IF 3 2区医学

Haemophilia Pub Date : 2025-10-25 DOI: 10.1111/hae.70155

Başak Koç, Bülent Zülfikar, Ufuk Arzu, İsmail Tarık Atasoy, Mert Ege, Gökhan Polat

{"title":"Predominance of Radiographical Femoral Component Loosening Signs in Total Knee Arthroplasty for Hemophilic Arthropathy.","authors":"Başak Koç, Bülent Zülfikar, Ufuk Arzu, İsmail Tarık Atasoy, Mert Ege, Gökhan Polat","doi":"10.1111/hae.70155","DOIUrl":"https://doi.org/10.1111/hae.70155","url":null,"abstract":"Aim: To compare the frequency and timing of femoral- versus tibial-sided radiographic radiolucencies after posterior-stabilized TKA in people with haemophilia (PwH) versus non-haemophilic controls.Methods: A retrospective analysis was conducted of all primary posterior-stabilised total knee arthroplasties (TKA) performed in the centre between December 2003 and December 2018. Patients in Group 1 (n = 53) were age- and implant-matched to controls without haemophilia in Group 2 (n = 58). Radiographs were obtained at 3, 6 and annually to 5 years. Loosening was defined as progressive radiolucent lines >1 mm and/or migration/osteolysis, adjudicated by two blinded surgeons.Results: Mean age at surgery was 35.7 ± 9.2 years in Group 1 and 60.5 ± 11.8 years in Group 2 (p < 0.001). Femoral component loosening occurred in 22.6% of PwH versus 5.2% of controls (p = 0.011), whereas tibial loosening rates were 11.3% versus 8.6% (p = 0.755). Mean time to femoral loosening was 27.6 ± 16.8 months (Group 1) versus 31.3 ± 13.9 months (Group 2; p = 0.788), and to tibial loosening was 20.4 ± 18.2 months versus 9.6 ± 9.5 months (p = 0.134). Cox regression showed no significant hazard difference for femoral loosening (HR 0.99, 95% CI 0.93-1.05; p = 0.693). Post hoc power for detecting femoral loosening differences was ∼80%.Conclusions: PwH undergoing TKA showed a higher frequency of femoral-sided radiographic radiolucencies. These findings should be interpreted as potential predisposition to early loosening. Tibial findings were comparable between groups.","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-10-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145367936","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}

引用次数: 0

HAEMFIX: Impact of Switching From SHL-FIX to EHL-FIX in Patients With Haemophilia B. 血友病B患者从SHL-FIX切换到EHL-FIX的影响

IF 3 2区医学

Haemophilia Pub Date : 2025-10-25 DOI: 10.1111/hae.70157

Jasmin Lonardi, Susan Halimeh, Sylvia von Mackensen, Lisa Kleinlein, Juliet Fleischer, Henri Funk, Julia Hölz, Johannes Holzapfel, Sabrina Juranek, Victoria Lieftüchter, Christoph Bidlingmaier, Martin Olivieri

