Results 91 to 100 of about 15,382 (226)

Meningococcal Sepsis in Patient with Paroxysmal Nocturnal Hemoglobinuria during Pegcetacoplan Therapy

Emerging Infectious Diseases
Complement C5 inhibitors bring an increased risk for Neisseria infections. A novel complement C3 inhibitor, pegcetacoplan, was recently approved to treat paroxysmal nocturnal hemoglobinuria, a condition commonly treated with complement C5 inhibitors. We
Leo Starck, Vuokko Nummi, Eira Poikonen, Anna-Elina Lehtinen, Pia Kivelä, Nathalie Friberg, Jukka Torvikoski, Maija Toropainen, Seppo Meri   +8 more
doaj   +1 more source

Complement Factor H‐Based Therapeutics: A Comprehensive Overview

European Journal of Immunology, Volume 56, Issue 7, July 2026.
Complement factor H is a central regulator of the complement system and thus a target for therapeutic intervention. In this review, we provide an overview of past and current factor H‐based therapeutic concepts and modalities to treat complement‐mediated diseases.
Sebastiaan M. W. R. Hamers, Emmy J. Buter, Richard B. Pouw   +2 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

Switching between complement inhibitors in paroxysmal nocturnal hemoglobinuria: Analysis of strategy, efficacy, and safety

HemaSphere, Volume 10, Issue 7, July 2026.
Abstract Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra‐orphan disease. In 2026, approved complement inhibitors (CIs) for PNH include three C5 inhibitors (C5i) and three proximal inhibitors (PIs). Clinical trials for the approved PI pegcetacoplan, iptacopan, and C5i + danicopan had clear protocols for changing from terminal to PI, extrapolated ...
Morag Griffin, Bruno Fattizzo, Jong Wook Lee, Richard J. Kelly, Roochi Trikha, Yasutaka Ueda, Jun‐ichi Nishimura, Christopher J. Patriquin, Alexander Röth, Petra Muus, Jens Panse, Miguel Gómez Álvarez, Alexandra Pike, Talha Munir, Shreyans Gandhi, Austin Kulasekararaj   +15 more
wiley   +1 more source

Secondary myelodysplastic syndrome and leukemia in acquired aplastic anemia and paroxysmal nocturnal hemoglobinuria.

Blood, 2020
Acquired aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are pathogenically related non-malignant bone marrow failure disorders linked to T-cell mediated autoimmunity and associated with an increased risk of secondary myelodysplastic ...
Lova Sun, D. Babushok
semanticscholar   +1 more source

Reconceptualizing Aplastic Anemia—Seed, Worm, Soil

Journal of Clinical Laboratory Analysis, Volume 40, Issue 14, July 2026.
Aplastic anemia (AA) encompasses a group of hematological syndromes often misdiagnosed, resulting in a decrease in the overall blood cell count and representing a form of bone marrow failure. We reinterpret AA based on the “seed, worm, and soil” doctrine.
Xintong Xu, Zining Wang, Jiaming Hu, Chunyan Liu, Rong Fu   +4 more
wiley   +1 more source

Herb-induced acute bone marrow intoxication and interstitial nephritis superimposing glomerular C1q deposition in a patient with paroxysmal nocturnal hemoglobinuria

Saudi Journal of Kidney Diseases and Transplantation, 2015
Paroxysmal nocturnal hemoglobinuria is a rare disease of the red blood cell membrane that renders it lyzable by the complement system, leading to chronic intravascular hemolysis. Renal hemosiderosis is a well-known complication of intravascular hemolytic
Deena T Boqari, Shatha Al Faraj, Maria Arafah, Nourah Aloudah, Khalid S Alkhairy, Ahmed Alsuhaibani, Khaled O Alsaad   +6 more
doaj   +1 more source

Characterization of breakthrough hemolysis events observed in the phase III randomized studies of ravulizumab versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria

Haematologica, 2020
Eculizumab is first-line treatment for paroxysmal nocturnal hemoglobinuria (PNH); however, approximately 11-27% of patients may experience breakthrough hemolysis (BTH) on approved doses of eculizumab.
R. Brodsky, R. D. de Latour, S. Rottinghaus, A. Röth, A. Risitano, I. Weitz, P. Hillmen, J. Maciejewski, J. Szer, J. W. Lee, A. Kulasekararaj, L. Volles, A. Damokosh, S. Ortiz, L. Shafner, Peng Liu, A. Hill, H. Schrezenmeier   +17 more
semanticscholar   +1 more source

Concurrent Loss of PIGA and ZRSR2 in a Patient With Paroxysmal Nocturnal Hemoglobinuria and Myelodysplastic Neoplasm

International Journal of Laboratory Hematology, EarlyView.
Fatma AlBulushi, Eric McGinnis
wiley   +1 more source

Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony-stimulating factor: a Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation

Haematologica, 2020
This follow-up study of a randomized, prospective trial included 192 patients with newly diagnosed severe aplastic anemia receiving antithymoglobulin and cyclosporine, with or without granulocyte colony-stimulating factor (G-CSF).
André Tichelli, Régis Peffault de Latour, Jakob Passweg, Cora Knol-Bout, Gérard Socié, Judith Marsh, Hubert Schrezenmeier, Britta Höchsmann, Andrea Bacigalupo, Sujith Samarasinghe, Alicia Rovó, Austin Kulasekararaj, Alexander Röth, Dirk-Jan Eikema, Paul Bosman, Peter Bader, Antonio Risitano, Carlo Dufour   +17 more
doaj   +1 more source