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Benefits and risks of non‐factor therapies: Redefining haemophilia treatment goals in the era of new technologies Haemophilia (IF 3.9) Pub Date : 2024-03-14 Maria Elisa Mancuso, Stacy E. Croteau, Robert Klamroth
IntroductionOver the last decades progress in haemophilia treatment has been remarkable and prophylaxis with clotting factor concentrates in haemophilia A and B has been established as the standard of care in individuals with haemophilia and a severe bleeding phenotype. Besides clotting factor products with prolonged half‐life non‐factor therapies were developed which enable prophylaxis via subcutaneous
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Diagnosis and treatment of von Willebrand disease in 2024 and beyond Haemophilia (IF 3.9) Pub Date : 2024-03-14 Paula James, Frank Leebeek, Caterina Casari, David Lillicrap
Manuscript Background and AimThe diagnosis and clinical care of patients with von Willebrand disease (VWD) has continued to evolve since the characterization of the von Willebrand factor (VWF) gene in 1985. This condition is almost certainly the most common inherited bleeding disorder, and the major symptomatic burden of the disease is experienced by females during their reproductive years. Diagnosis
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Benefits of physical activity self‐monitoring in patients with haemophilia: a prospective study with one‐year follow‐up Haemophilia (IF 3.9) Pub Date : 2024-03-12 Sofía Pérez‐Alenda, Juan J. Carrasco, Felipe Querol‐Fuentes, Juan Eduardo Megías‐Vericat, Felipe Querol‐Giner, María Luz Sánchez‐Sánchez, Santiago Bonanad, Rodrigo Núñez‐Cortés
IntroductionActivity wristbands have been shown to be effective in relation to self‐monitoring activity levels and increasing exercise adherence. However, previous reports have been based on short‐term follow‐ups in people with haemophilia (PWH).Aim(1) To evaluate compliance with physical activity (PA) recommendations in PWH during a 1‐year follow‐up period using activity wristbands to record daily
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Diagnosis and treatment challenges in lower resource countries: State‐of‐the‐art Haemophilia (IF 3.9) Pub Date : 2024-03-11 Johnny Mahlangu, Saliou Diop, Michelle Lavin
The 2022 World Federation of Haemophilia Annual Global Survey (AGS) reports that 454,690 patients with inherited bleeding disorders (IBD) have been identified globally. While this represents noteworthy progress, haemophilia epidemiology data indicate that 75% of people with inherited bleeding disorders living in low‐income and low‐to‐middle‐income countries have yet to be diagnosed. The AGS also revealed
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EAHAD haemophilia gene therapy clinical outcome database (EAHAD‐GTD) Haemophilia (IF 3.9) Pub Date : 2024-03-11 Wolfgang Miesbach, Ana Boban, Pratima Chowdary, Michiel Coppens, Miguel Crato, Victor Jimenez‐Yuste, Robert Klamroth, Mike Makris, Greta Mulders, Flora Peyvandi
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Using evidence‐based co‐design to develop a hybrid delivered exercise intervention that aims to increase confidence to exercise in people with haemophilia Haemophilia (IF 3.9) Pub Date : 2024-03-11 Stephanie Taylor, Karen Barker, David Stephensen, Esther Williamson
IntroductionDue to advances in treatments, people with haemophilia (PWH) are living longer. They are not as active as the general population due to joint damage and lack confidence to be active due to concerns about further bleeds and pain. There is a need to facilitate healthy aging through promotion of physical activity (PA) and exercise. Changing patient beliefs and increasing physical literacy
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Race and ethnicity and the success of immune tolerance induction among people with severe haemophilia A in the United States Haemophilia (IF 3.9) Pub Date : 2024-03-11 Stacey A. Fedewa, Christine L. Kempton
IntroductionImmune tolerance induction (ITI) is the only treatment to eradicate inhibitors in people with severe haemophilia A with inhibitors. Since the risk of inhibitor development is greater among Black and Hispanic persons, it has been hypothesized that race and ethnicity may influence ITI success. Limited studies have evaluated this hypothesis.AimTo examine the success of ITI according to race
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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 therapy Haemophilia (IF 3.9) Pub Date : 2024-03-11 Robbin Itzler, Tyler W. Buckner, Frank W. G. Leebeek, Joel Miller, Michael Recht, Douglass Drelich, Paul E. Monahan, Steven W. Pipe
