Results 31 to 40 of about 7,137 (182)

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

Pegcetacoplan Delivers Real‐World Therapeutic Benefits and Reduces Disease Burden for Patients With Paroxysmal Nocturnal Haemoglobinuria: A Systematic Literature Review of Pegcetacoplan Real‐World Clinical and Patient‐Reported Outcomes

European Journal of Haematology, EarlyView.
ABSTRACT Aims Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra‐rare, acquired, non‐malignant haematological disorder that, if left untreated, can lead to significant morbidity. This systematic literature review (SLR) summarized real‐world evidence (RWE) for pegcetacoplan, a complement 3/3b inhibitor (C3i) available since 2021.
Juan Carlos Vallejo Llamas, Koo Wilson, Zalmai Hakimi, Vikte Lionikaite, Elisabetta Metafuni   +4 more
wiley   +1 more source

Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria

Haematologica, 2012
Background In the era of eculizumab, identifying patients with paroxysmal nocturnal hemoglobinuria who may benefit from allogeneic stem cell transplantation is challenging.Design and Methods We describe the characteristics and overall survival of 211 ...
Régis Peffault de Latour, Hubert Schrezenmeier, Andrea Bacigalupo, Didier Blaise, Carmino A. de Souza, Stephane Vigouroux, Roelf Willemze, Louis Terriou, Andre Tichelli, Mohamad Mohty, Sophie de Guibert, Judith C. Marsh, Jakob Passweg, Jean Yves Mary, Gerard Socié   +14 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

Hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria: long-term results of a retrospective study on behalf of the Gruppo Italiano Trapianto Midollo Osseo (GITMO)

Haematologica, 2010
Background Paroxysmal nocturnal hemoglobinuria is an acquired clonal disorder of the hemopoietic stem cells for which the only curative treatment is allogeneic hematopoietic stem cell transplantation.Design and Methods The aim of this retrospective study
Stella Santarone, Andrea Bacigalupo, Antonio M. Risitano, Elena Tagliaferri, Erminia Di Bartolomeo, Anna Paola Iori, Alessandro Rambaldi, Emanuele Angelucci, Alessandra Spagnoli, Federico Papineschi, Stefania Tamiazzo, Marta Di Nicola, Paolo Di Bartolomeo   +12 more
doaj   +1 more source

The kidneys in paroxysmal nocturnal hemoglobinuria [PDF]

Blood, 1981
Long-term study of 21 PNH patients revealed an unexpectedly high incidence of functional and anatomic renal abnormalities. Most patients demonstrated varying degrees of hematuria and proteinuria distinct from hemoglobinuria. Evaluation of renal function revealed hyposthenuria, abnormal tubular function, and declining creatinine clearance ...
D A, Clark, S A, Butler, V, Braren, R C, Hartmann, D E, Jenkins   +4 more
openaire   +3 more sources

Assessment of the Performance of Siemens Scopio Digital Morphology on Bone Marrow Aspirates in Onco‐Hematology

International Journal of Laboratory Hematology, EarlyView.
ABSTRACT Objectives Digital morphology (DM) systems assisted by artificial intelligence are increasingly being introduced into hematology laboratories; however, data on their performance in routine clinical practice for bone marrow aspirates (BMA) remain limited.
Gina Zini, John Marra, Elena Rossi, Silvia Bellesi, Nicoletta Pelliccioni, Giuseppe d'Onofrio, Patrizia Chiusolo   +6 more
wiley   +1 more source

Short-term recurrent coronary artery thrombosis with acute myocardial infarction in a patient with aplastic anemia–paroxysmal nocturnal hemoglobinuria syndrome: a case report

Frontiers in Cardiovascular Medicine
BackgroundAcute myocardial infarction commonly occurs in patients with coronary artery disease, but rarely, it can develop under a hypercoagulable state.
Xue-Guo Fu, Yan-Hua Guo, Shi-Chao Wang, Wen-Quan Zhang   +3 more
doaj   +1 more source

Mapping of C5‐Blocking Monoclonal Antibodies Reveals New C5 Inhibitory Epitopes and Novel Modes of C5 Inhibition

Immunology, EarlyView.
We identify four distinct C5 inhibitory epitopes, including two novel antibodies that selectively block membrane attack complex (MAC) assembly while preserving C5 cleavage and C5a generation. These findings establish selective MAC inhibition as a new therapeutic strategy for complement‐mediated diseases.
Rebekah Sian Cooke, Kok Yung Lee, Georgina Elizabeth Menzies, Masashi Mizuno, Bryan Paul Morgan, Wioleta Milena Zelek   +5 more
wiley   +1 more source

High Proportion of PNH Type II Neutrophils Is Associated With Thrombosis in Patients Displaying a PNH Clone ≥ 1%

American Journal of Hematology, Volume 101, Issue 9, Page 2311-2328, September 2026.
ABSTRACT The clinical significance of PNH Type II white blood cells (WBCs) remains unclear. We assessed the relative percentage (rel%) of Type II neutrophils in 355 patients with a PNH clone ≥ 1% on neutrophils enrolled by 33 flow cytometry laboratories in the 5‐year French nation‐wide multicenter prospective observational study.
Orianne Wagner‐Ballon, Anne‐Catherine Lhoumeau, Anna Raimbault, Camille Debord, Jean‐Philippe Vial, Valérie Soenen, Mélanie Pannetier, Edouard Bonneville, Elsa Bera, Caroline Bret, Nicolas Chapuis, Véronique Latger‐Cannard, Caroline Mayeur‐Rousse, Véronique Harrivel, François Vergez, Marie Loosveld, Juliette Vo Xuan, Stéphanie Mathis, Gérard Socié, Flore Sicre de Fontbrune, Régis Peffault de Latour, Bernard Drenou, Agathe Debliquis, Magali Le Garff‐Tavernier, on behalf of the French PNH working group CytHem‐HPN, Thomas Boyer, Mikaël Roussel, Marie Guillon, Sabrina Bouyer, Isabelle Kittler, Franck Geneviève, Julien Guy, Sophie Brun, Claude Capron, Sébastien Lachot, Francine Garnache‐Ottou, Richard Veyrat‐Masson, Corine Brunet, Caren Brumpt, Ludovic Lhermitte. Finally, Louis Terriou, Nicolas Blin   +41 more
wiley   +1 more source