Results 51 to 60 of about 32,733 (212)

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

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

Hemoglobinúria paroxística noturna e gravidez Paroxysmal nocturnal hemoglobinuria in pregnancy

Revista Brasileira de Ginecologia e Obstetrícia, 2004
A hemoglobinúria paroxística noturna é doença rara, causada por mutação adquirida de um gene no sistema hematopoético com 16-18% dos casos diagnosticados durante a gravidez.
Marcelo Luís Nomura, Fernanda Garanhani de Castro Surita, Mary Angela Parpinelli, Egle Cristina Couto Carvalho, Renato Passini Júnior   +4 more
doaj   +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

Paroxysmal Nocturnal Hemoglobinuria: Biology and Treatment

, 2023
Paroxysmal nocturnal hemoglobinuria (PNH) is a nonmalignant clonal hematopoietic disorder characterized by the lack of glycosylphosphatidylinositol-anchored proteins (GPI-APs) as a consequence of somatic mutations in the phosphatidylinositol glycan ...
Valeria Visconte, Carlos Bravo-Perez, Nakisha D. Williams, Luca Guarnera   +3 more
core   +1 more source

Off-Pump Coronary Revascularization Using Bilateral Internal Thoracic Arteries in A Patient with Paroxysmal Nocturnal Hemoglobinuria: A Case Report

Brazilian Journal of Cardiovascular Surgery
Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-orphan disease. We report the first case in the literature of Off-Pump Coronary Revascularization Using Bilateral Internal Thoracic Arteries in a patient with paroxysmal nocturnal hemoglobinuria. A 36-
Juan Mariano Vrancic, Manuel Roque Cervetti, Julián Benavides, Daniel Navia   +3 more
doaj   +1 more source

The small population of PIG-A mutant cells in myelodysplastic syndromes do not arise from multipotent hematopoietic stem cells

Haematologica, 2012
Background Patients with paroxysmal nocturnal hemoglobinuria harbor clonal glycosylphosphatidylinositol-anchor deficient cells arising from a multipotent hematopoietic stem cell acquiring a PIG-A mutation.
Jeffrey J. Pu, Rong Hu, Galina L. Mukhina, Hetty E. Carraway, Michael A. McDevitt, Robert A. Brodsky   +5 more
doaj   +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

Evans' syndrome in paroxysmal nocturnal hemoglobinuria.

, 1985
We describe the first case of paroxysmal nocturnal hemoglobinuria with Evans' syndrome. The immunohematological studies of this patient, a 27-year-old man, revealed the presence of red cell and platelet autoantibodies, related to an episode of anemia and
GIRELLI, Gabriella, Conti L, Iurlo A, Gandolfo GM   +3 more
core   +1 more source