Haemophilia

Papers
(The TQCC of Haemophilia is 5. The table below lists those papers that are above that threshold based on CrossRef citation counts [max. 250 papers]. The publications cover those that have been published in the past four years, i.e., from 2021-06-01 to 2025-06-01.)
ArticleCitations
The detrimental effects of iron to the joint; aggravation by inflammation84
AHP Oral Abstracts52
51
CCS_Book of abstracts42
Recommendations for Psychosocial Support during Gene Therapy: Results From an EAHAD Interdisciplinary Roundtable39
Natural anticoagulants: A missing link in mild to moderate bleeding tendencies32
Into a brave new world: Haemophilia A & von Willebrand Disease Surgery with novel therapies32
Intracranial haemorrhage among a population of haemophilic patients in Nepal: Challenges and future direction30
Women and girls with haemophilia receiving care at specialized haemophilia treatment centres in the United States26
A systematic review of adeno‐associated virus gene therapy clinical trials for HIV – A potential solution for patients with haemophilia and HIV?25
The importance and evolution of bleeding disorder registries25
Haemophilia B, severe childhood obesity and other extra‐haematological features associated with similar 4Mb‐deletions on Xq27: Clinical findings, molecular insights and literature update25
The effects of emicizumab on in vitro coagulation and fibrinolysis parameters in patients with disseminated intravascular coagulation with and without addition of anti‐FVIII antibody24
Direct transfer of data of people with haemophilia from the Thai Haemophilia Treatment Centre Registry to the World Bleeding Disorders Registry of the World Federation of Hemophilia23
Descriptive analysis of bleeding symptoms in haemophilia carriers enrolled in the ATHNdataset23
Demographics, clinical profile and treatment landscape of patients with haemophilia B in China23
Targeting higher factor VIII levels for prophylaxis in haemophilia A: a narrative review23
Total joint arthroplasty in the patients with haemophilia: General or neuraxial anaesthesia?22
The legacy of haemophilia: Memories and reflections from three survivors21
Is it time for patient involvement in Haemophilia?21
Health‐related quality of life among adults with haemophilia in China: A comparison with age‐matched general population20
Haemophilia management and treatment: An Italian survey on patients’, caregivers’ and clinicians’ point of view20
How to assess, detect, and manage joint involvement in the era of transformational therapies: Role of point‐of‐care ultrasound20
Issue Information19
The long‐term clinical benefits and economic costs associated with increased use of prophylaxis among patients with haemophilia A in China: Population‐based predictions from 2018 to 203319
Determining the approximate factor VIII level of patients with severe haemophilia A on emicizumab using in vivo global haemostasis assays19
Effectiveness of therapeutic exercise and cognitive‐behavioural therapy combined protocol on functionality, pain and joint health in people with haemophilia: Secondary analysis of a controlled trial19
Comparison of the kinematic analysis of indoor and outdoor gait in people with haemophilia and total knee replacement18
PUBLICATION ONLY ABSTRACTS18
Health technology assessment for gene therapies in haemophilia17
Managing invasive procedures in haemophilia patients with limited resources, extended half‐life concentrates or non‐replacement therapies in 202217
Profiling of Anti‐FVIII Antibodies in Acquired Haemophilia A: ‘Insights into Domain Specificity, Isotype Variability, and Clinical Correlations’16
Real world management of individuals with severe FXI deficiency and its impact on clinical outcomes: Experience from a haemophilia treatment centre16
Eptacog beta for the management of patients with haemophilia A and B with inhibitors: A European perspective16
Low‐dose emicizumab for more equitable access to prophylaxis in resource limited countries16
The UK Infected Blood Inquiry: A Personal Reflection16
Test–retest reliability of a mobile application of the patient reported outcomes burdens and experiences (PROBE) study16
Initial von Willebrand factor antigen values in adolescent females predict future values15
Knowledge gaps in health‐related quality of life research performed in children with bleeding disorders – A scoping review15
Reply to: Gene therapy to cure haemophilia: Is robust scientific inquiry the missing factor?15
Accreditation model of European Haemophilia Centres in the era of novel treatments and gene therapy15
Efficacy of emicizumab in von Willebrand disease (VWD) patients with and without alloantibodies to von Willebrand factor (VWF): Report of two cases and review of literature14
Towards achieving a haemophilia‐free mind14
Emicizumab for the treatment of acquired hemophilia A: Retrospective review of a single‐institution experience14
