HaemophiliaPub Date : 2025-09-30DOI: 10.1111/hae.70133
Chieh-Yu Lin, Peng-Ta Liu, Ming-Ching Shen, Shao-Li Han
{"title":"Developmental Changes in Haemophilic Knee Joint Morphology: A Geometric Morphometric Study.","authors":"Chieh-Yu Lin, Peng-Ta Liu, Ming-Ching Shen, Shao-Li Han","doi":"10.1111/hae.70133","DOIUrl":"https://doi.org/10.1111/hae.70133","url":null,"abstract":"<p><strong>Introduction: </strong>Despite the well-established lifelong impact of haemophilia on joint health, there remains a significant gap in our understanding of the developmental morphological changes that occur in the bones of haemophilic individuals. This study applied geometric morphometric measurement (GMM) to analyse age-related morphological variations in the knee joints of haemophilia patients.</p><p><strong>Materials and methods: </strong>This retrospective study analysed 210 anteroposterior knee radiographs from 15 male haemophilia patients (ages 8-24), grouped into 8-15, 16-20, and 21-24 years. Twenty 2D bony landmarks were annotated by two raters (ICC = 0.98). Generalized Procrustes analysis, principal component analysis, Procrustes ANOVA, and canonical variate analysis were performed. Associations with Pettersson score (PS) and Haemophilia Joint Health Score (HJHS) were examined via multivariate regression.</p><p><strong>Results: </strong>The diversity in morphology of knee joints among the first five principal components was 83%. Procrustes ANOVA indicated significant shape differences (p < 0.001) among age groups. Furthermore, discriminant function analysis revealed the highest Mahalanobis distance (3.6212) between age group 1 and group 3, with significant differences (p < 0.001). Notable morphological alterations were identified, characterized by a medial displacement of the patella and a reduction in joint space.</p><p><strong>Conclusion: </strong>This study revealed significant age-related morphological alterations in the knee joints of individuals with hemophilia. The position of the patella was identified as a key morphological marker, suggesting its potential utility for long-term follow-up and the development of targeted rehabilitation programs.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145191609","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 : 2025-09-29DOI: 10.1111/hae.70138
Christopher Reilly-Stitt, Ian Jennings, Anna Williams, Steve Kitchen, Laura Aiken, Sean Platton, Priyanka Raheja, Will Lester
{"title":"UKNEQAS Blood Coagulation Factor Nine (FIX) Gene Therapy Supplementary Exercise 2025.","authors":"Christopher Reilly-Stitt, Ian Jennings, Anna Williams, Steve Kitchen, Laura Aiken, Sean Platton, Priyanka Raheja, Will Lester","doi":"10.1111/hae.70138","DOIUrl":"https://doi.org/10.1111/hae.70138","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145185490","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 : 2025-09-26DOI: 10.1111/hae.70134
Alok Srivastava, Savita Rangarajan, Cecil Ross, Shariq Ali, Shauna Andersson, Laurel A Menapace, Marja Puurunen, Marion Afonso
{"title":"Quality of Life and Treatment Satisfaction in People With Haemophilia on Fitusiran Prophylaxis: Evidence From a Subset of ATLAS-OLE Trial Participants Mainly From India.","authors":"Alok Srivastava, Savita Rangarajan, Cecil Ross, Shariq Ali, Shauna Andersson, Laurel A Menapace, Marja Puurunen, Marion Afonso","doi":"10.1111/hae.70134","DOIUrl":"https://doi.org/10.1111/hae.70134","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145148767","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 : 2025-09-16DOI: 10.1111/hae.70127
Anna Olsson, Petra Elfvinge, Eva Zetterberg, Linda Myrin-Westesson
{"title":"Prevalence and Impact of Heavy Menstrual Bleeding in Women With von Willebrand Disease Across Age Groups: A Retrospective Study.","authors":"Anna Olsson, Petra Elfvinge, Eva Zetterberg, Linda Myrin-Westesson","doi":"10.1111/hae.70127","DOIUrl":"https://doi.org/10.1111/hae.70127","url":null,"abstract":"<p><strong>Introduction: </strong>Heavy menstrual bleeding (HMB) is a common and burdensome symptom in women with von Willebrand disease (VWD). Although VWD and HMB have been studied, the prevalence and impact of HMB across different age groups remains underexplored.</p><p><strong>Aim: </strong>To investigate the prevalence and impact of HMB in women with VWD across different age groups.