IntroductionFor people with haemophilia B (PwHB), bleeding may occur despite prophylaxis, negatively affecting health‐related quality of life (HRQoL). The pivotal phase 3 HOPE‐B trial investigating the adeno‐associated virus gene transfer product, etranacogene dezaparvovec (EDZ), demonstrated sustained factor IX (FIX) activity and bleed protection in PwHB with baseline FIX levels ≤2%.AimAssess how
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Genetic variant detection in a South African haemophilia B population Haemophilia (IF 3.9) Pub Date : 2024-03-11 Chené Bester, Jean F. Kloppers, Walter J. Janse van Rensburg
BackgroundHaemophilia B is characterised by a deficiency of factor IX (FIX) protein due to genetic variants in the FIX gene (F9). Genetic testing may have a vital role in effectively managing haemophilia B. However, in many developing countries, comprehensive genetic variant detection is unavailable. This study aimed to address the lack of genetic data in our country by conducting genetic variant detection
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The role of the specialist nurse in comprehensive care for bleeding disorders in Europe: An integrative review Haemophilia (IF 3.9) Pub Date : 2024-03-04 Greta Mulders, Nanda Uitslager, Mary Kavanagh, Maj Friberg Birkedal, Gabriella Nicolo, Renester Fenton, Linda Myrin Westesson
Managing bleeding disorders (BDs) is complex, requiring a comprehensive approach coordinated by a multidisciplinary team (MDT). Haemophilia nurses (HNs) play a central role in the MDT, frequently coordinating care. As novel treatments bring change to the treatment landscape, ongoing education and development is key. However, understanding of the roles and tasks of HNs is lacking.
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Correction to Batt K, Xing S, KuharicM, et al. Real‐world analysis of patients with haemophilia A and haemophilia A carriers in the United States: Demographics, clinical characteristics and costs. Haemophilia (IF 3.9) Pub Date : 2024-03-06
Batt K, Xing S, KuharicM, et al. Real-world analysis of patients with haemophilia A and haemophilia A carriers in the United States: Demographics, clinical characteristics and costs. Haemophilia. 2023; 29(3):809–818. The first row of Medicaid N numbers was published incorrectly. It should read as follows: (N = 549 | N = 64 | N = 67 | N = 44). We apologize for this error.
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Immune gene polymorphisms associated with poor response to platelet transfusion and recombinant factor VII administration in Glanzmann thrombasthenia Haemophilia (IF 3.9) Pub Date : 2024-03-05 Majid Naderi, Ilia Mirzaei, Omid Seidizadeh, Abolfazl Parsi Moud, Hosna Sarani, Amir Avan, Mohsen Taheri, Danial Jahantigh, Mohammad Reza Keramati, Tayebeh Sohrabi
IntroductionPoor response to platelet and recombinant factor VII administration is a major problem in patients with Glanzmann Thrombasthenia (GT). The risk factors associated with poor response to treatment in these patients are unknown. Some genetic variations of cytokines may contribute to therapy resistance.AimsWe evaluated, for the first time, whether genetic polymorphisms on cytokine genes are
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Test–retest reliability of a mobile application of the patient reported outcomes burdens and experiences (PROBE) study Haemophilia (IF 3.9) Pub Date : 2024-03-05 Randall Curtis, Joanne Wu, Alfonso Iorio, Neil Frick, Michael Nichol, Declan Noone, Brian O'Mahony, David Page, Jeffrey Stonebraker, Alexandra Kucher, Elizabeth Clearfield, Mark W. Skinner, Federico Germini
IntroductionThe Patient Reported Outcomes, Burdens, and Experiences (PROBE) questionnaire is a patient‐reported outcome tool that assesses quality of life and disease burden in people with haemophilia (PWH).AimTo assesses the test–retest reliability of PROBE when completed using the mobile phone application.MethodsWe recruited PWH, including carriers, and individuals with no bleeding disorders who
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Radiosynovectomy in haemophilic synovitis and arthropathy of the knee: A scoping review Haemophilia (IF 3.9) Pub Date : 2024-03-05 Aaron Ray, Emma Rowbotham
IntroductionRadiosynovectomy is an established treatment for chronic synovitis in patients with haemophilia. Although its role in rheumatological diseases has diminished, it remains an accepted therapy for haemophilic synovitis.AimThe aim of this scoping review was to map and summarise the evidence surrounding radiosynovectomy in haemophilic knees, identify gaps in the literature and inform future