{"title":"HAEMFIX: Impact of Switching From SHL-FIX to EHL-FIX in Patients With Haemophilia B.","authors":"Jasmin Lonardi, Susan Halimeh, Sylvia von Mackensen, Lisa Kleinlein, Juliet Fleischer, Henri Funk, Julia Hölz, Johannes Holzapfel, Sabrina Juranek, Victoria Lieftüchter, Christoph Bidlingmaier, Martin Olivieri","doi":"10.1111/hae.70157","DOIUrl":"https://doi.org/10.1111/hae.70157","url":null,"abstract":"Introduction: Haemophilia B is an X-linked recessive bleeding disorder caused by coagulation factor IX (FIX) deficiency. Treatment involves intravenous replacement of FIX. Recently, extended half-life (EHL) FIX products have been introduced alongside standard half-life (SHL) products to optimize therapy.Aim: This study evaluated bleeding rates, joint health, factor consumption, dosage, and health-related quality of life (HRQoL) in patients switching from SHL- to EHL-FIX products, as well as in those exclusively treated with EHL-FIX.Methods: Retrospective data from the medical records of 37 children with haemophilia B treated between 2010 and 2023 at two German Haemophilia Care Centres were analysed. HRQoL was assessed cross-sectionally using haemophilia-specific and generic questionnaires.Results: Twenty-seven patients (median age: 12 years, range 2-19 years) switched from SHL- to EHL-FIX, while 10 received EHL-FIX from the start of prophylaxis. The mean annual bleeding rate (ABR) improved from 6.01 ± 7.01 (SHL) to 2.85 ± 3.42 (EHL). Factor consumption (159,577.8 ± 99,817.9 IU/year), dosage (118.9 ± 50.3 IU/kg/week) and infusion frequency (145 ± 35.6 infusions/year) decreased after switching (100,247.7 ± 46,268.6 IU/year; 56.4 ± 23.7 IU/kg/week; 55.1 ± 9.8 infusions/year). HRQoL improved in both self-reports and parent reports. No severe adverse events occurred.Conclusion: Switching from SHL-FIX to EHL-FIX in children with haemophilia B is safe and may improve outcomes by reducing bleeding rates, infusion frequency, and factor consumption while enhancing joint health and HRQoL.","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-10-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145367924","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}

引用次数: 0

The WFH Guidelines for the Management of Haemophilia: AAV Gene Therapy, 2025. WFH血友病管理指南：AAV基因治疗，2025。

IF 3 2区医学

Haemophilia Pub Date : 2025-10-25 DOI: 10.1111/hae.70113

Glenn F Pierce, Margareth C Ozelo, Johnny Mahlangu, Amy L Dunn, Marlene Beijlevelt, Rose Bender, Manuel Carcao, Donna Coffin, Enrico Ferri Grazzi, Graham R Foster, Julie Frantsve-Hawley, Alfonso Iorio, Shannon Jackson, Radoslaw Kaczmarek, Gwendolyn E Kaeser, Barbara A Konkle, Magdalena Lewandowska, Wolfgang Miesbach, Brian O'Mahony, Steven Pipe, Ulrike M Reiss, Amy K Wilson, Emna Gouider, Miguel Escobar

引用次数: 0

Supporting Patient-Centred Decisions on Gene Therapy for Haemophilia A: A Conceptual Model. 支持以患者为中心的A型血友病基因治疗决策：一个概念模型。

IF 3 2区医学

Haemophilia Pub Date : 2025-10-24 DOI: 10.1111/hae.70136

Giovanni Di Minno, Sarah Bigi, Serena Barello, Enrico Ferri Grazzi, Marco Follino, Maria Francesca Mansueto, Mariasanta Napolitano, Rita Carlotta Santoro, Giancarlo Castaman

引用次数: 0

Correction to "Abstract: The Investigation of Frequency of Prothrombin (FII) and Factor Leiden (FV) Mutations in Patients With Haemophilia". 对“摘要：血友病患者凝血酶原（FII）和莱顿因子（FV）突变频率的调查”的更正。

IF 3 2区医学

Haemophilia Pub Date : 2025-10-24 DOI: 10.1111/hae.70149

引用次数: 0

Burden of Care and Quality of Life Among Caregivers of Filipino Children With Haemophilia Before and During the COVID-19 Pandemic: A Cross-Sectional Study. 在COVID-19大流行之前和期间，菲律宾血友病儿童护理人员的护理负担和生活质量：一项横断面研究

