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Impact of Systematic Joint Examination (Ultrasound, Functional and Physical) on Treatment Management Decisions in Patients With Haemophilia A in France: Final Data From the Prospective, Observational A-MOVE Study 系统的联合检查(超声、功能和物理)对法国A型血友病患者治疗管理决策的影响:来自前瞻性观察性A- move研究的最终数据
IF 3 2区 医学
Haemophilia Pub Date : 2025-02-24 DOI: 10.1111/hae.70012
Nicolas Drillaud, Virginie Barbay, Jean Baptiste Valentin, Romain Jailler, Aurélien Lebreton, Brigitte Pan-Petesch, Sabine Marie Castet, Birgit Frotscher, Laurent Frenzel, Sandrine Jousse-Joulin, Hervé Chambost, Mikaela Alenäs, Markus Fusser, Corinne Gandossi, Meriem Zidi, Oussama Mahdout, Yohann Repessé, the A-MOVE study group
{"title":"Impact of Systematic Joint Examination (Ultrasound, Functional and Physical) on Treatment Management Decisions in Patients With Haemophilia A in France: Final Data From the Prospective, Observational A-MOVE Study","authors":"Nicolas Drillaud,&nbsp;Virginie Barbay,&nbsp;Jean Baptiste Valentin,&nbsp;Romain Jailler,&nbsp;Aurélien Lebreton,&nbsp;Brigitte Pan-Petesch,&nbsp;Sabine Marie Castet,&nbsp;Birgit Frotscher,&nbsp;Laurent Frenzel,&nbsp;Sandrine Jousse-Joulin,&nbsp;Hervé Chambost,&nbsp;Mikaela Alenäs,&nbsp;Markus Fusser,&nbsp;Corinne Gandossi,&nbsp;Meriem Zidi,&nbsp;Oussama Mahdout,&nbsp;Yohann Repessé,&nbsp;the A-MOVE study group","doi":"10.1111/hae.70012","DOIUrl":"10.1111/hae.70012","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Haemophilia management aims to prevent bleeding and preserve joint function. Changes in patients’ joint health may influence physicians' decisions to adjust treatment. The Haemophilia Joint Health Score (HJHS) and Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) score assess joint health but are not routinely used.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>To evaluate whether systematic joint examination with HJHS and/or HEAD-US had an impact on treatment management decisions in France, using final data from the A-MOVE study.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A-MOVE (NCT04133883) was a 12-month prospective, multicentre study, which enrolled persons with haemophilia A (all severities, aged 6–40 years) treated prophylactically or on demand with standard/extended half-life FVIII replacement. At baseline, 6 and 12 months, HJHS/HEAD-US and changes in patients’ management were assessed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Eighty-six patients from 20 sites were included in the final analysis; 68 had HJHS/HEAD-US assessments at 12 months. Over 12 months, 24.4% (<i>n</i> = 21/86) of patients experienced an impact on their haemophilia management due to HJHS/HEAD-US scores; these decisions were impacted by HJHS in about half of the patients (52.4%, <i>n</i> = 11/21) and HEAD-US in almost all patients (95.2%, <i>n</i> = 20/21). Both assessments contributed to a change in management decisions in about half of the patients (47.6%, <i>n</i> = 10/21). Twenty-nine patients (33.7%) had haemophilia management decisions impacted by factors other than HJHS/HEAD-US, including physical examination findings (<i>n</i> = 9) and the occurrence of bleeding episodes (<i>n</i> = 8).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Final data from the A-MOVE study show that systematic joint assessments, through functional/physical examination (HJHS) and ultrasound (HEAD-US), may impact treatment management decisions in persons with haemophilia A.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 2","pages":"239-246"},"PeriodicalIF":3.0,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70012","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143491750","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}
引用次数: 0
Development of a Single-Tube Reverse Transcription Real-Time PCR Assay for Diagnosis of F8 Intron 22 Inversion in Patients and Carriers of Haemophilia A a型血友病患者及携带者F8内含子22倒置单管反转录实时PCR检测方法的建立
IF 3 2区 医学
Haemophilia Pub Date : 2025-02-24 DOI: 10.1111/hae.70014
Javier Arzuaga-Mendez, Lucia Insunza, María José Ojinaga, Ana Moretó, Maite Moreno-Gamiz, Miriam Vara-Pampliega, Juan Carlos Garcia-Ruiz, Cristina Sierra-Aisa