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Emicizumab use in infancy: A survey of United States Pediatric Hemophilia Treatment Center Medical Directors Haemophilia (IF 3.9) Pub Date : 2024-02-29 Clay T. Cohen, Cristina Tarango, Jacquelyn M. Powers
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Low‐dose emicizumab for more equitable access to prophylaxis in resource limited countries Haemophilia (IF 3.9) Pub Date : 2024-02-28 Pier Mannuccio Mannucci, Cedric Hermans
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Pain attitudes and pain outcomes among people with bleeding disorders: Results from community voices in research Haemophilia (IF 3.9) Pub Date : 2024-02-28 Corey Pierce, Adam Branscum, Veronica L. Irvin, James Elander, Marit Bovbjerg, Michelle Witkop, Ellen Smit
IntroductionAmong people with bleeding disorders (PwBD), pain is a major problem and pain treatments are often ineffective. Understanding of psychological factors involved in pain processing is limited. Maladaptive pain attitudes are associated with worse pain outcomes and adaptive pain attitudes are associated with better outcomes in high pain conditions, but relationships between pain attitudes and
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Physical activity and factor VIII levels in patients with haemophilia: A real‐world prospective observational study Haemophilia (IF 3.9) Pub Date : 2024-02-22 Fabian Tomschi, Jamil Hmida, Steffen Herzig, Pia Ransmann, Marius Brühl, Alexander Schmidt, Marie Herzig, Georg Goldmann, Andreas C. Strauß, Johannes Oldenburg, Heinrich Richter, Thomas Hilberg
IntroductionRegular physical activity (PA) is recommended for patients with haemophilia (PwH). For PwH it is crucial to ensure a sufficient factor level to prevent PA‐induced bleedings. However, there is a gap in the literature dealing with specific factor levels, which are needed when performing specific types of PA.AimTo provide data on factor VIII (FVIII) levels at the start of PA performed by PwH
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The impact of obesity on chronic pain in patients with haemophilia: A multicentre retrospective analysis Haemophilia (IF 3.9) Pub Date : 2024-02-22 Keerthy Joseph, Adam Cuker, Skye Peltier, Mark T. Reding, Dunlei Cheng, Xiaoyan Han, Allyson M. Pishko
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Assessment of rare bleeding disorders in adolescents with heavy menstrual bleeding Haemophilia (IF 3.9) Pub Date : 2024-02-22 Ruchika Sharma, Victoria Johnson, Amy Pan, Austin Sellers, Marisol Betensky, Neil Goldenberg, Veronica H. Flood
There are a significant number of patients with mucocutaneous bleeding, specifically heavy menstrual bleeding (HMB), who do not have a diagnosed bleeding disorder. These patients receive nontargeted interventions and may have suboptimal treatments. Functional assays, particularly for fibrinolytic and rare platelet function defects, are not robust and not readily available.
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Safety and efficacy of combined dual antiplatelet therapy and factor VIII prophylaxis in patients with haemophilia A after acute coronary syndrome Haemophilia (IF 3.9) Pub Date : 2024-02-20 Pasquale Agosti, Simona Maria Siboni, Alessandro Ciavarella, Sara Arcudi, Federico Boggio, Roberta Gualtierotti, Flora Peyvandi
The increased life expectancy of patients with haemophilia A (HA) has led to a growing prevalence of cardiovascular risk factors and events. There is still scarce evidence on the safety and appropriate duration of dual antiplatelet therapy (DAPT) after acute coronary syndrome (ACS) in HA patients.
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Arthroscopic ankle surgery in people with haemophilia Haemophilia (IF 3.9) Pub Date : 2024-02-20 E. Carlos Rodriguez-Merchan, Hortensia De la Corte-Rodriguez, Primitivo Gomez-Cardero, Maria Teresa Alvarez-Roman, Victor Jimenez-Yuste
People with haemophilia (PWH) not administered primary haematological prophylaxis since childhood, that is, those treated haematologically on demand or not treated at all, often experience the degeneration of the ankles, leading to pain and functional impairment.
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Reliability and construct validity of the ACTIVLIM-Hemo and Haemophilia Activities List (HAL) questionnaires in individuals with haemophilia Haemophilia (IF 3.9) Pub Date : 2024-02-20 Sébastien Lobet, Cedric Hermans, Valérie-Anne Chantrain, Anthe Foubert, Catherine Lambert, Massimo Penta
The objective of this study is to assess the reliability and construct validity of ACTIVLIM-Hemo, a newly developed Rasch-built questionnaire designed to evaluate activity limitations in people with haemophilia (PwH), in comparison with the Haemophilia Activities List (HAL), which was developed using Classical Test Theory.