Wearable activity trackers in young people with haemophilia: What needs to be considered?14
Development of the World Federation of Hemophilia Shared Decision‐Making Tool14
Emicizumab prophylaxis in haemophilia A with inhibitors: Three years follow‐up from the UK Haemophilia Centre Doctors’ Organisation (UKHCDO)14
Reinvestigation of unidentified causative variants in FXI‐deficient patients: Focus on gene segment deletions14
Low‐dose immune tolerance induction therapy in children of Arab descent with severe haemophilia A, high inhibitor titres and poor prognostic factors for immune tolerance induction treatment success14
14
Plasma‐derived human factor X concentrate for the treatment of patients with hereditary factor X deficiency14
Clinical outcomes of low‐dose pharmacokinetic‐guided extended half‐life versus low‐dose standard half‐life factor VIII concentrate prophylaxis in haemophilia A patients14
Quantitative measurements of haemophilic joint tissues by point‐of‐care musculoskeletal ultrasound: Associations with clinical and functional joint outcome parameters14
Haemophilic arthropathy: Contemporary management challenges and a future scenario13
Quality of life of siblings of adolescents with severe haemophilia (FRATHEMO): An ancillary study to the TRANSHEMO project13
Influence of blood group and von Willebrand factor on population pharmacokinetics and dose individualization of recombinant factor VIII in Taiwanese patients with haemophilia A13
Occurrence of familial Mediterranean fever in haemophilia patients13
A survey on practice of circumcision in children with severe haemophilia in Eastern Mediterranean Region13
Impact of first COVID‐19 lockdown on paediatric and adult haemophilia patients treated in a French Haemophilia Comprehensive Care Centre13
AUTHOR INDEX13
Complete characterisation of two new large Xq28 duplications involving F8 using whole genome sequencing in patients without haemophilia A13
Anti‐platelet treatment challenges in Glanzmann thrombasthenia‐clinical practice when data lacks13
AUTHOR INDEX12
Assessment of rare bleeding disorders in adolescents with heavy menstrual bleeding12
Emicizumab and asparaginase, A first experience to share12
A 10‐year single‐institution retrospective review of treatment for acquired hemophilia A12
von Willebrand disease and von Willebrand factor12
IDEAL study: A real‐world assessment of pattern of use and clinical outcomes with recombinant coagulation factor IX albumin fusion protein (rIX‐FP) in patients with haemophilia B in Italy12
Efficacy and safety of recombinant activated factor VII in Glanzmann thrombasthenia: A systematic literature review12
Acquired haemophilia A in Finland: A nationwide study of incidence, treatment and outcomes12
Tandem and inverted duplications in haemophilia A: Breakpoint characterisation provides insight into possible rearrangement mechanisms12
A phase 1/2 safety and efficacy study of TAK‐754 gene therapy: The challenge of achieving durable factor VIII expression in haemophilia A clinical trials12
Inhibitor Eradication in Postpartum Acquired Haemophilia A: Real‐Life Case Series and Literature Review12
Factor XIII levels correlate with fibrinogen concentrations in patients with venous malformations12
Building the blueprint: Formulating a community‐generated national plan for future research in inherited bleeding disorders12
Development of a target product profile (TPP) for haemophilia point‐of‐care (POC) diagnostic devices for low‐resource countries and remote settings12
The lived experience of mothers of children with haemophilia in Jordan: A phenomenological study12
Pharmacokinetic profile of children with haemophilia A receiving low‐dose FVIII prophylaxis in Indonesia: A single centre experience12
Factors associated with physical activity in young people with haemophilia A on prophylaxis12
11
Estimated prophylactic dose required to achieve 3% trough as a function of age and concentrate class in multi‐country severe WAPPS‐Hemo haemophilia patients11
The state of oral health in patients with haemophilia in the Netherlands11
Efficacy, safety and bioequivalence of the human‐derived B‐domain‐deleted recombinant factor VIII TQG202 for prophylaxis in severe haemophilia A patients11
Alleviated bleeding phenotypes in a child with severe haemophilia A and thalassemia disease11
Diagnostic utility of bleeding assessment tools in congenital fibrinogen deficiencies11
Value contribution of etranacogene dezaparvovec gene therapy in moderately severe and severe haemophilia B through multi‐criteria decision analysis11
Systematic Literature Review of Outcomes Associated With Adherence to Haemophilia Drug Therapy11
Patient‐reported outcomes in autosomal inherited bleeding disorders: A systematic literature review11
Physical Activity Awareness and Understanding of Treatment Protection Among People With Haemophilia and Their Caregivers in Central Europe11
Determining causes of death among individuals with haemophilia in Afghanistan10
