Haemophilia最新文献

{"title":"Assessment of joint health in females with haemophilia: The carriers ultrasound project (CUP) study","authors":"Fernando F. Corrales-Medina,&nbsp;Kelli Fraga,&nbsp;Maria D'Almeida Bastos,&nbsp;Amina Rafique,&nbsp;Christine L. Kempton,&nbsp;Pooja Vijayvargia,&nbsp;Joanna A. Davis,&nbsp;Rachel S. Kronenfeld","doi":"10.1111/hae.15128","DOIUrl":"10.1111/hae.15128","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>The needs of haemophilia carriers (HC) have been historically overlooked. It is now recognised that HC manifests bleeding symptoms, including haemarthrosis. The natural history of joint health in HC is not yet defined.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>A multi-institutional cross-sectional study aimed to evaluate the characteristics of joint disease in HC, aged 18–40 years, compared to age-matched controls.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The carrier cohort included females with confirmed HC status. Controls had no personal or family history of bleeding disorders. All females with a history of joint trauma or surgery within 12 months or any history of joint replacement were excluded. Joint health was assessed by clinical history, Haemophilia Joint Health Score (HJHS) and point-of-care musculoskeletal ultrasonography (POC-MSKUS).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Thirty HC and 30 controls were enrolled. For HC, the median factor activity level was 52% (range 17%–100%). Carriers, regardless of baseline factor activity levels, reported higher prevalence of chronic joint pain (<i>p</i> &lt; .001) and swelling (<i>p</i> = .002) than controls. Heavy menstrual bleeding, epistaxis, gingival bleeding and easy bruising were also more prevalent in HC (<i>p</i> &lt; .001). Despite HC having a higher median HJHS score (5 vs. 0, <i>p</i> &lt; .001), no differences were observed when using POC-MSKUS. HC with a body mass index ≥25 mg/m² reported more haemarthrosis (<i>p</i> = .037).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>HC are at increased risk of joint-related symptoms and poorer joint health than age-matched controls. Dedicated follow-up to prevent and treat joint disease in HC is imperative. This study is also a call for additional investigation to clarify the association, or lack thereof, between factor activity and joint disease.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"30 6","pages":"1422-1428"},"PeriodicalIF":3.0,"publicationDate":"2024-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.15128","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142738966","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":"A core outcome set for prophylaxis and perioperative treatment of von Willebrand disease: The coreVWD initiative","authors":"Elizabeth Clearfield,&nbsp;Benjamin Kim,&nbsp;Sarah Ford,&nbsp;Nathan T. Connell,&nbsp;Maria E. Santaella,&nbsp;Michelle Lavin,&nbsp;Manon E. L. Degenaar-Dujardin,&nbsp;Emily Ayoub,&nbsp;Veronica H. Flood,&nbsp;Dawn Rotellini,&nbsp;Mark W. Skinner,&nbsp;Paula James,&nbsp;the coreVWD Panel","doi":"10.1111/hae.15122","DOIUrl":"10.1111/hae.15122","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Treatment options are expanding for von Willebrand disease (VWD). A core outcome set (COS)—a minimum set of agreed-upon outcomes to be used in every clinical trial for a given condition—provides guidance on which outcomes are most important to measure to ensure necessary data is collected for a variety of stakeholders and enable comparison across products and trials.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>coreVWD aimed to develop a COS for trials for prophylaxis and perioperative treatments for VWD.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A modified Delphi consensus process was used to condense/prioritize a long list of potential outcomes. Over three Delphi rounds, a multi-stakeholder panel (patients, clinicians, pharmaceutical company representatives, HTA organizations, payer, and government organization representatives) rated each outcome from 1 (not important to include in a COS) to 9 (essential to include). Outcomes were eliminated or retained based on pre-determined criteria; a special provision to elevate patient priorities was included. An in-person consensus meeting was held after Delphi round 2.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Thirty-nine panellists participated. The final COS for prophylaxis treatment included 18 outcomes, seven of which are part of a special subset selected for women, girls and people with the potential to menstruate. There were 11 outcomes in the final perioperative branch COS. Six outcomes overlapped both COS.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>The coreVWD COS represents a consensus list of outcomes for clinical trials for both factor and non-factor VWD therapies. These outcomes will be useful across the lifecycle of a product, from clinical development through regulatory and market access phases and into patient-provider decision-making.