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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 ≥ 12 years followed in a Comprehensive Hemophilia Care Program in Canada Haemophilia (IF 3.9) Pub Date : 2024-02-20 Davide Matino, Federico Germini, Anthony K. C. Chan, Kay Decker, Emma Iserman, Pierre Chelle, Andrea N. Edginton, Olayide Oladoyinbo, Elisabetta Trinari, Arun Keepanasseril, Alfonso Iorio
Damoctocog alfa pegol (BAY 94–9027, Jivi®) is an extended half-life recombinant factor (F)VIII replacement, indicated for the treatment of haemophilia A in patients aged ≥12 years. Following introduction of damoctocog alfa pegol in Canada in 2020, there have been no reports on routine clinical effectiveness and satisfaction, when switching from a previous FVIII product in Canada.
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Dentistry for patients with haemophilia: Trialling a safe and economical change in management Haemophilia (IF 3.9) Pub Date : 2024-02-20 Pritam Daniel Sundaresan, Estie Kruger, Mathew Lim, John McGeachie, Marc Tennant
While the dental management of patients with haemophilia has changed considerably in the last decade, haemophiliacs in Western Australia have continued to receive pre-operative factor support for dentistry regardless of the type of dental procedure.
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The frequency of sarcopenia in haemophilia patients: Effects on musculoskeletal health and functional performance Haemophilia (IF 3.9) Pub Date : 2024-02-14 Bora Uzuner, Sertaç Ketenci, Dilek Durmus, Hilmi Memis Atay
In patients with haemophilia A, chronic arthropathy develops over time as a result of recurrent joint bleeds, which leads to restricted mobility and disability in the affected joints. There are limited studies in the literature evaluating sarcopenia in patients with haemophilia. The present study aims to determine the prevalence of sarcopenia in severe haemophilia-A patients and to evaluate musculoskeletal
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Morphological analysis of patellofemoral joint in haemophilic arthropathy: A case-control study Haemophilia (IF 3.9) Pub Date : 2024-02-13 Arman Vahabi, Elcil Kaya Biçer, Kayahan Kayıkçı, Fahri Şahin, Kaan Kavaklı, İpek Tamsel, Semih Aydoğdu
Knees affected by haemophilic arthropathy exhibit distinct differences in both bone morphology and soft tissue behaviour. This study aims to analyse the morphological characteristics of the distal femur and patellofemoral joint in patients with haemophilia in comparison to normal healthy population.
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Peri-operative desmopressin combined with pharmacokinetic-guided factor VIII concentrate in non-severe haemophilia A patients Haemophilia (IF 3.9) Pub Date : 2024-02-11 Lorenzo G. R. Romano, Lisette M. Schütte, Reinier M. van Hest, Karina Meijer, Britta A. P. Laros-van Gorkom, Laurens Nieuwenhuizen, Jeroen Eikenboom, Floor C. J. I. Heubel-Moenen, Nanda Uitslager, Michiel Coppens, Karin Fijnvandraat, Mariëtte H. E. Driessens, Suzanne Polinder, Marjon H. Cnossen, Frank W. G. Leebeek, Ron A. A. Mathôt, Marieke J. H. A. Kruip
Non-severe haemophilia A patient can be treated with desmopressin or factor VIII (FVIII) concentrate. Combining both may reduce factor consumption, but its feasibility and safety has never been investigated.
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Unearthing the genotype-inhibitor phenotype association in severe haemophilia A: A north Indian cohort study Haemophilia (IF 3.9) Pub Date : 2024-02-11 Debadrita Ray, Ritika Sharma, Narender Kumar, Chander Hans, Harikishan Senee, Manu Jamwal, Jasmina Ahluwalia, Reena Das, Deepak Bansal, Arihant Jain
Various risk factors for inhibitor development in haemophilia A (HA) have been described but Indian data remains scanty.
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Targeting a higher plasma VWF level at time of delivery in pregnant individuals with von Willebrand disease: Outcomes at a single-institution cohort study Haemophilia (IF 3.9) Pub Date : 2024-02-11 Ming Y. Lim, George M. Rodgers, D. Ware Branch, Sara E. Simonsen
Guidelines on the management of pregnant individuals with von Willebrand disease (VWD) at the time of delivery recommend that von Willebrand factor (VWF) and factor VIII:C (FVIII:C) levels be ≥50% to prevent postpartum haemorrhage (PPH). Yet, high PPH rates persist despite these levels or with prophylactic factor replacement therapy to achieve these levels.