Adjunctive daratumumab can induce rapid remission in acquired haemophilia A with poor prognostic markers10
Converting factor and nonfactor usage into a single metric to facilitate benchmarking the resources consumed for haemophilia care across jurisdictions and over time10
Safety, pharmacokinetics and efficacy of a subcutaneous recombinant FVIII (OCTA101) in adult patients with severe haemophilia A10
Management of Persons With Haemophilia Requiring Antithrombotic Therapy: A Canadian Modified Delphi Consensus Study10
Performance on the iSTEP and 10 m‐ISWT in boys with haemophilia10
Book of Abstracts10
Ankle joint distraction is a promising alternative treatment for patients with severe haemophilic ankle arthropathy10
Psychometrics and applications of a novel self‐report measure of emicizumab adherence: VERITASNexGen10
Hybrid human‐porcine factor VIII proteins partially escape the inhibitory effects of anti‐factor VIII inhibitor alloantibodies having A2 or C2 domain specificity10
Transition readiness among adolescents and young adults with haemophilia in the Netherlands: Nationwide questionnaire study10
Haemophilia Carriers Experience Study (CARES): A mixed method exploration into the experience of women who are carriers of Haemophilia10
Transitioning patients with severe haemophilia A from emicizumab prophylaxis to valoctocogene roxaparvovec gene therapy: Real‐world clinical experience10
The haemophilia joint health score for the assessment of joint health in patients with haemophilia10
Haemophilia diagnostics with modern genomics9
Democratisation of physiotherapy for people with haemophilia9
Author Index9
Sexual issues in people with haemophilia: Awareness and strategies for overcoming communication barriers9
HRQoL and psychosocial aspects of burden on caregivers to children with moderate or severe von Willebrand disease9
Patient‐centred research agenda for females with bleeding disorders9
Revised terminal half‐life of nonacog alfa as derived from extended sampling data: A real‐world study involving 64 haemophilia B patients on nonacog alfa regular prophylaxis9
Real‐World Data on Patients With Acquired Haemophilia A in Japan Undergoing Rehabilitation or With Low Activities of Daily Living Scores: The ORIHIME II Study9
Clotting factor activity levels and bleeding risk in people with haemophilia playing sports9
Clinical characteristics and prognostic factors of acquired haemophilia A in Korea9
A case of acquired haemophilia A in a 70‐year‐old post COVID‐19 vaccine9
Immune checkpoint inhibitor‐induced acquired haemophilia: A pharmacovigilance analysis of the FDA adverse event reporting system9
The role of the specialist nurse in comprehensive care for bleeding disorders in Europe: An integrative review9
Molecular study of a large cohort of 109 haemophilia patients from Cuba using a gene panel with next generation sequencing‐based technology9
Retraction9
Prophylaxis use of clotting factor replacement products in people with non‐severe haemophilia: A review of the literature9
Single centre experience on Acquired Haemophilia A patients: Diagnosis, clinical management and analysis of factors predictive of response and outcome9
The relationship between chronic pain and psychosocial aspects in patients with haemophilic arthropathy. A cross‐sectional study9
Does the method of telehealth delivery affect the physiotherapy management of adults with bleeding disorders? A comparison of audioconferencing and videoconferencing9
Validity and reliability test of the Indonesian version of the PedHALshort questionnaire9
 8
European principles of care for physiotherapy provision for persons with inherited bleeding disorders: Perspectives of physiotherapists and patients8
POSTER PRESENTATIONS8
Harmonizing patient‐reported outcome measurements in inherited bleeding disorders with PROMIS8
Identifying performance‐based outcome measures of physical function in people with haemophilia (IPOP)8
8
Endothelial specific isoform of type XVIII collagen (COL‐18N): A marker of vascular integrity in haemophilic arthropathy8
Comparison of free‐living physical activity measurements between ActiGraph GT3X‐BT and Fitbit Charge 3 in young people with haemophilia8
Electronic diaries in the management of haemophilia gene therapy: Perspective of an expert group from the German, Austrian and Swiss Society on Thrombosis and Haemostasis (GTH)8
Is the world ready for gene therapy?8
Comprehensive Analysis of Surgical Interventions for Musculoskeletal Disorders in Patients With Bleeding Disorders: Report From a Subspecialty Unit in a Developing Country8
Occurrence rates of inherited bleeding disorders other than haemophilia and von Willebrand disease among people receiving care in specialized treatment centres in the United States8
Diagnosis and management of factor XI alloinhibitors in patients with congenital factor XI deficiency—A large single‐centre experience8