{"title":"Development of a Single-Tube Reverse Transcription Real-Time PCR Assay for Diagnosis of F8 Intron 22 Inversion in Patients and Carriers of Haemophilia A","authors":"Javier Arzuaga-Mendez,&nbsp;Lucia Insunza,&nbsp;María José Ojinaga,&nbsp;Ana Moretó,&nbsp;Maite Moreno-Gamiz,&nbsp;Miriam Vara-Pampliega,&nbsp;Juan Carlos Garcia-Ruiz,&nbsp;Cristina Sierra-Aisa","doi":"10.1111/hae.70014","DOIUrl":"10.1111/hae.70014","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Intron 22 inversion mutation of F8 (inv22) is the most frequent cause of Haemophilia A (HA) and is present in approximately 45% of severe HA cases. This mutation disrupts <i>F8</i> gene continuity, leading to a truncated protein. Traditional methods for detecting inv22, including inverse-shifting PCR and long-range PCR, are accurate but labour-intensive. F8 inv22 truncated mRNA transcript contains a short (51 base pairs) abnormal exon 23. Thus, reverse-transcription PCR has been proposed for the diagnosis of inv22 in patients with HA.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>The aim of this study was to design and validate a multiplex reverse-transcription real-time PCR (RT-qPCR) assay capable of detecting and differentiating between normal and inv22 F8 transcripts in a single reaction.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We designed an RT-qPCR assay that employs specific primers and TaqMan probes to detect the exon 22 to 23 junction present in the normal F8 transcript, and the junction between exon 22 and the abnormal sequence only present in F8 transcripts harbouring inv22. We tested our assay in 14 HA patients (six with inv22 and eight with other mutations), four HA female carriers (two with inv22 and two with other mutations), and six negative controls.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p><i>F8</i> expression in peripheral blood RNA was sufficient to be detected by RT-qPCR. The assay showed perfect concordance within the cohort to identify inv22 in both patients and carriers.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>RT-qPCR is an accurate method for diagnosing inv22 in patients and HA carriers. Moreover, it is simpler and faster than previous methods.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 2","pages":"263-268"},"PeriodicalIF":3.0,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143491627","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
American Thrombosis and Hemostasis Network: National Bleeding Disorders Foundation Data Commons: Better Together. 美国血栓和止血网络:国家出血性疾病基金会数据共享:更好地在一起。
IF 3 2区 医学
Haemophilia Pub Date : 2025-02-21 DOI: 10.1111/hae.70011
Tammuella E Chrisentery-Singleton, Philip M Gattone, Christine M Gerber, Kevin D Mills, Moses E Miles, Dawn Rotellini, Maria E Santaella, Leonard A Valentino, Crystal Watson, Michael Recht
{"title":"American Thrombosis and Hemostasis Network: National Bleeding Disorders Foundation Data Commons: Better Together.","authors":"Tammuella E Chrisentery-Singleton, Philip M Gattone, Christine M Gerber, Kevin D Mills, Moses E Miles, Dawn Rotellini, Maria E Santaella, Leonard A Valentino, Crystal Watson, Michael Recht","doi":"10.1111/hae.70011","DOIUrl":"https://doi.org/10.1111/hae.70011","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143472446","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
Haemophilia's Heartland: Pain, Poverty and Patient Care in Faisalabad 血友病的心脏地带:费萨拉巴德的痛苦、贫穷和病人护理。
IF 3 2区 医学
Haemophilia Pub Date : 2025-02-21 DOI: 10.1111/hae.70009
Eesha Yaqoob, Shiza Aftab, Noor-ul-Ain Shah, Nimirta Sahitia, Saad Javed
{"title":"Haemophilia's Heartland: Pain, Poverty and Patient Care in Faisalabad","authors":"Eesha Yaqoob,&nbsp;Shiza Aftab,&nbsp;Noor-ul-Ain Shah,&nbsp;Nimirta Sahitia,&nbsp;Saad Javed","doi":"10.1111/hae.70009","DOIUrl":"10.1111/hae.70009","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Haemophilia is an inherited bleeding disorder that causes significant pain and disability. Haemophilia A and B are the most common, with HemA affecting more men and being four times more prevalent.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A cross-sectional study was conducted in Faisalabad, Pakistan, to assess pain severity in haemophilia patients based on sociodemographic factors and management approaches. Data were collected through structured interviews and analysed using SPSS version 27.0, examining associations between variables and pain severity.