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Bone mineral density in haemophilia patients: A systematic review and meta-analysis Haemophilia (IF 3.9) Pub Date : 2024-02-11 Meiling Zhang, Ke Song, Weifei Wu
With the increase in life expectancy of haemophilia patients (PWH), the risk of osteoporosis increases, but there is little research on whether haemophilia is the cause of osteoporosis.
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Measurement of recombinant porcine factor VIII in patients with congenital haemophilia A and inhibitors in the presence of emicizumab Haemophilia (IF 3.9) Pub Date : 2024-02-11 Christian Pfrepper, Robert Klamroth, Carmen Escuriola Ettingshausen, Sirak Petros, Annelie Siegemund, Thomas Siegemund
Recombinant porcine factor VIII (rpFVIII) is a treatment option for break-through bleeds in patients with congenital haemophilia A with inhibitors (CHAwI) on emicizumab. However, there are limited data about the measurement of rpFVIII in the presence of emicizumab.
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COMMITTEE LIST Haemophilia (IF 3.9) Pub Date : 2024-02-05
EAHAD 2024 CONGRESS PRESIDENT Prof. Dr. Wolfgang Miesbach Germany
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Main Congress Oral Abstracts Haemophilia (IF 3.9) Pub Date : 2024-02-05
OR01 Results of the first-in-human investigation of HMB-001 for prophylactic management of glanzmann thrombasthenia S. Sivapalaratnam1,2,*; S. Austin3; A. Gosnell4; U. Lorch5; T. York5; A. Want5; C. Rea4 1Digital Environment Research Institute (DERI), Queen Mary University of London, London, UK; 2Barts Health NHS Trust, London, UK; 3St George's University Hospitals NHS Trust, London, UK; 4Hemab Therapeutics
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Publication Only Abstracts Haemophilia (IF 3.9) Pub Date : 2024-02-05
PU001 Adynovi advances the advate experience by improving the quality of life C. C. Tarniceriu1,2,* 1Hematology, Emergency County Hospital “Sf. Spiridon” Iași; 2Anatomy, University of Medicines and Pharmacy “Grigore T. Popa”, Iasi, Romania Introduction: Haemophilia A is known as a hereditary coagulopathy that is manifested by the appearance of haemorrhagic episodes as a result of a quantitative/qualitative
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AUTHOR INDEX Haemophilia (IF 3.9) Pub Date : 2024-02-05
A Álvarez Román, M. T., 38, 77, 83, 112, 169 Álvarez-Román, M. T., 28 A El-assy, O., 121 Aamir, K., 159 Abío Calvete, M. D. L. O., 148 Abad-Franch, L., 59, 90 Abd El Fattah, M., 80 Abdel Hamid, D., 145 Abdel Hamid, M. A., 145 Abdelalim, N., 145 Abdellah, A., 145 Abdelwahab, M., 171 Abu Zeid, T., 170 Acharya, S. S., 127 Acquati Lozej, J., 124 Acuña-Butta, P., 28 Acuña, P., 145, 169 Adamsen, I., 102
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Eptacog beta for the management of patients with haemophilia A and B with inhibitors: A European perspective Haemophilia (IF 3.9) Pub Date : 2024-02-05 Wolfgang Miesbach, Manuel Carcao, Johnny Mahlangu, Yesim Dargaud, Victor Jimenez-Yuste, Cédric Hermans
Eptacog beta (activated), a recombinant human factor VIIa (rFVIIa), was approved by the US Food and Drug Administration (FDA) in 2020 (SEVENFACT®, LFB & HEMA Biologics) and the European Medicines Agency (EMA) in 2022 (CEVENFACTA®, LFB). In Europe, eptacog beta is indicated for the treatment of bleeds and the prevention of bleeds during surgery or invasive procedures in adults and adolescents (≥12 years
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Long-term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 years post-treatment Haemophilia (IF 3.9) Pub Date : 2024-02-05 Emily Symington, Savita Rangarajan, Will Lester, Bella Madan, Glenn F. Pierce, Priyanka Raheja, Tara M. Robinson, Dane Osmond, Chris B. Russell, Christian Vettermann, Suresh K. Agarwal, Mingjin Li, Wing Yen Wong, Michael Laffan
Valoctocogene roxaparvovec uses an adeno-associated virus serotype 5 (AAV5) vector to transfer a factor VIII (FVIII) coding sequence to individuals with severe haemophilia A, providing bleeding protection.
