, which included 10 roundtable meetings with input from expert nurses across Europe. The emerging PoNC were critically reviewed by members of the European Haemophilia Consortium (EHC), EAHAD Executive Committee, EAHAD Physiotherapists Committee and EAHAD Psychosocial Professionals Committee. Based on their feedback, the authors made further refinements, resulting in the final version of the PoNC.</p><p><strong>Conclusion: </strong>In summary, PwBD should have access to nurses who provide coordinated person-centred care across all aspects of their lives. This includes education, treatment management, life-stage support, surgical coordination, genetic counselling and special attention to the needs of women and girls. The nurses must also stay informed about research developments to ensure up-to-date care. The PoNC strengthen nursing care by providing a framework to address patient needs and may guide the development of care strategies, empowering patients and HCPs to advocate for high-quality care. These principles promote standardised nursing care in EHCCC and EHTC, to be applied alongside local policies and EAHAD's accreditation standards.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143988520","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":"Recombinant Activated Factor VIIa Combined With Tranexamic Acid Control Haemostasis in Patients With Severe Factor XI Deficiency Undergoing Urological Procedures.","authors":"Neta Sternbach, Yaron Ehrlich, Anfisa Stenevsky, Daniel Saidian, Darawsha Abd Elhalim, Jack Baniel, Pia Raanani, Galia Spectre","doi":"10.1111/hae.70052","DOIUrl":"https://doi.org/10.1111/hae.70052","url":null,"abstract":"<p><strong>Introduction: </strong>Patients with severe Factor XI deficiency face unique challenges during urological procedures due to an elevated risk of bleeding, balanced against the use of tranexamic acid (TXA), which may form blood clots. Given the limitations associated with plasma-based treatment, there have been reports of successful and safe off-label use of recombinant activated Factor VII (rFVIIa) in combination with TXA in general surgery.</p><p><strong>Aim: </strong>Report our experience with this approach in urological surgeries involving tissues with heightened fibrinolytic activity.</p><p><strong>Methods: </strong>A retrospective case series analysed seven patients with severe FXI deficiency who underwent seven urological procedures. Five patients were classified as bleeders, and two developed antibodies to FXI following prior exposure to plasma. The use of off-label rFVIIa was approved by our institutional authorities. Patients received TXA 1 g four times daily for 7-10 days, and a single dose of 10-15 mcg/kg of rFVIIa at the end of surgery (based on previous thrombin generation studies). Patients were connected to a continuous irrigation system postoperatively and monitored for urinary bleeding.</p><p><strong>Results: </strong>All procedures were performed without significant bleeding complications, except for one patient who remained on aspirin therapy and experienced manageable bleeding. None of the patients required discontinuation of TXA due to blood clots, nor did they require blood transfusions or additional doses of rFVIIa. No thrombotic complications were observed.</p><p><strong>Conclusion: </strong>Combination of low-dose rFVIIa with TXA is a feasible and attractive option for patients undergoing urologic procedures. Further implications of this protocol should be implemented in clinical practice.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144011201","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":"Physical Activity Awareness and Understanding of Treatment Protection Among People With Haemophilia and Their Caregivers in Central Europe.","authors":"Angelika Batorova, Atanas Banchev, Ana Boban, Brigitte Brand, Melen Brinza, Barbara Faganel Kotnik, Csongor Kiss, Gediminas Puras, Jan Rajnoch, Ester Zapotocka","doi":"10.1111/hae.70038","DOIUrl":"https://doi.org/10.1111/hae.70038","url":null,"abstract":"<p><strong>Background: </strong>Physical activity is now considered generally beneficial for persons with haemophilia (PWH). However, the specific type and extent of activity and its impact on quality of life (QoL) and bleed protection during exercise is under-researched. This knowledge gap should significantly impact PWH's ability to manage their condition optimally.</p><p><strong>Objective: </strong>To explore levels of physical activity and understand bleed protection during exercise among PWH as part of the Liberate Life project.</p><p><strong>Methods: </strong>A web-based survey was conducted across eight European countries between August and September 2023. The online survey was developed by haemophilia treatment experts and patient association representatives according to the Checklist for Reporting Results of Internet E-Surveys (CHERRIES) method. The 24-question survey, distributed to PWH or their parents/caregivers, covered topics such as demographics, the impact of haemophilia on daily life, bleed protection understanding and involvement in physical activities. An activity index was used to categorize PWH as 'more' or 'less' active.</p><p><strong>Results: </strong>Of 374 respondents analysed, PWH engagement in physical activities, mainly low-impact activities like walking, swimming or cycling, was high (90.9%). Most respondents (74.9%) were 'less active' according to the activity index. There was a significant knowledge gap in respondents' understanding of bleed protection provided by factor and non-factor therapies. Joint health (68.7%), disease severity (69.3%) and capability for physical activity (62.3%) were indicated as key variables influencing PWH QoL.</p><p><strong>Conclusion: </strong>These findings underscore the need for educational programs to improve PWH's understanding of protective factor levels and the role of non-factor therapies in managing haemophilia.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144015007","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":"Invited Medical Presentation Abstracts","authors":"","doi":"10.1111/hae.70031","DOIUrl":"https://doi.org/10.1111/hae.70031","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 S2","pages":"3-20"},"PeriodicalIF":3.0,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70031","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143938910","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":"Book of Abstracts","authors":"","doi":"10.1111/hae.70032","DOIUrl":"https://doi.org/10.1111/hae.70032","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 S2","pages":"21-109"},"PeriodicalIF":3.0,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70032","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143938911","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":"Study on Psychiatric Aspects, Psychosocial and Cognitive Functioning in Adolescents With Haemophilia.","authors":"Hemant Choudhary, Rajesh Sagar, Tulika Seth, Rachna Bhargava, Bichitra Nanda Patra","doi":"10.1111/hae.70046","DOIUrl":"https://doi.org/10.1111/hae.70046","url":null,"abstract":"<p><strong>Introduction: </strong>Haemophilia, an inherited bleeding disorder, impacts physical and mental health. With improving survival, the focus is on mental health issues.