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"30 6","pages":"1357-1365"},"PeriodicalIF":3.0,"publicationDate":"2024-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.15122","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142739449","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":"UKHCDO gene therapy taskforce: Guidance for implementation of haemophilia gene therapy into routine clinical practice for adults","authors":"Pratima Chowdary,&nbsp;Beatriz Duran,&nbsp;Paul Batty,&nbsp;Gillian Lowe,&nbsp;April Jones,&nbsp;Debra Pollard,&nbsp;Sara Boyce,&nbsp;Jayashree Motwani,&nbsp;Bahareh Amirloo,&nbsp;Kathryn Musgrave,&nbsp;David Hopper,&nbsp;Stephen Classey,&nbsp;Sarah Whitaker,&nbsp;Nicola Dunn,&nbsp;Annette Bowyer,&nbsp;Susan Shapiro","doi":"10.1111/hae.15125","DOIUrl":"10.1111/hae.15125","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>2022 was a landmark year with two adeno-associated viral vectors (AAVs) receiving conditional marketing authorization from EMA for the treatment of persons with severe haemophilia A and severe to moderately severe haemophilia B and a third in 2024. Gene therapy is a transformative, irreversible treatment with long-lasting effects, necessitating development of new clinical pathways to ensure optimal outcomes.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>To develop a consensus framework and service specification for delivery of AAV gene therapy for haemophilia in adults within the UK using the hub-and-spoke model proposed by the European Association of Haemophilia and Allied Disorders and the European Haemophilia Consortium.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The UK Haemophilia Centre Doctors Organisation (UKHCDO) set up a working party to develop expert consensus guidance, working with NHS England to ensure alignment with NHS England commissioning and the national service specification.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>These guidelines detail the patient pathway, counselling and governance requirements for the hub-and-spoke model. The national service specification requires the hub site to manage governance for AAV-based gene therapy. Proposed regional and national multidisciplinary teams will harmonize clinical practices incorporating expertise from various specialities and professional groups. Key requirements identified include standardized documentation and multidisciplinary collaboration. Nationally agreed patient information and counselling checklists will streamline the informed consent process and facilitate data collection for long-term safety and efficacy monitoring.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>These guidelines provide a structured framework for the delivery of liver-directed gene therapy. Whilst specific to the United Kingdom they provide a framework for the implementation of gene therapy in other countries for haemophilia and other monogenic disorders.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 1","pages":"26-38"},"PeriodicalIF":3.0,"publicationDate":"2024-11-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11780224/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142675037","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":"Assessment of haemophilic joints in a low-resourced area using clinical tools: The effect of different types of prophylaxis","authors":"Ilham Youssry,&nbsp;Rania El-Taweel,&nbsp;Hadeel Seif,&nbsp;Rania Sami,&nbsp;Karim Okasha,&nbsp;Ebtehal Taha,&nbsp;Assad Haffar,&nbsp;Marwa Abd Elhady","doi":"10.1111/hae.15115","DOIUrl":"10.1111/hae.15115","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Haemophilic arthropathy (HA) is the most frequent complication in people with haemophilia (PWH). MRI is the gold standard to assess HA, however, there are limitations to its use in low-resourced areas.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p><b>Primary</b>; to compare clinical-functional, laboratory, and ultrasonographic joint scores with MRI scores to determine a reasonable alternative to MRI. <b>Secondary</b>; to identify the effect of various replacement therapies on the degree of joint involvement.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Material and method</h3>\u0000 \u0000 <p>Fifty PWH with at least one affected joint, with or without inhibitors, and receiving either on-demand treatment or secondary prophylaxis, were included. All participants had a joint assessment by clinical HJHS 2.1, functional FISH, HEAD-US, and MRI DENVER scores. Also, serum COMP level was assessed by ELISA for the PWH and 50 healthy subjects as control.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The <b><span>HJHS</span> <span>2</span><span>.