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Knowledge gaps in health-related quality of life research performed in children with bleeding disorders – A scoping review Haemophilia (IF 3.9) Pub Date : 2024-02-05 Elise J. Huisman, Caroline Mussert, Guannan Bai, Hein Raat, Marjon H. Cnossen
Bleeding disorders (BDs) may influence health-related quality of life (HRQoL) in children and caregivers. Measuring HRQoL gives insight into domains requiring support and provides an opportunity to evaluate the effects of novel therapies.
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Recombinant porcine factor VIII in patients with congenital haemophilia A with inhibitors undergoing surgery: Phase 3, single-arm, open-label study Haemophilia (IF 3.9) Pub Date : 2024-02-05 Christian Pfrepper, Paolo Radossi, Jerzy Windyga, Kaan Kavakli, Roger Schutgens, Nazan Sarper, Joan Gu, Kayode Badejo, Nisha Jain
Recombinant porcine factor VIII (rpFVIII; susoctocog alfa) is predicted to provide functional FVIII activity in patients with congenital haemophilia A with inhibitors (CHAWI).
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The value-based healthcare approach to haemophilia: Development of outcome measures for the evaluation of care of people with haemophilia Haemophilia (IF 3.9) Pub Date : 2024-02-05 Paolo Angelo Cortesi, Carla Fornari, Sara Conti, Berardino Pollio, Elena Boccalandro, Andrea Buzzi, Christian Carulli, Antonio Coppola, Raimondo De Cristofaro, Matteo Nicola Dario Di Minno, Gerard Dolan, Enrico Ferri Grazzi, Arianna Fornari, Roberta Gualtierotti, Cedric Hermans, Victor Jiménez-Juste, Gili Kenet, Angelo Lupi, Carlo Martinoli, Maria Francesca Mansueto, Gabriella Nicolò, Annarita Tagliaferri
Considering the advances in haemophilia management and treatment observed in the last decades, a new set of value-based outcome indicators is needed to assess the quality of care and the impact of these medical innovations.
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Safety of recombinant activated factor VII for treatment of breakthrough bleeds in patients with congenital haemophilia A and inhibitors receiving emicizumab prophylaxis: Review of the real-world evidence Haemophilia (IF 3.9) Pub Date : 2024-01-30 Gili Kenet, Teruhisa Fujii
Emicizumab is used as a subcutaneous prophylaxis for prevention of bleeding episodes in patients with haemophilia A (HA) with and without inhibitors. While low bleeding rates were observed in clinical trials, patients still experience breakthrough bleeds (BTBs) with emicizumab in the real-world. Current guidelines recommend use of recombinant activated factor VII (rFVIIa) for treatment of BTBs in patients
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A post hoc comparative real-world analysis of HEAD-US score for joint health assessment of patients with severe haemophilia A and B in Spain Haemophilia (IF 3.9) Pub Date : 2024-01-28 María Teresa Álvarez-Román, Víctor Jiménez-Yuste, Mónica Martín-Salces, Hortensia De la Corte-Rodríguez, Santiago Bonanad, Ramiro Núñez, Nuria Fernández-Mosteirín, Luis Javier García-Frade, Carlo Martinoli, Hae Kyung Kim
Joint damage due to haemarthrosis can be effectively monitored with point-of care ultrasound using the Haemophilia Early Arthropathy Detection with US (HEAD-US) scoring system. A post hoc comparative analysis of the joint status of patients with severe haemophilia A (HA) or B (HB) was performed.
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Impact of inherited bleeding disorders on maternal bleeding and other pregnancy outcomes: A population-based cohort study Haemophilia (IF 3.9) Pub Date : 2024-01-24 Arafat Ul Alam, Cynthia Wu, Padma Kaul, Venu Jain, Haowei (Linda) Sun
Increasing rate of postpartum haemorrhage (PPH) has been observed between 2003 and 2010 in Canada. Inherited bleeding disorders contribute to the risk of PPH.
