Results 31 to 40 of about 1,795 (145)

Determinants of Immune Tolerance Induction Outcomes in 237 Persons With Severe Hemophilia A

European Journal of Haematology, EarlyView.
ABSTRACT Immune tolerance induction (ITI) to eradicate an immune response against factor VIII (FVIII) has been used in hemophilia A (HA) with various success rates, costs, and treatment burden. The present study aimed to identify markers predictive of ITI success.
Marcus Fager Ferrari, Josefin Roslund, Eric Manderstedt, Erik Berntorp, Jan Astermark, Malmö and Hemophilia Inhibitor Genetics Study Investigators   +5 more
wiley   +1 more source

FVIIIa Mimetics: New Approaches and Next‐Generation Initiatives

Haemophilia, EarlyView.
ABSTRACT Emicizumab has revolutionized hemophilia A care, yet limitations regarding the “ceiling” of hemostatic efficacy (equivalent to mild hemophilia) and global access persist. This review critically examines two distinct paradigms shaping the future of care: Innovation and Access. Regarding innovation, we synthesize the latest clinical data on next‐
Tadashi Matsushita, Keiji Nogami, Chandrakala Shanmukhaiah   +2 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

Emicizumab in tolerized patients with hemophilia A with inhibitors: A single‐institution pediatric cohort assessing inhibitor status

Research and Practice in Thrombosis and Haemostasis, 2021
Background The majority of patients with hemophilia A with inhibitors who undergo immune tolerance induction (ITI) achieve successful tolerance and transition to factor VIII (FVIII) prophylaxis. A portion of these patients have switched to emicizumab for
Glaivy Batsuli, Amanda Greene, Shannon L. Meeks, Robert F. Sidonio Jr   +3 more
doaj   +1 more source

Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A

HemaSphere, 2022
The objective was to undertake an early cost-effectiveness assessment of valoctocogene roxaparvovec (valrox; Roctavian) compared to factor (F)VIII prophylaxis or emicizumab (Hemlibra; Roche HQ, Bazel, Switzerland) in patients with severe Hemophilia A (HA)
Renske M.T. ten Ham, Sikon M. Walker, Marta O. Soares, Geert W.J. Frederix, Frank W.G. Leebeek, Kathelijn Fischer, Michiel Coppens, Stephen J. Palmer   +7 more
doaj   +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

Laboratory Assessment of Emicizumab Levels in Hemophilia A: Influence of Assay Selection on Reported Results

Clinical and Applied Thrombosis/Hemostasis
Background Emicizumab has emerged as a promising therapy for hemophilia A (HA), employing a bypassing mechanism to restore hemostasis. However, since the traditional factor assays cannot be used for measuring the effect of emicizumab, treatment ...
Charlotte Gran MD, PhD, Tony Frisk MD, PhD, Margareta Homström MD, PhD, Maria Magnusson MD, PhD, Susanna Ranta MD, PhD, Anna Sjöström MD, PhD, Marjan Shafaati Lambert, Nida Mahmoud Hourani Soutari BSc, Jovan P. Antovic MD, PhD, Roza Chaireti MD, PhD   +9 more
doaj   +1 more source

AOZORA study: 3-year interim analysis of safety and joint health in pediatric people with hemophilia A receiving emicizumab prophylaxis

Research and Practice in Thrombosis and Haemostasis
Background: Recurrent joint bleeding in people with hemophilia A (PwHA) can cause hemophilic arthropathy, resulting in limited movement and chronic pain.
Midori Shima, Hideyuki Takedani, Kaoru Kitsukawa, Masashi Taki, Akira Ishiguro, Chiai Nagae, Azusa Nagao, Daisuke Nosaka, Yui Kyogoku, Hiroki Oki, Keisuke Iwasaki, Keiji Nogami   +11 more
doaj   +1 more source

Italian Patients Journey for Gene Therapy in Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Gene therapy (GT) provides sustained FVIII levels without repeated infusions in Hemophilia A (HA) patients thus overcoming a major limitation of replacement therapy. However, issues remain e.g., patient selection criteria, duration and variability of transgene expression, quality of life and long‐term safety.
Giovanni Di Minno, Gaia Spadarella, Alessandra Borchiellini, Anna Borrelli, Andrea Buzzi, Giancarlo Castaman, Michele Cecchi, Raimondo De Cristofaro, Vincenzo La Mura, Angelo Lupi, Giuseppe Malcangi, Maria Elisa Mancuso, Maria Francesca Mansueto, Cristina Santoro, Rita Carlotta Santoro, Luigi Piero Solimeno, Annarita Tagliaferri, Ezio Zanon, Isabella Garagiola, Flora Peyvandi   +19 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