Results 31 to 40 of about 1,547 (116)

Normalisation of haemoglobin and control of breakthrough haemolysis with increased frequency pegcetacoplan dosing in treated paroxysmal nocturnal haemoglobinuria

eJHaem, 2023
Paroxysmal nocturnal haemoglobinura is an acquired life‐threatening haemolytic condition, which is generally well controlled with terminal complement blockade with eculizumab. Whilst almost all patients treated with terminal complement inhibitors develop
Amanda K. Davis, Nicholas Bingham, Jeff Szer   +2 more
doaj   +1 more source

Efficacy and safety of current treatments for paroxysmal nocturnal hemoglobinuria: A systematic review

Clinical Immunology Communications, 2023
Paroxysmal nocturnal hemoglobinuria (PNH) is a non-malignant clonal disorder of the pluripotent hematopoietic stem cell. Currently, Eculizumab, Ravulizumab, and Pegcetacoplan are the approved drugs to treat PNH. In order to assess the efficacy and safety
Yara Maria da Silva Pires, Aline de Fátima Bonetti, Jessica Telma Ciecilinsky, Astrid Wiens Souza   +3 more
doaj   +1 more source

The cost–effectiveness of pegcetacoplan in complement treatment-naive adults with paroxysmal nocturnal hemoglobinuria in the USA

Journal of Comparative Effectiveness Research, 2023
Aim: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by hemolytic anemia, bone marrow failure and thrombosis, and is associated with high healthcare burden.
Jesse Fishman, Koo Wilson, Aleksandra Drzewiecka, Michal Pochopien, David Dingli   +4 more
doaj   +1 more source

Rise of the planet of rare anemias: An update on emerging treatment strategies

Frontiers in Medicine, 2023
Therapeutic options for rare congenital (hemoglobinopathies, membrane and enzyme defects, congenital dyserythropoietic anemia) and acquired anemias [warm autoimmune hemolytic anemia (wAIHA), cold agglutinin disease CAD, paroxysmal nocturnal ...
Bruno Fattizzo, Bruno Fattizzo, Irene Motta, Irene Motta   +3 more
doaj   +1 more source

Switching patients with PNH from pegcetacoplan to iptacopan: a case series

Hematology
Objectives Limited published data exist on switching therapy from pegcetacoplan to iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH).Methods Three patient cases were collected from the Mayo Clinic, Cleveland Clinic, and Levine Cancer ...
Mark Orland, Carmelo Gurnari, Srinivasa Sanikommu, Jaroslaw Maciejewski, David Dingli   +4 more
doaj   +1 more source

Evaluating the Effects of C3 Inhibition on Geographic Atrophy Progression from Deep-Learning OCT Quantification: A Split-Person Study

Ophthalmology and Therapy, 2023
Introduction To evaluate the effect pegcetacoplan, a C3 and C3b inhibitor, on the rate of progression of geographic atrophy (GA) as assessed by spectral domain optical coherence tomography (SD-OCT) using a split-person study design and deep-learning ...
Dun Jack Fu, Veronika Lipkova, Bart Liefers, Sophie Glinton, Livia Faes, Alex McKeown, Lukas Scheibler, Nikolas Pontikos, Praveen J. Patel, Gongyu Zhang, Pearse A. Keane, Konstantinos Balaskas   +11 more
doaj   +1 more source

Population Pharmacokinetics of Pegcetacoplan in Patients with Geographic Atrophy or Neovascular Age-related Macular Degeneration

Ophthalmology Science
Objective: To develop a population pharmacokinetic (PK) model to characterize serum pegcetacoplan concentration-time data after intravitreal administration in patients with geographic atrophy (GA) or neovascular age-related macular degeneration (nAMD ...
Ryan L. Crass, PharmD, Komal Prem, MD, Francois Gauderault, PhD, Ramiro Ribeiro, MD, PhD, Caroline R. Baumal, MD, Brandon Smith, PhD, Daniel Epling, PhD, Sunny Chapel, PhD   +7 more
doaj   +1 more source

Can intravitreal injections with higher volume cause higher intraocular eye pressure? Considerations for anti-complement injections in normal and glaucomatous eyes

International Journal of Retina and Vitreous, 2023
The approval of Syfovre® (pegcetacoplan) and Iverzay® (avacincaptad pegol) for the treatment of geographic atrophy (GA) marks a significant advancement in retinal disease therapy, offering both complement 3 and complement 5 inhibitors.
Carsten H. Meyer, Gustavo Barreto Melo, Arshad M. Khanani   +2 more
doaj   +1 more source

Pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria - efficacy and comparison with eculizumab

Introduktion: Paroxysmal nokturn hemoglobinuri (PNH) är en kronisk, förvärvad, mycket sällsynt sjukdom som kännetecknas av komplementmedierad intravaskulär hemolys och är potentiellt livshotande.
Pelkonen, Essi
core   +4 more sources

Pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal haemoglobinuria (PEGASUS): 48-week follow-up of a randomised, open-label, phase 3, active-comparator, controlled trial

, 2022
Background: In the PEGASUS trial, the complement C3 inhibitor, pegcetacoplan, showed superiority to eculizumab in improving haematological outcomes in adult patients with paroxysmal nocturnal haemoglobinuria and suboptimal response to eculizumab at 16 ...
Mohammed Al-Adhami, Röth, Alexander, Panse, Jens Peter, Szer, Jeff, Panse, Jens, Jeff Szer, Deschatelets, Pascal, Kensuke Usuki, Griffin, Morag, Hillmen, Peter, Hisakazu Nishimori, Jens Panse, Britta Höchsmann, Temitayo Ajayi, Risitano, Antonio M., Al-Adhami, Mohammed, Weitz, Ilene C., Kiladjian, Jean-Jacques, Risitano, Antonio M, Höchsmann, Britta, Usuki, Kensuke, Jean-Jacques Kiladjian, de Castro, Carlos M., de Latour, Régis Peffault, Alexander Röth, Weitz, Ilene C, Nishimori, Hisakazu, de Castro, Carlos M, Ilene C Weitz, Cedric Francois, Ajayi, Temitayo, Régis Peffault de Latour, Grossi, Federico, Peter Hillmen, Morag Griffin, Carlos M de Castro, Francois, Cedric, Antonio M Risitano, Pascal Deschatelets, Federico Grossi   +39 more
core   +1 more source