</p><p><strong>Methods: </strong>This retrospective nationwide study included 136 women aged 18-55 years with type 1 (von Willebrand factor activity ≤ 0.35 IU/mL), 2 or 3 VWD. Data were collected using a questionnaire assessing HMB and its impact on life, and clinical data were retrieved from medical records.</p><p><strong>Results: </strong>HMB was reported by 93% of women in one or more age groups. During adolescence, 85% reported HMB. Reported HMB decreased with age: 63% at 20-29 years, 45% at 30-39 years, and 36% at 40-55 years. Of the 136 women, 60% had sought medical care due to HMB. Forty-nine percent of these had sought medical care at two or more healthcare facilities. The majority (86%) stated that HMB had negatively impacted their lives. Several aspects were affected, including school/work performance, social and physical activities, with a decreasing impact observed with increasing age. Despite the use of hormonal therapy (76%) and/or haemostatic agents (76%), 54% had had iron deficiency. Thirty-five percent were diagnosed after age 19, and 27% were lost to follow-up.</p><p><strong>Conclusions: </strong>The findings emphasise the need for increased awareness during adolescence, more structured follow-up and improved individualised management strategies to prevent and treat HMB.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069314","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":"Arthroscopic Ankle Surgery Under Ultrasound-Guided Peripheral Nerve Block in Patients With Haemophilic Ankle Arthropathy-A Report of Five Cases.","authors":"Yasunari Ikuta, Tomoyuki Nakasa, Teruhisa Fujii, Naoya Yamasaki, Saori Ishibashi, Satoru Sakurai, Dan Moriwaki, Nobuo Adachi","doi":"10.1111/hae.70132","DOIUrl":"https://doi.org/10.1111/hae.70132","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069336","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 : 2025-09-16DOI: 10.1111/hae.70131
Yesim Dargaud, Arianna Colombo, Maria Elisa Mancuso
{"title":"The Mirage of Factor Equivalence: Examining the Complexities of Non-Factor Therapies in Haemophilia.","authors":"Yesim Dargaud, Arianna Colombo, Maria Elisa Mancuso","doi":"10.1111/hae.70131","DOIUrl":"https://doi.org/10.1111/hae.70131","url":null,"abstract":"<p><strong>Background: </strong>This article provides a critical analysis of the 'factor equivalence' concept as applied to non-factor therapies (NFTs) for haemophilia, highlighting its limitations and clinical implications. Although factor equivalence estimates serve as a comparative tool for evaluating pro-coagulant effects across different therapies, they may fail to accurately represent the true haemostatic potential or clinical efficacy due to the diverse mechanisms of action of different molecules and patient-specific factors.</p><p><strong>Aim: </strong>The article challenges the widespread adoption of factor equivalence as a universal benchmark to quantify the haemostatic potential of NFTs.</p><p><strong>Discussion and conclusion: </strong>It advocates for patient- and context-specific laboratory assessments that reflect each therapy's unique pharmacological profile. This approach is particularly relevant to enhance personalised treatment strategies, especially in high-risk situations such as surgery or severe breakthrough bleeding, thereby optimising the use of novel haemostatic therapies.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069385","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 : 2025-09-16DOI: 10.1111/hae.70128
Barbara Konkle, Donna Coffin, Cédric Hermans, Mayss Naccache, Brian O'Mahony, Glenn F Pierce
{"title":"The Critical Need to Consolidate All Gene Therapy Data in Haemophilia.","authors":"Barbara Konkle, Donna Coffin, Cédric Hermans, Mayss Naccache, Brian O'Mahony, Glenn F Pierce","doi":"10.1111/hae.70128","DOIUrl":"https://doi.org/10.1111/hae.70128","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":"e70128"},"PeriodicalIF":3.0,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145069353","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 : 2025-09-16DOI: 10.1111/hae.70126
{"title":"Correction to \"Extension Study With rVIII-SingleChain in Previously Untreated Patients (PUPs) With Severe Hemophilia A\".","authors":"","doi":"10.1111/hae.70126","DOIUrl":"https://doi.org/10.1111/hae.70126","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145075176","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}