</span><span>1</span></b> scores had a significant positive correlation with HEAD-US and the MRI DENVER scores. The <b><span>FISH score</span></b> had a significant negative correlation with HJHS 2.1, HEAD-US, and MRI DENVER Scores. The <b><span>serum COMP</span></b> level was comparable between the PWH and the controls. The HEAD-<b><span>US score</span></b> had a significant positive correlation with the MRI score. All of the joints’ scores for the PWH on Emicizumab prophylaxis showed significantly lower HJHS 2.1 and MRI DENVER scores but higher FISH score than the joint scores of the patients receiving other types of prophylaxis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The clinical-functional joints assessment scores (HJHS 2.1, FISH) were objective tools that correlated significantly with the HEAD-US and the Denver MRI scores. Emicizumab prophylaxis led to better joint status.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"30 6","pages":"1414-1421"},"PeriodicalIF":3.0,"publicationDate":"2024-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142647516","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":"Challenges in the diagnosis and management of patients with rare coagulation disorders in Lebanon and consequences of a social and economic crisis","authors":"Roula Farah,&nbsp;Michel Boustany,&nbsp;Philippe Saad,&nbsp;Alessandro Casini,&nbsp;Philippe de Moerloose","doi":"10.1111/hae.15123","DOIUrl":"10.1111/hae.15123","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Rare coagulation disorders (RCDs) constitute an important health risk. Data on epidemiology, quality of life (QoL), access to care, and impact of the ongoing economic crisis on RCDs in Lebanon is limited.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>We aimed to address these gaps by evaluating effect of the crisis on the management of RCDs.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We performed a retrospective chart review of RCD pediatric patients in a tertiary hospital between 2003 and 2023. Patients with deficiencies of fibrinogen, factor (F)II, FV, combined FV and FVIII, FVII, FXI, FXII, FXIII, and congenital deficiency of vitamin K-dependent factors (VKCFDs) underwent a qualitative assessment of the impact of the economic crisis on care and quality of life by an interview aimed at investigating obstacles to diagnosis, disparities in access to treatment, impact of the crisis on QoL and disease management, and opinion on governmental efforts to solve the health crisis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>46 patients were included. The response rate for the interview was 63%. Among the cohort, 21 (72.4%) reported difficulty accessing treatment since the start of the crisis and 18 (62%) reported “lack of healthcare coverage for necessary treatments” as the main issue. Most participants reported that the Lebanese government did not adequately address their needs during the crisis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Our study showcased that management of RCD patients in Lebanon has been severely affected by the economic crisis. Combined efforts by public and private sectors are needed to appropriately address this issue. Lessons can be learned from the Lebanese experience to appropriately screen for actionable factors in vulnerable populations.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"31 1","pages":"63-68"},"PeriodicalIF":3.0,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638661","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":"One day at a time: Life with Glanzmann thrombasthenia – Qualitative results from the GT 360 study","authors":"Kate Khair,&nbsp;Simon Fletcher,&nbsp;Kathryn Jenner,&nbsp;Michael Holland","doi":"10.1111/hae.15126","DOIUrl":"10.1111/hae.15126","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Glanzmann thrombasthenia (GT) is a platelet function disorder. Symptoms include bruising and bleeding, which may be severe and life-threatening. The day-to-day experiences of those affected remain poorly documented.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aims</h3>\u0000 \u0000 <p>The Glanzmann's 360 study aimed to better understand the daily realities of living with GT, exploring psychological, social and medical challenges to identify unmet need.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>People with GT over 18 and caregivers of children with GT participated. Mixed methods research was used, including a survey and in-depth interviews. The interviews, reported here, were audio-recorded, transcribed and thematically analysed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Nineteen interviews were conducted; 14 with people with GT and five caregivers. Mean age of those with GT was 32.9 years (range 2–78 years). Eight themes emerged: Diagnosis, Bleeding and Treatment, Menstruation, Fatigue, Identity, Abuse, Pregnancy, and The Future. Three participants described misdiagnosis. Caregivers reported early recognition and prompt diagnosis of their children. Bruising, the most commonly reported symptom, led to assumption of abuse. Six (32%) participants reported joint bleeds. External bleeding was normalised. Access to home treatment and local care were limited. Fatigue caused by anaemia was described. All women reported heavy menstrual bleeding. Choices around pregnancy were limited by ‘medical pessimism’. Nevertheless, participants remained hopeful for the development of improved treatment options.