Understanding the Experiences of Young People Living With Haemophilia-A UK Social Media Study [0.03%]
英国社交媒体研究:了解活在阴影里的年轻人的生活体验——以血友病患者为例
Emma Gayton,Ranjit Nagra,Bekki Millward et al.
Emma Gayton et al.
Introduction: Haemophilia is a rare congenital bleeding disorder that presents significant physical and psychological challenges, especially for young people, with limited real-world evidence about their experiences in th...
Current State of Moderate Congenital Haemophilia A and the Need for Preventive Treatment [0.03%]
中度先天性甲型血友病的现状及预防治疗的需求
Masahiro Takeyama,Atsuki Yamashita,Tomoko Yamaguchi et al.
Masahiro Takeyama et al.
Introduction: The severity of haemophilia A is classified by the degree of factor VIII (FVIII) deficiency, rather than by clinical manifestations. However, FVIII activity alone does not necessarily accurately reflect clin...
Quality of Life in People With Haemophilia: Results in the Framework of a Nationwide Registry in Colombia [0.03%]
哥伦比亚国家血友病登记处框架下血友病患者的生活质量结果分析
Juliana Alexandra Hernández Vargas,Adriana Linares,María Helena Solano et al.
Juliana Alexandra Hernández Vargas et al.
Introduction: Real-world data on health-related quality of life (HRQoL) and its predictors in people with haemophilia (PWH) are crucial to evaluate the effect of comprehensive management. It may be useful for clinicians a...
Lack of Inhibitor Development in Previously Treated People With Haemophilia Switching Factor Products-Final Results of Athn 2: Factor Switching Study [0.03%]
甲型血友病患者接受过因子类产品治疗后缺乏抑制物的产生——ATHN2:关于因子转换的研究最终结果
Erin Cockrell,Martin Chandler,Tammuella Chrisentery-Singleton et al.
Erin Cockrell et al.
The XX Factor in Hemophilia: Diagnostic, Therapeutic, and Research Gaps for Women and Girls [0.03%]
血友病中的XX因素:女性和女孩的诊断、治疗和研究缺口
Kelsey Uminski,Ellen Cusano
Kelsey Uminski
Introduction: Hemophilia has traditionally been viewed as an X-linked disorder affecting men and boys, with women and girls labeled as "carriers", presumed to be clinically unaffected. This paradigm has contributed to und...
Patient-Reported Outcome Measures and Clinical Performance in Hemophilia: Implications for Person-Centered Care [0.03%]
血友病患者报告结果测量及临床疗效对以病人为中心的治疗模式的意义
Tatyane Oliveira Rebouças Brito,Luiz Ivando Pires Ferreira Filho,Luany Elvira Mesquita Carvalho et al.
Tatyane Oliveira Rebouças Brito et al.
Objectives: To examine the association between patient-reported outcome measures (PROMs) and objective measures of clinical performance in children, adolescents, and adults with haemophilia, highlighting their implication...
Why "Treat First" Stalls: Knowledge, Scope, and System Determinants of Delayed Haemostatic Therapy in the Emergency Department [0.03%]
为什么“先治疗”会延迟:急诊科止血治疗延误的知识、范围和系统决定因素
Emily Kraft,Lindsay Cowley,Vanessa Bourck et al.
Emily Kraft et al.
Background: Persons with bleeding disorders (PwBD) frequently experience delays to haemostatic therapy in emergency departments (EDs). Identifying clinician knowledge gaps and workflow barriers is essential to accelerate ...
Extended Half-Life Recombinant Factor VIII Conjugated with Modifying Substances Does Not Affect Fibrin Clot Formation or Stability in Haemophilia A Blood Samples [0.03%]
带有修饰物的长效重组凝血因子VIII不影响甲型血友病血液样品中纤维蛋白凝块的形成或稳定性
Naruto Shimonishi,Keiji Nogami
Naruto Shimonishi
Introduction: Various extended half-life recombinant factor VIII (EHL-FVIII) products have been designed to improve the pharmacokinetic properties of FVIII, allowing prolonged haemostatic coverage and reducing the injecti...
The Analysis of the FIX-inhibitor Risks Associated With the F9 Genotype in Patients With Haemophilia B Exposes the Involvement of Nonsense Mediated-decay: Argentinean and International Series [0.03%]
FIX抑制剂的风险与血友病B患者的F9基因型相关的分析揭示了 nonsense介导的衰减作用:阿根廷和国际系列研究
Betiana Michelle Ziegler,Miguel Martín Abelleyro,María Emilia Villegas et al.
Betiana Michelle Ziegler et al.
Introduction: Haemophilia B (HB) associates with deleterious variants in F9. HB management with FIX-concentrate infusions may be ineffective in 3%-9% patients who develop FIX-inhibitors. ...
Prevalence of F8 Intron 22 Inversion in Severe Haemophilia A: Molecular Insights From a Cohort of Punjab Province of Pakistan [0.03%]
巴基斯坦旁遮普省重度甲型血友病患者中F8第22内含子倒位的流行率:队列分子见解
Muhammad Kashif Mughal,Ghulam Mustafa,Yusra Rashid et al.
Muhammad Kashif Mughal et al.
Introduction: Haemophilia A (HA) is caused by an inherited deficiency of factor VIII. Intron 22 inversion is the most common genetic mutation that causes severe disease. The study aims to determine the prevalence of F8 In...