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A sample of 200 patients was selected from the 800 registered patients at a haemophilia treatment centre (HTC) in Faisalabad. All participants had severe haemophilia, with 65.5% residing in rural areas. Severe pain was reported by 58% of patients, with higher prevalence among rural residents (35% vs. 22.5% urban, <i>p</i> = 0.004) and those with monthly income below Rs. 15,000 (21%, <i>p</i> &lt; 0.001). Plasma transfusion significantly reduced severe pain risk (OR = 0.59, 95% CI: 0.42–0.83, <i>p</i> = 0.003), while self-management methods increased it (OR = 1.79, 95% CI: 1.06–3.02, <i>p</i> = 0.03). Distance from treatment centres significantly impacted pain severity, with 21.5% of patients living within 10–50 miles reporting severe pain. Management practices significantly influenced patient outcomes (<i>p</i> &lt; 0.001).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Patients face significant pain management challenges owing to sociodemographic factors, low income and limited access to specialized care. Addressing these gaps requires early diagnosis, better access to treatment centres and multidisciplinary pain management strategies. While geographic and economic barriers are considered, future research should include detailed data on rural healthcare quality, assimilate longitudinal data and delve into the links between mental health, pain severity and treatment ease of access.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 2","pages":"231-238"},"PeriodicalIF":3.0,"publicationDate":"2025-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143472447","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
Investigation of the Relationship Between Self-Management and Kinesiophobia in Haemophiliac Individuals Experiencing Intra-Articular Bleeding 血友病患者关节内出血患者自我管理与运动恐惧症关系的研究。
IF 3 2区 医学
Haemophilia Pub Date : 2025-02-21 DOI: 10.1111/hae.70013
Dilan Çiftci Akan, Mehmet Can Uğur, Esra Oksel
{"title":"Investigation of the Relationship Between Self-Management and Kinesiophobia in Haemophiliac Individuals Experiencing Intra-Articular Bleeding","authors":"Dilan Çiftci Akan,&nbsp;Mehmet Can Uğur,&nbsp;Esra Oksel","doi":"10.1111/hae.70013","DOIUrl":"10.1111/hae.70013","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>This study aims to examine the relationship between self-management skills and kinesiophobia in haemophilia patients. The findings of the study may contribute to the development of strategies that can help haemophilia patients overcome their fear of movement and improve their quality of life (QoL).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>This study is planned to examine the relationship between disease self-management in haemophiliac individuals and kinesiophobia that may arise from intra-articular bleeding associated with haemophilia.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>In this study, both descriptive and analytical research was conducted on individuals who are members of patient associations. Patient Identification Form, Chronic Disease Self-Management Scale and Tampa Scale for Kinesiophobia were used as data collection tools. Data were collected face to face. Descriptive statistics methods (mean, standard deviation) were used in the evaluation of study data. Quantitative data comparisons were made with the Student's <i>t</i>-test for normally distributed parameters and the Mann–Whitney <i>U</i> test for non-normally distributed parameters. When there were more than two groups, One-Way ANOVA was used, and in advanced analyses, linear regression analysis was used.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A positive correlation was found between the ‘self-stigmatization’ subdimension of the Chronic Disease Self-Management Scale and kinesiophobia. Additionally, a negative correlation was observed between healing compliance and kinesiophobia levels. Flexibility in healing cracks was found in the reduction of kinesiophobia.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Self-management of the disease in hemophilic individuals affects compliance with treatment, kinesiophobia, activity level and QoL.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 2","pages":"304-312"},"PeriodicalIF":3.0,"publicationDate":"2025-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70013","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143472449","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}
