Results 31 to 40 of about 820 (140)

Traumatic Tonsillar Hemorrhage during Hemophilia A Treatment with Emicizumab

, 2022
Reports on the treatment of bleeding associated with emicizumab administration are scarce. Herein, we report the case of an eight-year-old boy with moderate hemophilia A with an inhibitor who experienced tonsillar hemorrhage while undergoing emicizumab ...
Fumiya Inoue, Shunji Igarashi, Yasushi Noguchi, Kazuki Terada, Kazuki Furudate   +4 more
core   +1 more source

Anti-emicizumab antibodies and their relevance in clinical practice

Research and Practice in Thrombosis and Haemostasis
Background: Emicizumab is licensed for treatment of people with hemophilia A (HA) of all ages, with and without factor (F)VIII inhibitors. It is well tolerated, and most of the treatment-related adverse events are of mild intensity and transient.
Carla Valsecchi, Lucia Schiavone, Sara Arcudi, Adriana Torri, Cristina Novembrino, Flora Peyvandi   +5 more
doaj   +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

Final Analysis of the Phase 1/2 Trial of Valoctocogene Roxaparvovec for Severe Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Valoctocogene roxaparvovec is an adeno‐associated virus vector serotype 5 (AAV5)‐mediated gene therapy for severe haemophilia A (HA). Aim Report the final safety and efficacy results of the phase 1/2 trial of valoctocogene roxaparvovec. Methods An open‐label phase 1/2 trial (NCT02576795) enrolled adult males with severe HA (factor
Priyanka Raheja, Savita Rangarajan, Will Lester, Bella Madan, Glenn F. Pierce, Emily Symington, Carolyn Millar, Dane Osmond, Mingjin Li, Konstantia‐Maria Chavele   +9 more
wiley   +1 more source

A hemophilia A mouse model for the in vivo assessment of emicizumab function

, 2020
International audienceThe bispecific antibody emicizumab is increasingly used for hemophilia A treatment. However, its specificity for human factors IX and X (FIX and FX) has limited its in vivo functional analysis to primate models of acquired ...
Kawecki, Charlotte, Peyron, Ivan, Kauskot, Alexandre, Lenting, Peter, J., Ferrière, Stephen, Muczynski, Vincent, Christophe, Olivier, D., Nathwani, Amit, Casari, Caterina, Denis, Cecile   +9 more
core   +1 more source

Essentials of Laboratory Issues in the Patients with Emicizumab Therapy

, 2023
The recent development of emicizumab (Hemlibra, previously referred to as ACE910; Hoffman-La Roche) extends treatment options for hemophilia A patients, with and without anti-factor VIII (FVIII) inhibitors, and provides an alternative to FVIII ...
Sang Hyuk Park
core   +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

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

Real‐World Use of Emicizumab in Patients With Acquired Haemophilia A: An Interim Safety Analysis of a Large‐Scale Post‑Marketing Surveillance Study

Haemophilia, EarlyView.
ABSTRACT Introduction Acquired haemophilia A (AHA) is a rare autoimmune disorder where the development of autoantibodies to factor (F)VIII neutralise its function, leading to bleeding. Emicizumab has been approved for treating AHA in Japan. Aim This post‐marketing study was performed to primarily examine the use and safety of emicizumab, and indirectly
Midori Shima, Katsuyuki Fukutake, Yoshiyuki Ogawa, Yuta Kamei, Chiaki Sugita, Akinori Yuri, Ryota Kobayashi, Akiko Ioka, Tadashi Matsushita   +8 more
wiley   +1 more source