Breaking barriers in haemophilia A care: One‐year real‐world experience with emicizumab prophylaxis at Civil Service Hospital, Kathmandu, Nepal8
COMMITTEE LIST8
Haemostatic effect of adding tranexamic acid to emicizumab prophylaxis in severe haemophilia A: A preclinical study8
Does difference between label and actual potency of factor VIII concentrate affect pharmacokinetic‐guided dosing of replacement therapy in haemophilia A?8
Real‐world experience of rIX‐FP prophylaxis at dosing intervals of 14 days or more in adult patients with haemophilia B in Italy – Results from IDEAL Part B8
Gene therapy: Practical aspects of implementation8
European principles of care for women and girls with inherited bleeding disorders8
Activity and participation in haemophiliacs: Item response modelling based on international classification of functioning, disability and health8
Clinical, biological, and genetic features in an afibrinogenemia patient series in Algeria7
Efficacy, safety and cost of emicizumab prophylaxis in haemophilia A patients with inhibitors: A nationwide observational study in Taiwan7
Hemizygous FGG p.Ala108Gly in a hypofibrinogenemic patient with a heterozygous 14.8 Mb deletion encompassing the entire fibrinogen gene cluster7
Experiences and Needs Regarding Information Provision in Children With Haemophilia: A Qualitative Study on Caregivers’ and Healthcare Providers’ Perspectives7
Issue Information7
Clinical and Economic Burden of Patients with Haemophilia A and B in France: Analysis of a Nationwide Claims Database7
7
Monitoring of von Willebrand factor inhibitors in patients with type 3 von Willebrand disease using a quantitative assay7
Bone marrow oedema syndrome as a cause of chronic knee pain in a person with severe Haemophilia A7
Comparison of one‐stage and chromogenic factor VIII assays to tailor the dose of recombinant factor VIII‐Fc fusion protein (rFVIIIFc, efmoroctocog alfa) in adult patients with haemophilia A: Single‐ce7
Treatment of haematuria with factor concentrate in people with haemophilia is associated with low rates of complications7
Five new F10 variants in hereditary factor x deficiency detected by high‐throughput sequencing7
7
Modulation of Haemostatic Balance in Combined von Willebrand Disease and Antithrombin Deficiency: A Comprehensive Family Study7
Sexual functioning in men with haemophilia: Data from the haemophilia in the Netherlands‐6 study7
Issue Information7
Clinical conditions and risk factors for inhibitor‐development in patients with haemophilia: A decade‐long prospective cohort study in Japan, J‐HIS2 (Japan Hemophilia Inhibitor Study 2)7
Indirect treatment comparisons of the gene therapy etranacogene dezaparvovec versus extended half‐life factor IX therapies for severe or moderately severe haemophilia B7
Mutational landscape, inhibitor development, and health‐care burden in non‐severe haemophilia A: A single‐centre Australian experience7
Accuracy and clinical role of digital templating for total knee arthroplasty performed on haemophilic knees7
Haemophilia care in Asia: Learning from clinical practice in some Asian countries7
Abstracts7
Committee List7
Preoperative bleeding risk assessment with ISTH‐BAT and laboratory tests in patients undergoing elective surgery: A prospective cohort study7
Psychophysical assessment of pain in adults with moderate and severe haemophilia: A cross‐sectional study6
Diagnosis of rare bleeding disorders6
Bridging the gap: Survey highlights challenges and solutions in outreach and identification of people with inherited bleeding disorders6
Efficacy and Safety of Aspiration and Intra‐Articular Injection of Tranexamic Acid in Acute Knee Hemarthrosis of Adult Haemophilic Patients: A Randomized Clinical Trial Study6
Diagnosis and treatment of von Willebrand disease in 2024 and beyond6
Bleeding patterns in patients before and after diagnosis of von Willebrand disease: Analysis of a US medical claims database6
Methylome analysis for haemophilia A intron 22 inversion patients with and without inhibitors: A pilot study6
Emicizumab assays evaluations with four different reagents in severe haemophilia A patients: Concentration from baseline to maintenance therapy6
Deciphering a novel complex inversion affecting F8 in a family with severe haemophilia A by optical genome mapping6
Dental habits and oral health in children and adolescents with bleeding disorders: A single‐institution cross‐sectional study6
Low immunogenicity of emicizumab in persons with haemophilia A6
A distinct common p.Gln317* mutation among causative LMAN1 genetic mutations of combined factor V and factor VIII deficiency in five Taiwanese families6
Research Letter: Patient Attitudes Towards Haemophilia Gene Therapy at a US Haemophilia Treatment Center6
6
Acquired haemophilia A: A 15‐year population‐based review of incidence rate, patient demographics and treatment outcomes6
Pain while injecting emicizumab predominant in children, a report of Dutch patient experiences6