引用次数: 0
Psychosocial Care During Novel Therapies in Haemophilia: A Psychological Framework. 血友病新疗法中的社会心理护理:一个心理学框架。
IF 3 2区 医学
Haemophilia Pub Date : 2025-02-17 DOI: 10.1111/hae.70008
Lotte Haverman, Lorynn Teela, Gráinne O'Brien, Petra Bučková, Gaby Golan, Gloria Rooney, Karolien Bartels, Ana Torres-Ortuño, Christina Burgess
{"title":"Psychosocial Care During Novel Therapies in Haemophilia: A Psychological Framework.","authors":"Lotte Haverman, Lorynn Teela, Gráinne O'Brien, Petra Bučková, Gaby Golan, Gloria Rooney, Karolien Bartels, Ana Torres-Ortuño, Christina Burgess","doi":"10.1111/hae.70008","DOIUrl":"https://doi.org/10.1111/hae.70008","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-02-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143440649","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
Recommendations for Psychosocial Support during Gene Therapy: Results From an EAHAD Interdisciplinary Roundtable. 基因治疗中的社会心理支持建议:来自EAHAD跨学科圆桌会议的结果。
IF 3 2区 医学
Haemophilia Pub Date : 2025-02-14 DOI: 10.1111/hae.70005
Lorynn Teela, Gráinne O'Brien, Petra Bučková, Gaby Golan, Gloria Rooney, Karolien Bartels, Ana Torres-Ortuño, Christina Burgess, Lotte Haverman
{"title":"Recommendations for Psychosocial Support during Gene Therapy: Results From an EAHAD Interdisciplinary Roundtable.","authors":"Lorynn Teela, Gráinne O'Brien, Petra Bučková, Gaby Golan, Gloria Rooney, Karolien Bartels, Ana Torres-Ortuño, Christina Burgess, Lotte Haverman","doi":"10.1111/hae.70005","DOIUrl":"https://doi.org/10.1111/hae.70005","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-02-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143414006","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
Factor VIII Activity and Factor VIII Inhibitors Can Be Measured Accurately in Plasma Containing Mim8 by Using Specific Chromogenic Assays 因子VIII活性和因子VIII抑制剂可以用特异性显色法准确测量含有Mim8的血浆。
IF 3 2区 医学
Haemophilia Pub Date : 2025-02-14 DOI: 10.1111/hae.70007
William Pickering, Mary Robinson, Caroline Cogswell, Wan Hui Ong Clausen, Karin Nana Weldingh, Mirella Ezban
{"title":"Factor VIII Activity and Factor VIII Inhibitors Can Be Measured Accurately in Plasma Containing Mim8 by Using Specific Chromogenic Assays","authors":"William Pickering,&nbsp;Mary Robinson,&nbsp;Caroline Cogswell,&nbsp;Wan Hui Ong Clausen,&nbsp;Karin Nana Weldingh,&nbsp;Mirella Ezban","doi":"10.1111/hae.70007","DOIUrl":"10.1111/hae.70007","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Patients with Haemophilia A treated with Mim8 may require concomitant Factor VIII (FVIII) replacement; hence, accurate assessment of FVIII activity (FVIII:C) and FVIII inhibitors in the presence of Mim8 is important.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>Assess which chromogenic substrate assays (CSAs) accurately monitor FVIII:C and FVIII inhibitor levels in the presence of Mim8.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>FVIII and Mim8 were spiked into congenital FVIII-deficient plasma to obtain mixtures containing various FVIII:C levels (0–100 IU/dL) and Mim8 concentrations (0, 3, 6 and 12 µg/mL). Five CSAs using different activated Factor IX and X sources (bovine, bovine–human or human) were used to measure FVIII:C. Bovine and bovine–human CSAs were then used to measure FVIII:C of standard half-life (SHL) and extended half-life (EHL) FVIII products. FVIII inhibitor levels were assessed using two different bovine CSAs in congenital FVIII-deficient plasma spiked with various Mim8 concentrations (5, 10, 20 and 40 µg/mL) and FVIII inhibitor levels (0.2, 1.0 and 4.8 BU).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>High levels of interference were observed using human CSAs. Bovine CSAs accurately measured FVIII:C of SHL and EHL FVIII products in the presence of Mim8 without interference. In bovine–human CSAs, interference was observed at 5 IU/dL FVIII, increasing up to four-fold with increasing Mim8 levels. FVIII inhibitor levels were accurately measured using bovine CSAs without Mim8 interference.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>FVIII:C of SHL and EHL products and FVIII inhibitor levels can be accurately monitored in the presence of Mim8 using bovine CSAs at all FVIII levels, and bovine–human CSAs at FVIII concentrations &gt;20 IU/dL.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 2","pages":"319-327"},"PeriodicalIF":3.0,"publicationDate":"2025-02-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.70007","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143414003","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}