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Non-surgical correction of knee flexion deformity in persons with haemophilia: A staged multidisciplinary approach Haemophilia (IF 3.9) Pub Date : 2024-01-21 Merlyn Tilak, Judy Ann John, Arun Paul, Anumeha Srivastava, Divya Singh, Andriya Rajendran, Prince Thakkar, Naveen Cherian, Sandeep Albert, Pradeep Poonnoose, Abraham Sunder Singh, Kavitha M. Lakshmi, Nambiathayil Abubacker Fouzia, Aby Abraham, Alok Srivastava
Flexion deformity of the knee is a common complication following recurrent haemarthrosis in persons with haemophilia (PWH) on episodic factor replacement therapy, restricting independent mobility. There is limited literature on the comprehensive management of this condition. This report provides the outcome of a staged multidisciplinary approach for the correction of knee flexion deformity (KFD) even
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Expert United Kingdom consensus on the preservation of joint health in people with moderate and severe haemophilia A: A modified Delphi panel Haemophilia (IF 3.9) Pub Date : 2024-01-19 Mike Laffan, Paul McLaughlin, Jayashree Motwani, Jayanthi Alamelu, Steve Austin, Stephen Classey, Gerard Dolan, Marie Eales, Richard Gooding, John Grainger, Catherine Harrison, April Jones, Anne M. Kelly, Lara Oyesiku, Ryan Rodgers, David Stephensen, Kate Talks, Shaneil Sonecha, Andrew Danquah
For people with haemophilia A (PwHA), bleeding in the joints leads to joint damage and haemophilia-related arthropathy, impacting range of motion and life expectancy. Existing guidelines for managing haemophilia A support healthcare professionals (HCPs) and PwHA in their efforts to preserve joint health. However, such guidance should be reviewed, considering emerging evidence and consensus as presented
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Joint bleeds in mild hemophilia: Prevalence and clinical characteristics Haemophilia (IF 3.9) Pub Date : 2024-01-19 John B. Chiari, Stephanie Prozora, Richard Feinn, Evangelia Louizos, Patrick G. Gallagher, Robert Bona
Joint bleeds are a common and frequent complication associated with hemophilia, increasing the risk of hemophilic arthropathy. It is important to define and characterize the presence of joint complications in mild hemophilia to develop strategies to mitigate disease burden.
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Health-related quality of life of Indonesian children with hemophilia Haemophilia (IF 3.9) Pub Date : 2024-01-19 Elsa Maimon, Pudjo Hagung Widjajanto, Mei Neni Sitaresmi
Measuring health-related quality of life (HRQoL) in haemophilia patients provides a comprehensive patient's functional and the impact of the disease and its treatment.
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Safety and efficacy of damoctocog alfa pegol prophylaxis in patients with severe haemophilia A: Results of an interventional, post-marketing study Haemophilia (IF 3.9) Pub Date : 2024-01-16 Pål André Holme, Lone Hvitfeldt Poulsen, Claudia Tueckmantel, Monika Maas Enriquez, María Teresa Alvarez Román, Raimondo De Cristofaro
Damoctocog alfa pegol (BAY 94-9027, Jivi®) is an approved extended half-life factor VIII (FVIII) for treatment of previously treated patients with haemophilia A aged ≥12 years. We report the final results of an interventional, post-marketing study of damoctocog alfa pegol prophylaxis in patients with severe haemophilia A.
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Patient and caregiver preferences for haemophilia treatments: A discrete-choice experiment Haemophilia (IF 3.9) Pub Date : 2024-01-10 Viridiana Cano Garcia, Carol Mansfield, Anna Pierce, Colton Leach, Jane Cavanaugh Smith, Marion Afonso
An evolving haemophilia treatment landscape provides new possibilities for previously unattainable lifestyles.
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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-centre, real-world experience of surgery Haemophilia (IF 3.9) Pub Date : 2023-12-27 Stéphanie Désage, Christophe Nougier, Sandrine Meunier, Valérie Chamouard, Emilie Jousselme, Yesim Dargaud, Anne Lienhart
Efmoroctocog alfa (rFVIIIFc) is an extended half-life FVIII used notably in surgery for patients with haemophilia A. More information is needed of its usage in real-life.
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Cost-effectiveness analysis of emicizumab prophylaxis in patients with haemophilia A in India Haemophilia (IF 3.9) Pub Date : 2023-12-26 Tulika Seth, M. Joseph John, Prantar Chakrabarti, Chandrakala Shanmukhaiah, Shailendra Prasad Verma, Nita Radhakrishnan, Tuphan Kanti Dolai
Emicizumab is the initial subcutaneously administered bispecific antibody approved as a prophylactic treatment for patients with haemophilia A (PwHA).
