Results 61 to 70 of about 15,382 (226)

Complement inhibition in paroxysmal nocturnal hemoglobinuria: From biology to therapy

International Journal of Laboratory Hematology
Complement inhibitors are the mainstay of paroxysmal nocturnal hemoglobinuria (PNH) treatment. The anti‐C5 monoclonal antibody eculizumab was the first treatment to improve hemolysis, thrombotic risk, and survival in PNH although at the price of a life ...
Francesco Versino, B. Fattizzo
semanticscholar   +1 more source

Hemolytic paroxysmal nocturnal hemoglobinuria: 20 years of medical progress.

Seminars in hematology (Print), 2022
Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by intravascular hemolysis, thrombosis and bone marrow failure. Prior to the availability of specific therapy, PNH led to the death of around half of affected individuals, mainly through ...
R. Peffault de Latour, K. Hosokawa, A. Risitano   +2 more
semanticscholar   +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

Pregnancy in Classical Paroxysmal Nocturnal Hemoglobinuria and Aplastic Anemia–Paroxysmal Nocturnal Hemoglobinuria: A High-Risk Constellation

Frontiers in Medicine, 2020
Pregnancies in paroxysmal nocturnal hemoglobinuria (PNH) are associated with increased morbidity and mortality. Retrospective studies suggest that outcome has improved with the advent of the complement inhibitor eculizumab.
Ferras Alashkar, Fuat H. Saner, Colin Vance, Ute Schmücker, Dörte Herich-Terhürne, Ulrich Dührsen, Angela Köninger, Alexander Röth   +7 more
doaj   +1 more source

Expert consensus on the management of pharmacodynamic breakthrough-hemolysis in treated paroxysmal nocturnal hemoglobinuria

Hematology
Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, non-malignant hematologic disease characterized by complement-mediated hemolysis (with or without hemoglobinuria), fatigue, increased susceptibility to thrombosis, and bone ...
D. Dingli, Carlos De Castro III, Jamie L. Koprivnikar, A. Kulasekararaj, Jaroslaw P. Maciejewski, B. Mulherin, J. Panse, V. Pullarkat, Alexander Röth, J. Shammo, L. Terriou, Ilene Weitz, Irina Yermilov, S. Gibbs, M.S. Broder, David O. Beenhouwer, D. Kuter   +16 more
semanticscholar   +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

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

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

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

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