Results 41 to 50 of about 2,525 (118)

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

Expert Consensus on the Diagnosis and Monitoring of Paroxysmal Nocturnal Hemoglobinuria (PNH): An Algorithmic Approach in an Era of New Treatments

European Journal of Haematology, EarlyView.
ABSTRACT Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disorder caused by a defect of glycosylphosphatidyl‐anchored proteins, leading to an uncontrolled complement‐mediated hemolysis. The advent of complement inhibitors in clinical practice radically changed patients' outcomes and survival.
Bruno Fattizzo, Monia Marchetti, Elisa Cannizzo, Arianna Gatti, Massimo Geuna, Anna Paola Iori, Maddalena Raia, Simona Sica, Francesco Buccisano   +8 more
wiley   +1 more source

Australian consensus recommendations for the management of increased meningococcal infection risk in adults with neurological diseases treated with complement inhibitors

Internal Medicine Journal, EarlyView.
Abstract Complement inhibitor therapy carries a risk of serious infections, including meningococcal disease. Here we provide evidence‐based recommendations and expert consensus for immunisation and prophylactic treatment of patients receiving, or planning to receive, complement inhibitors for neurological conditions in the Australian setting.
Katherine A. Buzzard, Michael Barnett, Heidi Beadnall, Mirasol Forcadela, Lesley‐Ann Hall, Victoria G. Hall, Todd Hardy, Danny Hsu, Paul Kinsella, Mastura Monif, Sudarshini Ramanathan, Stephen W. Reddel, Sean Riminton, Douglas F. Johnson   +13 more
wiley   +1 more source

Positive impact of eculizumab therapy on surgery for Budd- Chiari syndrome in a patient with paroxysmal nocturnal hemoglobinuria and a longterm history of thrombosis

Hematology Reports, 2016
Paroxysmal nocturnal hemoglobinuria (PNH) is associated with severe end-organ damage and a high risk of thrombosis. Budd- Chiari syndrome, which develops after thrombotic occlusion of major hepatic blood vessels, is relatively common in PNH and has been ...
Silvia De-la-Iglesia, Hugo Luzardo, Angelina Lemes, Melissa Torres, Maria Teresa Gómez-Casares, Naylen Cruz, Teresa Molero   +6 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

Breakthrough Hemolysis in Paroxysmal Nocturnal Hemoglobinuria: Mechanistic Insights and Management Strategies

Transfusion, EarlyView.
Abstract Background Paroxysmal nocturnal hemoglobinuria (PNH) is a clonal hematopoietic stem cell disorder caused by somatic mutations in the PIGA gene, resulting in loss of glycosylphosphatidylinositol (GPI)‐anchored proteins, including the complement regulatory proteins, CD55 and CD59.
Ganesh Raman, Meghana Vivek, Rida Hasan, Siobán Keel, Laura Connelly‐Smith   +4 more
wiley   +1 more source

Eltrombopag Added to Standard Immunosuppressive Treatment as Front‐Line Therapy for Severe Aplastic Anemia: Long‐Term Outcomes of the Phase‐3 Randomized Superiority EBMT‐SAAWP RACE Study

American Journal of Hematology, Volume 101, Issue 10, Page 2520-2532, October 2026.
ABSTRACT The RACE study (NCT02009747) compared horse antithymocyte globulin (hATG) plus cyclosporine A (CsA) ± eltrombopag as initial immunosuppressive treatment (IST) for severe aplastic anemia. Here we report the final 2‐year analysis of this prospective randomized phase III study.
Antonio M. Risitano, Simona Iacobelli, Austin Kulasekararaj, Marleen van Os, Sofie R. Terwel, Joe Tuffnell, Brian Piepenbroek, Morag Griffin, Constantijn J. M. Halkes, Christian Recher, Fiorenza Barraco, Edouard Forcade, Juan Carlos Vallejo, Beatrice Drexler, Jean‐Baptiste Mear, Roochi Trikha, Shreyans Gandhi, Anna Maria Raiola, L. G. M. Daenen, Marco R. de Groot, Etienne Daguindau, Erfan Nur, Wilma Barcellini, Nigel H. Russell, Louis Terriou, Anna Paola Iori, Walter Barberi, Anna Sureda, Isabel Sánchez‐Ortega, Blanca Xicoy, Isidro Jarque, James Cavenagh, Flore Sicre de Fontbrune, Camilla Frieri, Talha Munir, Jennifer M. L. Tjon, Suzanne Tavitian, Aline Praire, Laurence Clement, Florence Rabian, Luana Marano, Anita Hill, Elena Palmisani, Petra Muus, Serena Marotta, Fabiana Cacace, Marica Laurino, Jakob R. Passweg, Gérard Socié, Ghulam J. Mufti, Carlo Dufour, Régis Peffault de Latour, Severe Aplastic Anaemia Working Party of the EBMT   +52 more
wiley   +1 more source

Reversible Eltrombopag‐Associated Bone Marrow Fibrosis With Hematologic Recovery After Drug Withdrawal in ASXL1‐Mutated Hypoplastic Myelodysplastic Syndrome: A Case Report

eJHaem, Volume 7, Issue 5, October 2026.
ABSTRACT Hypoplastic myelodysplastic syndrome (MDS) often overlaps clinically with aplastic anemia and frequently involves clonal hematopoiesis. Thrombopoietin receptor agonists (TPO‐RAs), including eltrombopag, are increasingly used to treat cytopenias in hypoplastic marrow failure syndromes; however, their long‐term effects on clonal dynamics and ...
Kyoko Yoshihara, Ikuo Matsuda, Yasuhito Nannya, Mami Samori, Naru Tomigaki, Saki Takahashi, Chisato Ohe, Satoshi Yoshihara   +7 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, Volume 117, Issue 4, Page 799-813, October 2026.
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

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

International Journal of Laboratory Hematology, Volume 48, Issue 5, Page 1000-1007, October 2026.
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