IF 3 2区医学

Haemophilia Pub Date : 2025-10-17 DOI: 10.1111/hae.70148

Noah S Sacluti, Flerida G Hernandez

{"title":"Burden of Care and Quality of Life Among Caregivers of Filipino Children With Haemophilia Before and During the COVID-19 Pandemic: A Cross-Sectional Study.","authors":"Noah S Sacluti, Flerida G Hernandez","doi":"10.1111/hae.70148","DOIUrl":"https://doi.org/10.1111/hae.70148","url":null,"abstract":"Introduction: The pandemic affected the quality of life (QoL) of children with haemophilia, nevertheless their caregivers. The disease burden to caregivers and its influence on well-being may become a determinant of the quality of care they provide, impacting the overall patient's outcome.Aims: This study aimed to determine the effects of the pandemic on the caregiver burden, QoL, anxiety and/or depressive symptoms of caregivers.Methods: Validated questionnaires: Modified Caregiver Strain Index Pilipino (MCSI-P), Hospital Anxiety and Depression Scale (Pilipino) (HADS-P) and WHO-5 Well Being Index (Pilipino) were given to caregivers of paediatric haemophilia patients in the patient roster.Results: Among the 72 participants, 21 had caregiver strain/burden, while five had impaired QoL pre-pandemic. During the pandemic, the prevalence of caregiver strain (55.6%) and impairment in QoL (20.8%) significantly increased. While the pre-pandemic prevalence of caregiver burden was comparable from data in published literature, there was a significant increase during the pandemic. Anxiety (8.3%) and depressive symptoms (4.2%) among caregivers were below the pre-pandemic range and did not significantly increase during the pandemic. Impaired QoL was not associated with depressive/anxiety symptoms, but it was observed that caregivers showed more anxiety than depressive manifestations.Conclusion: The pandemic significantly increased the prevalence for caregiver burden. Increased rates of psychological symptoms were observed during the pandemic, but not statistically significant. There was no association between impaired QoL and caregivers' psychological symptoms. While much focus was given to the patients themselves, paramount attention to caregiver burden and compromised QoL should be evaluated and addressed as well.","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145312798","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}

引用次数: 0

Canadian Clinical Experience on Switching From Standard Half-life Recombinant Factor VIII (rFVIII), Octocog Alfa, to Extended Half-life rFVIII, Damoctocog Alfa Pegol, in Persons With Haemophilia A ≥ 12 Years Followed in a Comprehensive Haemophilia Care Program in Canada. 加拿大血友病综合护理项目中，A型血友病患者从标准半衰期重组因子VIII (rFVIII) （Octocog Alfa）转向延长半衰期rFVIII （Damoctocog Alfa Pegol）的临床经验

IF 3 2区医学

Haemophilia Pub Date : 2025-10-14 DOI: 10.1111/hae.70145

Davide Matino, Anthony K C Chan, Kay Decker, Emma Iserman, Andrea N Edginton, Olayide Oladoyinbo, Elisabetta Trinari, Arun Keepanasseril, Alfonso Iorio

引用次数: 0

Management of von Willebrand Disease With a Factor VIII-Poor von Willebrand Factor Concentrate: Results From the Paediatric Cohort of a Prospective Observational Post-Marketing Study. 血管性血友病的治疗与因子viii -贫血管性血友病因子浓缩：来自一项前瞻性观察性上市后研究的儿科队列结果

IF 3 2区医学

Haemophilia Pub Date : 2025-10-14 DOI: 10.1111/hae.70129

Jenny Goudemand, Annie Borel-Derlon, Ségolène Claeyssens, Hervé Chambost, Guillaume Mourey, Annie Harroche, Sandrine Meunier, Céline Henriet, Thierry Leroi, Sophie Susen, Yohann Repesse