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The psychosocial impact of haemophilia from patients’ and caregivers’ point of view: The results of an Italian survey Haemophilia (IF 3.9) Pub Date : 2023-12-26 Arianna Fornari, Ippazio Cosimo Antonazzo, Angiola Rocino, Daniele Preti, Anna Fragomeno, Francesco Cucuzza, Nicola Ceresi, Cristina Santoro, Antonietta Ferretti, Rita Facchetti, Paolo Cozzolino, Chiara Biasoli, Cristina Cassone, Antonio Coppola, Paolo Angelo Cortesi, Lorenzo Giovanni Mantovani
A huge amount of data about psychosocial issues of people with haemophilia (PwH) are available; however, these materials are fragmentary and largely outdated, failing to reflect the impact of current treatment strategies.
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Increasing optimal coagulation factor dosing in the paediatric emergency department: Update to a quality improvement study Haemophilia (IF 3.9) Pub Date : 2023-12-25 Nicole E. Kendel, Eman Abdelghani, Joseph R. Stanek, Brockton Mitchell, Michael W. Dunn, Vilmarie Rodriguez, Amy L. Dunn
Prompt, appropriate coagulation factor replacement according to injury and bleeding severity in persons with haemophilia is required to prevent acute and long-term complications.
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Observational cohort study of long-term outcomes of liver transplantation in haemophilia Haemophilia (IF 3.9) Pub Date : 2023-12-18 Margaret V. Ragni, Jessica Callis, Nabil Daoud, Brian Hu, Matthew Manuel, Jarren Santos, Jonathan Schwartz, Kenneth D. Friedman, Peter Kouides, Philip Kuriakose, Andrew D. Leavitt, Ming Y. Lim, Nicoletta Machin, Michael Recht, Tammuella Chrisentery-Singleton
Gene therapy is now a reality for individuals with haemophilia, yet little is known regarding the quality-of-life impact of factor correction. As few data exist, and recognizing the analogy to liver transplantation (OLTX), we identified OLTX+ and OLTX- men in the ATHNdataset to compare post-OLTX factor VIII and IX on quality of life (QoL) by Haem-A-QoL and PROMIS-29.
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Occupational therapy in the management of haemophilia patients: A scoping review Haemophilia (IF 3.9) Pub Date : 2023-12-18 Marta Elisa Seoane-Martín, Rubén Cuesta-Barriuso, María Carmen Rodríguez-Martínez
Haemophilia is a haematological disease, although most haemorrhages occur in the locomotor system. Patients are physically disabled from an early age and have a poorer perception of quality of life. In the day-to-day lives of patients and their families, psychosocial well-being, the disease's physical, personal, and social impact, as well as work-related problems are the most complicated aspects of
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Gene therapy for haemophilia A and B, from basic principles to clinical implementation: An illustrated review Haemophilia (IF 3.9) Pub Date : 2023-12-18 Cihan Ay, Laurent Frenzel, Karen Pinachyan, Sandra Le Quellec
With recent approval of the first two gene therapies for haemophilia A and B, educational materials about AAV-based gene therapy are needed by the haemophilia community for a better understanding of this novel therapeutic approach and helping healthcare providers and patients making personalized choices amongst an increasing array of therapeutic options.
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Management strategies in persons with inherited haemophilia requiring antithrombotic therapy: A scoping review Haemophilia (IF 3.9) Pub Date : 2023-12-12 Kelsey Uminski, Yan Xu, Amin Zahrai, Amanda Hodgson, Tzu-Fei Wang, Lisa Duffett, Alan Tinmouth, Roy Khalife
Thromboembolic events are increasingly reported in the aging haemophilia population. The purpose of this study was to understand current practices and identify knowledge and research gaps in the management of persons with haemophilia requiring antithrombotic therapy for cardiovascular disorders (CVD) or venous thromboembolism (VTE).
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A single-centre experience of 29 total ankle replacement in haemophiliac patients: Therapeutic management, factor consumption and cost Haemophilia (IF 3.9) Pub Date : 2023-12-11 Louise Favrelle, Jean-Baptiste Masson, Stéphanie Parat, Emmanuelle Carre, Michel Fessy, Catherine Rioufol, Anne Lienhart, Valérie Chamouard, Jean-Luc Besse
In patients with haemophilia, repeated bleeding in large joints leads to chronic haemophilic arthropathy, a rare disease that can be managed surgically with ankle arthrodesis or with total ankle replacement (TAR). TAR has been reported to provide good surgical results in the medium/long-term and allow preservation of joint mobility but the medical therapeutic management of the patients has not been