Clinical features and genetic defect in six index patients with congenital fibrinogen disorders: Three novel mutations with one common mutation in Taiwan's population6
A systematic review and narrative synthesis of footwear and orthotic devices used in the management of ankle haemarthrosis and haemarthropathy in haemophilia6
A national assessment of standards of care for inherited bleeding disorders in Canada6
POSTER ABSTRACTS6
Blood Group O Does Not Increase the Risk of Inhibitors in Severe Haemophilia A: Data from the PedNet Study Group6
Low vitamin C status and hypermobility‐related disorders in patients with bleeding disorder of unknown cause6
Molecular spectrum of inherited FVII deficiency in North India revealed a recurrent variant with a founder effect5
EAHAD statement on the ISTH clinical practice guideline for treatment of congenital haemophilia A and B5
The effect of carriers’ reproductive choices and pregnancy history on sporadic severe haemophilia: A 20‐year retrospective study through a regional registry5
Issue Information5
Comprehensive laboratory assessment of lonoctocog alfa versus octocog alfa in severe haemophilia A5
Effect of emicizumab prophylaxis on bone and joint health markers in people with haemophilia A without factor VIII inhibitors in the HAVEN 3 study5
Interferences by factor VIII and lupus anticoagulant in the modified one‐stage assay for emicizumab5
What can we expect for adolescents and adults with haemophilia switched to low‐dose prophylaxis from episodic treatment for over 3 years? A real‐world snapshot in China5
Nephrotic syndrome in two haemophilia B children with inhibitor under low‐dose immune tolerance induction combined with rituximab‐based immunosuppressant protocol5
Women and girls with inherited bleeding disorders: Focus on haemophilia carriers and heavy menstrual bleeding5
Real‐world effectiveness of eptacog beta in patients with haemophilia and inhibitors: A multi‐institutional case series5
Invited Medical Presentation Abstracts5
Depression and anxiety in persons with Von Willebrand disease5
Real‐world data for the use of emicizumab in haemophilia A patients with inhibitors – First nationwide report from Korea5
The effect of a virtual child disease management programme on burden and social adjustment of caregivers of children with coagulation factor deficiencies5
Patient preferences for gene therapy in haemophilia: Results from the PAVING threshold technique survey5
Vaccination in children with inherited bleeding disorders: Does the use of plasma an d factor affect the response to the vaccine: An institutional registry5
UK haemophilia consultant access to foot and ankle services and concurrent patient impact questionnaire responses to foot and ankle interventions5
Race and ethnicity and the success of immune tolerance induction among people with severe haemophilia A in the United States5
Bleeding control improves after switching to emicizumab: Real‐world experience of 177 children in the PedNet registry5
Defining success in haemophilia care – Are we doing it right?5
Emicizumab dose up‐titration in case of suboptimal bleeding control in people with haemophilia A5
Effects of a supervised therapeutic exercise program on musculoskeletal health and gait in patients with haemophilia: A pilot study5
Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE‐B trial 2 years after gene therapy5
Prophylaxis in children with haemophilia in an evolving treatment landscape5
Women and girls’ participation in haemophilia clinical trials5
Acquired bleeding disorders5
Real‐world evidence on efmoroctocog alfa in patients with haemophilia A: A systematic literature review of treatment experience in Europe5
Psychological and cognitive factors involved in decision‐making process of haemophilia carriers in reproductive choices5
Differences and similarities in patient‐reported outcomes among men and women with haemophilia5
Management and outcomes of paediatric patients on emicizumab prophylaxis undergoing surgical procedures: Experience from a large haemophilia centre in the UK5
5
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 ≥ 15
Prophylaxis with emicizumab in children under 12 years old with haemophilia A without inhibitors in Uruguay: National experience5
Eradication of FIX inhibitor in haemophilia B children using low‐dose immune tolerance induction with rituximab‐based immunosuppressive agent(s) in China5
Origin of pathogenic variant and mosaicism in families with a sporadic case of haemophilia B5
Association of factor expression levels with annual bleeding rate in people with haemophilia B5
In vitro evaluation of global coagulation potentials in the co‐presence of plasma‐derived factors Viia/X products (Byclot®) and emicizumab in patients with haemophilia A and inhibitors and 5
Issue Information5
Von Willebrand Factor (VWF) multiplex activity assay differentiation of type 1 von Willebrand Disease (VWD) and variant VWD5
0.28649997711182