{"title":"Management of von Willebrand Disease With a Factor VIII-Poor von Willebrand Factor Concentrate: Results From the Paediatric Cohort of a Prospective Observational Post-Marketing Study.","authors":"Jenny Goudemand, Annie Borel-Derlon, Ségolène Claeyssens, Hervé Chambost, Guillaume Mourey, Annie Harroche, Sandrine Meunier, Céline Henriet, Thierry Leroi, Sophie Susen, Yohann Repesse","doi":"10.1111/hae.70129","DOIUrl":"https://doi.org/10.1111/hae.70129","url":null,"abstract":"Introduction: Although clinical experience of a triple-secured, plasma-derived, von Willebrand factor (pdVWF), almost devoid of Factor VIII (FVIII) in adults with von Willebrand disease (VWD), is widely reported, its use in children is less documented.Aim: To explore the safety and efficacy of this concentrate in real-life, in children <12 years old.Methods: Data from 30 paediatric patients enrolled in a prospective, 3-year observational, post-marketing study in France were analysed. Efficacy and safety were assessed in two cohorts: 0-6 and 6-11 years old.Results: The population included 14 children <6 years of age and 16 children aged 6-11 years. Most patients (80%) were severely affected (von Willebrand factor ristocetin cofactor activity [VWF:RCo] ≤ 15 IU/dL), including 30% with Type 3 VWD. Children received pdVWF for 16 major bleeds, 138 minor bleeds, 7 major surgeries, 8 minor surgeries and 12 invasive procedures. Efficacy was rated excellent or good in 100% of major bleeds or surgeries. The dose per infusion was approximately 50 IU/kg. By age group, median doses per infusion varied from 49 to 73 IU/kg for patients <6 years and from 46 to 59 IU/kg for patients 6-11 years, according to clinical situation. FVIII coadministration/correction was more frequent in Type 3 VWD, regardless of patient's age. Of five children receiving long-term prophylaxis, breakthrough bleeding occurred in 1.6% of infusions and median annualised bleeding rate was 0.8. No safety concerns were raised.Conclusion: This analysis enlarges clinical experience of an FVIII-poor pdVWF in the paediatric population. This concentrate offers safe and effective treatment regardless of VWD severity.","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":"e70129"},"PeriodicalIF":3.0,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145286095","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}

引用次数: 0

Inhibitors and Obesity: The Prevalence and Strength of Association in People With Haemophilia. 抑制剂和肥胖：血友病患者的患病率和相关性。

IF 3 2区医学

Haemophilia Pub Date : 2025-10-13 DOI: 10.1111/hae.70139

Lisa Littner, Jianzhong Hu, Matthew Manuel, Christine Gerber, Cristina Tarango

{"title":"Inhibitors and Obesity: The Prevalence and Strength of Association in People With Haemophilia.","authors":"Lisa Littner, Jianzhong Hu, Matthew Manuel, Christine Gerber, Cristina Tarango","doi":"10.1111/hae.70139","DOIUrl":"https://doi.org/10.1111/hae.70139","url":null,"abstract":"Introduction: Haemophilia is an X-linked bleeding disorder characterized by deficient or defective clotting factors. Obesity in persons with haemophilia (PwH) is associated with joint mobility issues, decreased range of motion and increased target joints. Complications of inhibitors, antibodies that neutralize clotting factor concentrate, can limit physical activity, potentially exacerbating obesity risk. This study examines the association between obesity and inhibitor status in PwH and explores the impact of emicizumab, a bispecific monoclonal antibody, on obesity trends.Methods: A retrospective cohort analysis of the American Thrombosis and Hemostasis Network ATHNdataset from 2013 to 2021 was conducted. The study included 19,074 males with haemophilia A or B with at least one recorded body mass index measurement. Statistical analyses included logistic regression, generalized estimating equations and difference-in-difference models to assess obesity trends and associations with inhibitors and emicizumab use.Results: Obesity prevalence significantly increased in paediatric and adult PwH (p < 0.001). Children had a significantly increased risk of inhibitors compared to adults with either haemophilia A or B. A history of target joints and emicizumab use was associated with an increased obesity risk. No significant difference in obesity prevalence was found in adult or paediatric PwH with or without exposure to emicizumab. No significant association was observed between obesity and inhibitor status.Conclusion: Obesity prevalence in PwH continues to rise, aligning with trends in the general population. The link between target joints and obesity highlights the need to reduce obesity and improve joint health; future studies should explore causality and how physical activity mediates these associations.","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145280008","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}

引用次数: 0

Cover Image, Volume 31, Issue 4 封面图片，第31卷，第4期

IF 3 2区医学

Haemophilia Pub Date : 2025-10-11 DOI: 10.1111/hae.70142

Mathangi Kumar, Sulochana Badagabettu, Keerthilatha M. Pai, Annamma Kurien, Archana MV, Shreemathi S. Mayya, Sreejith Govindan

引用次数: 0

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