Results 51 to 60 of about 15,382 (226)

Phase 3 randomized COMMODORE 1 trial: Crovalimab versus eculizumab in complement inhibitor‐experienced patients with paroxysmal nocturnal hemoglobinuria

American journal of hematology/oncology
Crovalimab, a novel C5 inhibitor, allows for low‐volume, every‐4‐ week, subcutaneous self‐administration. COMMODORE 1 (NCT04432584) is a phase 3, global, randomized trial evaluating crovalimab versus eculizumab in C5 inhibitor‐experienced patients with ...
P. Scheinberg, D. Clé, Jin Seok Kim, E. Nur, M. Yenerel, W. Barcellini, Debra Bonito, V. Giai, M. Hus, Yoojin Lee, Cristina Barrenetxea Lekue, J. Panse, Yasutaka Ueda, Simon Buatois, Brittany Gentile, A. Kiialainen, Himika Patel, S. Sreckovic, Marianne Uguen, John Edwards, Zsolt Nagy, A. Kulasekararaj   +21 more
semanticscholar   +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

Baseline clinical characteristics and disease burden in patients with paroxysmal nocturnal hemoglobinuria (PNH): updated analysis from the International PNH Registry

Annals of Hematology, 2020
The International Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry (NCT01374360) was initiated to optimize patient management by collecting data regarding disease burden, progression, and clinical outcomes.
H. Schrezenmeier, A. Röth, David J Araten, Y. Kanakura, L. Larratt, J. Shammo, A. Wilson, Gilda Shayan, J. Maciejewski   +8 more
semanticscholar   +1 more source

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

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

Insights Into the Emergence of Paroxysmal Nocturnal Hemoglobinuria

Frontiers in Immunology, 2022
Paroxysmal Nocturnal Hemoglobinuria (PNH) is a disease as simple as it is complex. PNH patients develop somatic loss-of-function mutations in phosphatidylinositol N-acetylglucosaminyltransferase subunit A gene (PIGA), required for the biosynthesis of ...
Melissa A. Colden, Sushant Kumar, Bolormaa Munkhbileg, D. Babushok   +3 more
semanticscholar   +1 more source

Paroxysmal Nocturnal Hemoglobinuria [PDF]

Blood, 1967
Abstract 1. Crude bovine thrombin contains most, if not all, the proteins of bovine serum. 2. The effect of crude bovine thrombin in enhancing the lysis of paroxysmal nocturnal hemoglobinuria erythrocytes has been shown to be an effect on the erythrocytes themselves. 3.
STUART F. BLUM, FRANK H. GARDNER, Dominica Paci   +2 more
openaire   +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, EarlyView.
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

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