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>People with GT face significant and often overlooked challenges. There is need for improved medical and psychological treatment and support, increased awareness of these needs amongst healthcare professionals, and research into developing comprehensive care models that address medical and psychosocial needs of people with GT.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"30 6","pages":"1373-1382"},"PeriodicalIF":3.0,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.15126","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638664","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":"Evaluating the impact of the self-BAT screening tool on patient outcomes: Results of the let's talk period project","authors":"Laura McDonald,&nbsp;Julie Grabell,&nbsp;Jennifer Leung,&nbsp;Wilma Hopman,&nbsp;Paula James","doi":"10.1111/hae.15113","DOIUrl":"10.1111/hae.15113","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>One of the many challenges in diagnosing bleeding disorders is distinguishing between normal and abnormal bleeding symptoms. Letstalkperiod.ca is an educational website that includes an online self-administered bleeding assessment tool (Self-BAT) which is a validated screening tool that enables patients to independently determine their bleeding scores (BS).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>The aim of this study was to evaluate patient outcomes for those referred with an abnormal Self-BAT BS compared to those referred without the prior use of the Self-BAT.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This was a retrospective, observational study. After obtaining REB approval, chart review was performed for patients evaluated for a suspected bleeding disorder in a tertiary care centre between 2016 and 2023.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>351 patients (310 female) were identified for inclusion with a mean age of 41 years. Of these patients, 30 were referred for a positive/abnormal Self-BAT BS and the remainder were referred for other reasons. Patients referred for a positive Self-BAT BS required interventions for their bleeding symptoms more often (73.3% vs. 36.7%, <i>p</i> ≤ .001). Though they were not diagnosed with an <i>inherited</i> bleeding disorder more often (6.7% vs. 10.7%, <i>p</i> = .754), patients referred for a positive self-BAT were more likely to be diagnosed with a bleeding disorder when the definition was expanded to include bleeding disorder of unknown cause (56.7% vs. 31.9%, <i>p</i> = .008).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Results of this study suggest that the Self-BAT at letstalkperiod.ca can be a useful tool for patients and physicians to identify those needing referral to tertiary haematology clinics for evaluation and management of bleeding symptoms.</p>\u0000 </section>\u0000 </div>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":"30 6","pages":"1400-1405"},"PeriodicalIF":3.0,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.15113","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638662","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":"Unexpected pulmonary embolism in a giant haemophilia pelvic pseudotumor resection surgery: A case report and literature review.","authors":"Zhu Wei, Ren Miao, Feng Bin, Weng Xisheng","doi":"10.1111/hae.15118","DOIUrl":"https://doi.org/10.1111/hae.15118","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2024-11-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142618716","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":"Knee arthrodesis in treatment of haemophilic arthropathy of the knee.","authors":"Arman Vahabi, Elcil Kaya Biçer, Fahri Şahin, Kaan Kavaklı, Semih Aydoğdu","doi":"10.1111/hae.15117","DOIUrl":"https://doi.org/10.1111/hae.15117","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2024-11-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142618714","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":"The impact of emicizumab on the clinical validation of new therapies for haemophilia A.","authors":"Cedric Hermans, Catherine Lambert, Sébastien Lobet, Evelien Krumb, An Van Damme","doi":"10.1111/hae.15112","DOIUrl":"https://doi.org/10.1111/hae.15112","url":null,"abstract":"","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2024-11-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142604376","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}