</p><p><strong>Aim: </strong>We examined and compared mental health issues in adolescents with haemophilia (AwH) in India with those of healthy controls. Additionally, we examined the relationship between quality of life and these mental health issues.</p><p><strong>Methods: </strong>Standardized assessments were applied (between 2021 and 2022) on male cases (n = 31) and controls (n = 31) to measure psychiatric morbidity, psychosocial, and neuro-cognitive functioning. Correlational analysis of quality of life with various mental health issues was also performed.</p><p><strong>Results: </strong>AwH had more emotional, social and behavioural problems, poor quality of life, more cognitive dysfunction, with no statistically significant difference in self-esteem as compared to healthy controls. There was a significant negative correlation between the quality of life and psychiatric morbidities, emotional and behavioural problems and cognitive dysfunction.</p><p><strong>Conclusion: </strong>These findings corroborate evidence from other countries and emphasize the need for assessment of mental health issues and integration of mental health professionals in clinical services for AwH in India.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144008718","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":"Global Comparative Antithrombin Field Study: Impact of Laboratory Assay Variability on the Assessment of Antithrombin Activity Measurement at Fitusiran Clinical Decision-Making Points","authors":"Ekta Seth Chhabra,&nbsp;Ali Sadeghi-Khomami,&nbsp;Mingjie Liu,&nbsp;Guy Young,&nbsp;Steven W. Pipe,&nbsp;Margareth C. Ozelo,&nbsp;Cecile Le Camus,&nbsp;Myew-Ling Toh,&nbsp;Marek Demissie,&nbsp;Flora Peyvandi","doi":"10.1111/hae.70045","DOIUrl":"10.1111/hae.70045","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Fitusiran is a subcutaneous, investigational small interfering RNA therapeutic that lowers antithrombin (AT) to increase thrombin generation and rebalance haemostasis in people with haemophilia A or B with or without inhibitors.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>To evaluate and compare the performance of commercially available in vitro diagnostic (IVD) AT activity assays.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Field study sample kits with plasma AT activity levels (100, 36, 14 and 9 IU/dL or % of normal) were created and distributed to global haemostasis laboratories. Values were assigned based on Siemens INNOVANCE AT activity assay using BCS-XP analyser. Reliability (relative accuracy estimate), intra- and inter-laboratory variability of IVDs in measuring AT activity in plasma samples using various commercially available AT assays was assessed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>At normal AT activity level (i.e., 100%), all AT assays reliably measured AT activity with acceptable recovery. Accurate results were observed for all samples across sites using Siemens INNOVANCE AT assay. Increased variability was observed for all other assays at low AT levels. Siemens Berichrom and Stago STA-Stachrom assays accurately measured 100% and 36% AT activity; however, lab-to-lab variability was observed for ≤15% AT activity (CV &gt;20%). All laboratories for the Stago STA-Stachrom assay failed to measure 9% AT activity. The HemosIL assay significantly underestimated AT activity levels ≤36%. There were no reported values for the 14% and 9% AT samples.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Siemens INNOVANCE AT assay can reliably measure AT activity at clinical decision points of 15–35% of normal and is most suitable for clinical management of patients taking fitusiran.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 3","pages":"566-574"},"PeriodicalIF":3.0,"publicationDate":"2025-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70045","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143990255","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":"Development of an Online Educational Case-Based Tool to Supplement Haematology Trainee Understanding of Haemostatic Disorders and Their Management.","authors":"Kelsey Uminski, Mike Paget, Natalia Rydz, Dawn Goodyear","doi":"10.1111/hae.70044","DOIUrl":"https://doi.org/10.1111/hae.70044","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143962262","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":"Haemophilia Prophylaxis in the Age of Innovation: Exploring Opportunities for Personalized Treatment.","authors":"Yesim Dargaud, Fabienne Volot, Stephanie Desage, Claire Pouplard, Valerie Chamouard, Anne Lienhart","doi":"10.1111/hae.70015","DOIUrl":"https://doi.org/10.1111/hae.70015","url":null,"abstract":"<p><strong>Introduction: </strong>Recent advancements in haemophilia treatment have introduced a range of innovative therapies, including activated FVIII mimetics, ultra-extended half-life recombinant FVIII, rebalancing agents and gene therapy. These developments have transformed treatment options and expanded prophylaxis strategies.</p><p><strong>Aim: </strong>This article reviews key criteria and personalization strategies for the optimization of prophylaxis, tailored to the unique needs of each haemophilia patient on an individual basis.</p><p><strong>Discussion and conclusion: </strong>Although these new therapies offer significant promise in controlling bleeding and enhancing quality of life, challenges such as variable efficacy, potential long-term risks and high costs remain. Additionally, the absence of validated surrogate markers for non-factor therapies limits their optimal use in clinical settings. The primary goal of haemophilia care is to provide optimal personalized treatment that enables patients to lead unrestricted lives. However, the introduction of these novel treatments necessitates a shift in treatment paradigms and the development of improved evaluation tools for better personalization. Choosing the most appropriate treatment requires careful clinical follow-up, taking into account patient preferences, disease burden, and individual and environmental factors that affect patients' lives. Ongoing research and real-world studies are essential to assess long-term efficacy and safety. In addition, improving educational resources-such as digital platforms, virtual reality tools and personalized educational materials tailored to patients' life goals-and effective patient engagement strategies will be critical to achieving successful treatment personalization and adherence.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144008063","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}