引用次数: 0
Comprehensive Analysis of Surgical Interventions for Musculoskeletal Disorders in Patients With Bleeding Disorders: Report From a Subspecialty Unit in a Developing Country. 出血性疾病患者肌肉骨骼疾病手术干预的综合分析:来自一个发展中国家亚专科单位的报告。
IF 3 2区 医学
Haemophilia Pub Date : 2025-02-14 DOI: 10.1111/hae.70010
Arman Vahabi, Abdussamet Kuyucu, Elcil Kaya Biçer, Fahri Şahin, Kaan Kavaklı, Semih Aydoğdu
{"title":"Comprehensive Analysis of Surgical Interventions for Musculoskeletal Disorders in Patients With Bleeding Disorders: Report From a Subspecialty Unit in a Developing Country.","authors":"Arman Vahabi, Abdussamet Kuyucu, Elcil Kaya Biçer, Fahri Şahin, Kaan Kavaklı, Semih Aydoğdu","doi":"10.1111/hae.70010","DOIUrl":"https://doi.org/10.1111/hae.70010","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-02-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143413951","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
Bleeding Episodes in Patients With Haemophilia B Receiving Prophylactic Factor IX Treatment: A Systematic Review and Meta-Analysis 接受预防性因子IX治疗的B型血友病患者出血事件:系统回顾和荟萃分析
IF 3 2区 医学
Haemophilia Pub Date : 2025-02-14 DOI: 10.1111/hae.70006
Massimo Franchini, Samantha Pasca, Carlo Mengoli, Daniele Focosi, Pier Mannuccio Mannucci
{"title":"Bleeding Episodes in Patients With Haemophilia B Receiving Prophylactic Factor IX Treatment: A Systematic Review and Meta-Analysis","authors":"Massimo Franchini,&nbsp;Samantha Pasca,&nbsp;Carlo Mengoli,&nbsp;Daniele Focosi,&nbsp;Pier Mannuccio Mannucci","doi":"10.1111/hae.70006","DOIUrl":"10.1111/hae.70006","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Prophylaxis with coagulation factor concentrates is the mainstay of treatment in severe hemophilia A and B. Data on bleeding rates in persons with congenital haemophilia B (PwcHB) receiving prophylaxis are inconsistent.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>This systematic review and meta-analysis were aimed at assessing bleeding outcomes, including annualised bleeding rates (ABR) and the proportion of patients with zero bleeding events, in PwcHB receiving prophylaxis with plasma-derived or recombinant FIX products with standard (rSHL) or extended half-life (rEHL).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A systematic search was conducted using the bibliographic database Medline, Embase and Cochrane Central Register. The protocol was registered on PROSPERO (registration number: CRD42024592785).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The search yielded 2440 citations and a total of 42 studies (2 randomised and 40 nonrandomised) were included in the final analysis. The pooled estimated mean (95% confidence interval [CI]) ABR was significantly lower in PwcHB treated prophylactically with rEHL FIX than in those receiving rSHL FIX products (1.29 [95% CI: 0.91, 1.66] vs. 3.12 [95% CI: 2.63, 3.62], <i>p</i> &lt; 0.01). The proportion of participants with zero bleeding events was significantly higher in PwcHB treated prophylactically with rEHL FIX than in those receiving rSHL FIX (0.53 [95% CI: 0.37, 0.69] vs. 0.24 [95% CI: 0.14, 0.39], <i>p</i> = 0.01). The ABR did not differ according to age groups (more or less than 12 years).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The results of this meta-analysis suggest that compared to standard half-life FIX concentrates, prophylaxis with rEHL FIX products is associated with a reduction in ABR and a higher proportion of patients with no bleeding episodes.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 2","pages":"173-186"},"PeriodicalIF":3.0,"publicationDate":"2025-02-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143413950","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}
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