Results 161 to 170 of about 147,268 (335)

Performing Large‐Scale Genetic Analysis in the Bleeding Disorders Community

Haemophilia, EarlyView.
ABSTRACT Inherited bleeding disorders encompass a diverse group of conditions caused by genetic defects affecting coagulation factors, fibrinogen, von Willebrand factor, or platelet function. Despite major advances in quantitative and functional laboratory assays, a substantial diagnostic gap remains, particularly in patients with mild or atypical ...
Anna R. Blankstein, Sterre P. E. Willems, Saskia E. M. Schols, Rosanna Asselta, Gillian Lowe, Neil V. Morgan, Paula D. James   +6 more
wiley   +1 more source

Rare Bleeding Disorders and Bleeding Disorder of Unknown Cause: Current Understanding and Recent Developments

Haemophilia, EarlyView.
ABSTRACT Rare bleeding disorders (RBDs) represent a diverse group of inherited conditions involving coagulation factors or platelets. These conditions, such as Glanzmann thrombasthenia (GT) or severe coagulation factor deficiencies, are uncommon. In contrast, bleeding disorder of unknown cause (BDUC) is a diagnosis of exclusion without an identifiable ...
Alessandro Casini, Robert Klamroth, Bauke Haisma, Dino Mehic, Saskia E. M. Schols   +4 more
wiley   +1 more source

The Swiss Haemophilia Registry–Report From the First 8 Years

Haemophilia, EarlyView.
ABSTRACT Introduction Patient registries capture disease related information and provide a valuable source for real‐world data on rare diseases and their management. The Swiss Haemophilia Registry (SHR) was established in 2015 on the basis of a new Swiss federal human research act.
Alessandra Bosch, Lorenzo Alberio, Pierre Fontana, Lukas Graf, Johanna A. Kremer Hovinga, Nicolas von der Weid, Mattia Rizzi, Manuela Albisetti, the Swiss Haemophilia Network   +8 more
wiley   +1 more source

Seventh Åland Island Meeting on von Willebrand Disease

Haemophilia, EarlyView.
ABSTRACT Introduction The seventh Åland Island Meeting on von Willebrand Disease (VWD) was held on the Åland archipelago in Finland, from 26 to 28 September 2024. Aim The meeting brought together experts in the field of VWD from around the world to share the latest advances and knowledge in VWD.
Riitta Lassila, Ruben Bierings, Nathan T. Connell, Fernando F. Corrales‐Medina, Augusto B. Federici, Veronica H. Flood, Floor Heubel‐Moenen, Jill M. Johnsen, Geoffrey Kuppens, Davide Matino, Carolyn Millar, Ingmar Schoen, Anish Sharda, Robert F. Sidonio Jr, Alok Srivastava, Timea Szanto, Angela C. Weyand, Erik Berntorp   +17 more
wiley   +1 more source

Beyond Standard Half‐Life: Real‐world Pharmacokinetics of Efanesoctocog Alfa in a Single Centre

Haemophilia, EarlyView.
ABSTRACT Introduction Efanesoctocog alfa (EFA) is an ultra–extended half‐life factor VIII (FVIII) developed to address limitations of conventional prophylaxis in haemophilia A. Although the XTEND trials reported low interindividual pharmacokinetic (PK) variability, real‐world data remain important to better characterize PK profiles across patient ...
Laurent Sattler, Agathe Herb, Léa Pierre, Jordan Wimmer, Manon Dolt, Olivier Feugeas, Dominique Desprez   +6 more
wiley   +1 more source

Optimizing Perioperative Management of Haemophilia B With rFIX‐FP: Pharmacokinetic Validation of the Hemoptidose Tool

Haemophilia, EarlyView.
Abstract Background Surgical management of haemophilia B requires precise factor IX replacement to ensure adequate haemostasis while optimizing factor consumption. Extended half‐life rFIX‐FP simplifies perioperative management but exhibits substantial pharmacokinetic variability.
Xavier Delavenne, Célia Vollard, Nicolas Drillaud, Sabine Castet, Nicolas Luyck, Emilie Hénin, Julien Denis‐Le Seve, Fabienne Volot, Brigitte Tardy, Stéphanie Désage, Benoît Guillet   +10 more
wiley   +1 more source

Responsiveness and Minimal Important Change of the Haemophilia Activities List in Patients With Inherited Bleeding Disorders

Haemophilia, EarlyView.
ABSTRACT Introduction The Haemophilia Activities List (HAL) is a disease‐specific patient‐reported outcome measure (PROM) that is widely used in both healthcare and research in people with inherited bleeding disorders (PWBD), but information on responsiveness and minimal important change (MIC) is lacking.
Johan Blokzijl, Isolde A. R. Kuijlaars, Martijn F. Pisters, Cindy Veenhof, Janjaap van der Net, Roger E. G. Schutgens, Kathelijn Fischer, Merel A. Timmer   +7 more
wiley   +1 more source

An Ex Vivo Pharmacodynamic Study of KN057, a Tissue Factor Pathway Inhibitor Neutralizing Antibody, in Plasma Samples From Patients With Haemophilia or VWD3

Haemophilia, EarlyView.
ABSTRACT Introduction Tissue factor pathway inhibitor (TFPI), a key regulator of tissue factor‐initiated coagulation through FXa‐dependent inhibition of the tissue factor‐FVIIa complex, has emerged as a promising target for restoring thrombin generation.
Mankai Ju, Feng Xue, Wei Sun, Fang Li, Ting Xu, Renchi Yang   +5 more
wiley   +1 more source

Optimizing Emergency Department Care for People With Bleeding Disorders: A Scoping Review of Barriers and Interventions for Improvement

Haemophilia, EarlyView.
ABSTRACT Background Emergency department (ED) care is critical for managing acute bleeding events in people with bleeding disorders. Despite international guidelines recommending haemostatic treatment within 30–60 min, delays and deviations from best practices are common and associated with poorer outcomes.
Ling‐Yi Guo, Diane L. Lorenzetti, Natalia Rydz, Dawn Goodyear, Julia Hews‐Girard, Garrick Mok, Roy Khalife, Kelsey Uminski   +7 more
wiley   +1 more source

Small Bowel Lesions and Bleeding Risk in Hemodialysis Patients: A Narrative Review

Hemodialysis International, EarlyView.
ABSTRACT Background Gastrointestinal bleeding is very common among hemodialysis patients. This high bleeding risk is caused by uremic platelet dysfunction, vascular fragility, intradialytic hemodynamic instability, and widespread antithrombotic therapy.
Andreas Smyrlis, Konstantia Kantartzi, Konstantinos Mimidis, Stylianos Panagoutsos   +3 more
wiley   +1 more source