Results 31 to 40 of about 82,322 (154)

Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor‐Naive Patients With Paroxysmal Nocturnal Haemoglobinuria

eJHaem
Background Paroxysmal nocturnal haemoglobinuria (PNH) is characterised by haemolytic anaemia, bone marrow failure and thrombosis. The single‐arm phase 3 APPOINT‐PNH trial (NCT04820530) investigating iptacopan monotherapy in complement inhibitor‐naive ...
Matthew Holt, Richard J. Kelly, Jilles M. Fermont, Soudeh Ansari, Marion Dahlke, Isabelle Brindel, Lynda Maafa, Flore Sicre deFontbrune, Régis Peffault deLatour   +8 more
doaj   +1 more source

Thrombolytic therapy is effective in paroxysmal nocturnal hemoglobinuria: a series of nine patients and a review of the literature

Haematologica, 2012
Background Thrombosis is the major risk factor for death in patients with paroxysmal nocturnal hemoglobinuria. Previous case reports indicate that venous thrombosis in patients with paroxysmal nocturnal hemoglobinuria is amenable to thrombolysis.Design ...
David J. Araten, Rosario Notaro, Howard T Thaler, Nancy Kernan, Farid Boulad, Hugo Castro-Malaspina, Trudy Small, Andromachi Scaradavou, Heather Magnan, Susan Prockop, Sara Chaffee, Jason Gonsky, Raymond Thertulien, Roberto Tarquini, Lucio Luzzatto   +14 more
doaj   +1 more source

Beyond Nature's Blueprint: Macrocyclic Peptides Containing Unnatural Amino Acids in Therapeutic Development

Angewandte Chemie International Edition, EarlyView.
Strategic incorporation of unnatural amino acids transforms macrocyclic peptides into drug‐like molecules capable of engaging challenging targets. These building blocks enhance stability, permeability, and bioavailability, accelerating the development of next‐generation peptide therapeutics.
Krishna K. Sharma, Komal Sharma, Anku Sharma, Kamya Rao, Rahul Jain, Brett VanVeller   +5 more
wiley   +1 more source

Polymorphism of the complement receptor 1 gene correlates with the hematologic response to eculizumab in patients with paroxysmal nocturnal hemoglobinuria

Haematologica, 2014
Complement blockade by eculizumab is clinically effective in hemolytic paroxysmal nocturnal hemoglobinuria. However, the response is variable and some patients remain dependent on red blood cell transfusions.
Tommaso Rondelli, Antonio M. Risitano, Régis Peffault de Latour, Michela Sica, Benedetta Peruzzi, Patrizia Ricci, Wilma Barcellini, Anna Paola Iori, Carla Boschetti, Veronica Valle, Veronique Frémeaux-Bacchi, Maria De Angioletti, Gerard Socie, Lucio Luzzatto, Rosario Notaro   +14 more
doaj   +1 more source

COVID‐19 infection presenting as paroxysmal nocturnal hemoglobinuria

Clinical Case Reports, 2021
A diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) was elicited during acute COVID‐19 infection. COVID‐19 spike proteins trigger the alternative pathway of complement. Acute SARS‐CoV‐2 infection possibly expanded an existing PIG‐A mutation.
Adam Hines, Nausheen Hakim, Jacqueline Barrientos   +2 more
doaj   +1 more source

Exploring individualized crovalimab dosing in PNH through in silico modelling: Potential for improved convenience and cost efficiency

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, acquired haematopoietic stem cell disorder. Crovalimab, a complement C5‐inhibitor, is approved for PNH and can be self‐administered subcutaneously every 4 weeks, offering a more convenient route than intravenous C5‐inhibitors.
Mendy ter Avest, Simon E. Koele, Nicole C. A. J. van de Kar, Saskia M. C. Langemeijer, Rob ter Heine   +4 more
wiley   +1 more source

Paroxysmal nocturnal hemoglobinuria: Pandora’s box?

Journal of Mind and Medical Sciences, 2020
Introduction. Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired non-malignant hematological disorder which affects the pluripotent hematopoietic stem cell.
Mihnea-Alexandru Găman, Iulia Ursuleac, Daniel Coriu   +2 more
doaj   +1 more source

Venous Thromboembolism in Hematologic Malignancies: Incidence, Risk Factors, and the Role of Direct Oral Anticoagulants

European Journal of Haematology, EarlyView.
ABSTRACT Thrombotic events, particularly venous thromboembolism (VTE), are a significant source of morbidity and mortality among patients with hematologic malignancies. These patients face unique challenges due to treatment‐related complications such as thrombocytopenia, coagulopathy, and heightened bleeding risk.
Mario Biglietto, Silvia Sorella, Erminia Baldacci, Massimo Breccia, Sabina Chiaretti, Marcello Capriata, Livia Donzelli, Gianna Maria D'Elia, Salvatore Giacomo Morano, Ilaria Del Giudice, Maurizio Martelli, Cristina Santoro, Antonio Chistolini   +12 more
wiley   +1 more source

Clonal Dynamics of GPI‐Deficient Cells in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH): A Retrospective Follow‐Up Analysis

European Journal of Haematology, EarlyView.
ABSTRACT This retrospective, single‐center study aimed to characterize clonal dynamics of GPI‐deficient cells in patients with paroxysmal nocturnal hemoglobinuria (PNH) or PNH/aplastic anemia (AA) syndrome using multiparameter flow cytometry including FLAER.
Sandra M. Frey, Friederike Poppenborg, Ute Schmücker, Nicole Preising, H. Christian Reinhardt, Alexander Röth, Ferras Alashkar   +6 more
wiley   +1 more source

Paroxysmal nocturnal hemoglobinuria revealed by atypical thromboses: A case of cerebral, hepatic, and portal vein involvement leading to cirrhosis

Radiology Case Reports
Paroxysmal nocturnal hemoglobinuria is a rare clonal stem cell disorder characterized by ongoing intravascular hemolysis, frequent bone marrow dysfunction, and a markedly elevated risk of thrombosis.
Majda Malghi, MD, Mehdi Zouaoui, MD, Ibtissam Bamaarouf, MD, Sara Messaoudi, MD, Youssef Hnach, MD, Mbarek Azouaoui, MD, Nourdin Aqodad, MD   +6 more
doaj   +1 more source