Results 11 to 20 of about 82,322 (154)

Paroxysmal nocturnal hemoglobinuria and telomere length predicts response to immunosuppressive therapy in pediatric aplastic anemia [PDF]

Haematologica, 2015
Acquired aplastic anemia is an immune-mediated disease characterized by severe defects in stem cell number resulting in hypocellular marrow and peripheral blood cytopenias. Minor paroxysmal nocturnal hemoglobinuria populations and a short telomere length
Atsushi Narita, Hideki Muramatsu, Yuko Sekiya, Yusuke Okuno, Hirotoshi Sakaguchi, Nobuhiro Nishio, Nao Yoshida, Xinan Wang, Yinyan Xu, Nozomu Kawashima, Sayoko Doisaki, Asahito Hama, Yoshiyuki Takahashi, Kazuko Kudo, Hiroshi Moritake, Masao Kobayashi, Ryoji Kobayashi, Etsuro Ito, Hiromasa Yabe, Shouichi Ohga, Akira Ohara, Seiji Kojima   +21 more
doaj   +2 more sources

Paroxysmal nocturnal hemoglobinuria in systemic lupus erythematosus: a case report [PDF]

Journal of Medical Case Reports, 2011
Introduction Paroxysmal nocturnal hemoglobinuria is an acquired disorder of hemopoiesis and is characterized by recurrent episodes of intravascular hemolysis due to an increased sensitivity to complement-mediated hemolysis.
Nakamura Norio, Sugawara Toshiyuki, Shirato Ken-ichi, Kumasaka Ryuichiro, Nakamura Masayuki, Shimada Michiko, Fujita Takeshi, Murakami Reiichi, Shimaya Yuko, Osawa Hiroshi, Yamabe Hideaki, Okumura Ken   +11 more
doaj   +3 more sources

Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria [PDF]

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   +3 more sources

Frequency of paroxysmal nocturnal hemoglobinuria in patients attended in Belém, Pará, Brazil [PDF]

Revista Brasileira de Hematologia e Hemoterapia, 2011
BACKGROUND: Paroxysmal nocturnal hemoglobinuria is a hematological disease with complex physiopathology. It is genetically characterized by a somatic mutation in the PIG-A gene (phosphatidylinositol glycan anchor biosynthesis, class A), in which the best
Lacy Cardoso de Brito Junior, Maria do Socorro de Oliveira Cardoso, Euzamar Gaby Rocha, Herika Anijar, Mariana Cunha, João Carlos Pina Saraiva   +5 more
doaj   +2 more sources

Paroxysmal nocturnal hemoglobinuria clone in 103 Brazilian patients: diagnosis and classification [PDF]

Revista Brasileira de Hematologia e Hemoterapia, 2015
Background: Paroxysmal nocturnal hemoglobinuria is an acquired chronic hemolytic ane- mia, which often manifests as peripheral blood cytopenias and thrombosis. Objective: The aim of this study is to describe a Brazilian population of paroxysmal nocturnal
Ana Paula de Azambuja, Mariester Malvezzi, Marco Antonio Bitencourt, Michel Michels Oliveira, Larissa Alessandra Medeiros, Ricardo Pasquini   +5 more
doaj   +2 more sources

Loss of expression of neutrophil proteinase-3: a factor contributing to thrombotic risk in paroxysmal nocturnal hemoglobinuria

Haematologica, 2011
Background A deficiency of specific glycosylphosphatidyl inositol-anchored proteins in paroxysmal nocturnal hemoglobinuria may be responsible for most of the clinical features of this disease, but some functional consequences may be indirect. For example,
Anna M. Jankowska, Hadrian Szpurka, Mark Calabro, Sanjay Mohan, Andrew E. Schade, Michael Clemente, Roy L. Silverstein, Jaroslaw P. Maciejewski   +7 more
doaj   +2 more sources

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   +2 more sources

Small-molecule factor D inhibitors selectively block the alternative pathway of complement in paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome [PDF]

Haematologica, 2017
Paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome are diseases of excess activation of the alternative pathway of complement that are treated with eculizumab, a humanized monoclonal antibody against the terminal complement ...
Xuan Yuan, Eleni Gavriilaki, Jane A. Thanassi, Guangwei Yang, Andrea C. Baines, Steven D. Podos, Yongqing Huang, Mingjun Huang, Robert A. Brodsky   +8 more
doaj   +2 more sources

Population Pharmacokinetic and Pharmacokinetic/Pharmacodynamic Analyses of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria

Drugs in R&D
Background and Objective Paroxysmal nocturnal hemoglobinuria is a rare blood disorder characterized by life-threatening hemolysis and thrombosis. Complement C5 inhibitor therapy improves symptoms and life prognosis; however, it can result in insufficient
Ryan L. Crass, Brandon Smith, Sven Adriaens, Sunny Chapel, Grant Langdon   +4 more
doaj   +2 more sources

A mutation in a functional Sp1 binding site of the telomerase RNA gene (hTERC) promoter in a patient with Paroxysmal Nocturnal Haemoglobinuria [PDF]

, 2004
Background<br/><br/> Mutations in the gene coding for the RNA component of telomerase, hTERC, have been found in autosomal dominant dyskeratosis congenita (DC) and aplastic anemia.
Ulku, B., Bessler, M., Bilsland, A., Mason, Philip J, Vulliamy, T., Serakinci, Nedime, Dokal, I., Ar, C., Ar, C, Marrone, Anna, Dokal, Inderjeet, Ulku, B, Bessler, Monica, Erzik, C, Serakinci, N, Serakinci, N., Marrone, A, Zhao, Jiangqin, Zhao, J, Dokal, I, Bilsland, A, Mason, PJ, Bilsland, Alan, Ulku, Birsen, Keith, W.N., Bessler, M, Vulliamy, T, Vulliamy, Tom, Keith, W Nicol, Erzik, Can, Marrone, A., Erzik, C., Zhao, J., Mason, P.J., Ar, Cem, Keith, WN, Tom Vulliamy, Alan Bilsland, W Nicol Keith, Jiangqin Zhao, Monica Bessler, Anna Marrone, Inderjeet Dokal, Nedime Serakinci, Philip J Mason, Cem Ar, Can Erzik, Birsen Ulku   +47 more
core   +2 more sources