Results 11 to 20 of about 12,940 (196)

Clinical Profile, Humanistic and Economic Burden of Paroxysmal Nocturnal Hemoglobinuria in Patients Treated With C5 Inhibitors. [PDF]

EJHaem
ABSTRACT Objectives To evaluate disease burden among paroxysmal nocturnal hemoglobinuria (PNH) patients prescribed C5 inhibitors (C5i). Methods Data were drawn from the Adelphi Real World PNH Disease Specific Programme, a cross‐sectional survey of physicians and PNH patients in Australia, Canada, France, Germany, Italy, Spain, the United Kingdom, and ...
Gandhi S, Radsak MP, Ueda Y, Balp MM, Wiyani A, Somenzi O, Snellman J, Taylor Y, Simons A, Panse J.   +9 more
europepmc   +2 more sources

Population PK-PD Modeling of Danicopan Add-On Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria Treated With Ravulizumab or Eculizumab. [PDF]

CPT Pharmacometrics Syst Pharmacol
ABSTRACT Danicopan is a first‐in‐class orally administered complement factor D inhibitor, approved as an add‐on therapy to ravulizumab or eculizumab for the treatment of clinically significant extravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Chen J, Yang F, Li H, Pradhan R, Ortiz S.   +4 more
europepmc   +2 more sources

Paroxysmal nocturnal hemoglobinuria [PDF]

Vnitřní lékařství, 2018
Paroxysmal nocturnal hemoglobinuria (PNH) results from a deficiency in inhibitors of activated complement. This lack leads to complement mediated intravascular hemolysis, to activation of coagulation system with increased risk of thrombotic complications and to various degree of bone marrow failure.
openaire   +3 more sources

The prothrombotic state in paroxysmal nocturnal hemoglobinuria: a multifaceted source

Haematologica, 2018
Paroxysmal nocturnal hemoglobinuria is a rare acquired hematologic disorder, the most serious complication of which is thrombosis. The increased incidence of thrombosis in paroxysmal nocturnal hemoglobinuria is still poorly understood, but unlike many ...
Barnaby Peacock-Young, Fraser L. Macrae, Darren J. Newton, Anita Hill, Robert A S Ariëns   +4 more
doaj   +1 more source

Eculizumab treatment: stochastic occurrence of C3 binding to individual PNH erythrocytes [PDF]

, 2017
C5 blockade by eculizumab prevents complement-mediated intravascular hemolysis in paroxysmal nocturnal hemoglobinuria (PNH). However, C3-bound PNH red blood cells (RBCs), arising in almost all treated patients, may undergo extravascular hemolysis ...
A Hill, A Hill, A Nicholson-Weller, A Roth, AM Risitano, AM Risitano, AM Risitano, AM Risitano, AM Risitano, Antonio M. Risitano, B Nilsson, CE Schoot van der, DJ Araten, DS Holt, E Lach-Trifilieff, E Marchiafava, G Gronowicz, GL Logue, GV Seaman, J Nishimura, J Takeda, JD Tamerius, JE May, JP Atkinson, JV Dacie, L Luzzatto, L Luzzatto, L Luzzatto, M Bessler, M Harboe, M Kono, M Loschi, M Sica, M Sica, MA Lindorfer, Maria De Angioletti, MH Holguin, Michela Sica, MJ Harder, MK Pangburn, MK Pangburn, P Hillmen, P Hillmen, P Hillmen, Patrizia Ricci, PJ Lachmann, PJ Lachmann, RJ Kelly, RL Sparrow, Rosario Notaro, RP Latour de, RP Rother, S Berentsen, SK Krishnan, T Olutogun, T Rondelli, TH Ham, Tommaso Rondelli, UM Reiss, VM Holers, VP Ferreira, WF Rosse, WF Rosse, Z Fishelson, Z Lin   +64 more
core   +2 more sources

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

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   +1 more source

Detection of paroxysmal nocturnal hemoglobinuria clones in patients with myelodysplastic syndromes and related bone marrow diseases, with emphasis on diagnostic pitfalls and caveats

Haematologica, 2009
Background The presence of paroxysmal nocturnal hemoglobinuria clones in the setting of aplastic anemia or myelodysplastic syndrome has been shown to have prognostic and therapeutic implications. However, the status of paroxysmal nocturnal hemoglobinuria
Sa A. Wang, Olga Pozdnyakova, Jeffrey L. Jorgensen, L. Jeffrey Medeiros, Dariusz Stachurski, Mary Anderson, Azra Raza, Bruce A. Woda   +7 more
doaj   +1 more source

Extravascular hemolysis and complement consumption in Paroxysmal Nocturnal Hemoglobinuria patients undergoing eculizumab treatment [PDF]

, 2017
Los datos asociados con este artículo están disponibles en: http://dx.doi.org/10.1016/j.imbio.2016.09.002.Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hemolytic anemia characterized by complement-mediated intravascular hemolysis that is ...
Anter, Jaouad, Ataúlfo Gonzalez-Fernández, Fernando, Forés, Rafael, García, Sheila Pinto, López, Alicia, López-Trascasa, Margarita, Martin Merinero, Hector, Ojeda, Emilio, Rodríguez de Córdoba, Santiago, Subías Hidalgo, Marta, Villegas, Ana   +10 more
core   +2 more sources

Paroxysmal cold hemoglobinuria

Kazan medical journal, 2021
Paroxysmal cold hemoglobinuria, first described by Dresler in 1854, is characterized by the fact that after cooling a chill sets in and the temperature rises to 39-40 with all the sensations accompanying this state. Then there are pains in the lumbar region and in the abdomen, frequent urge to urinate with cramps, nausea, vomiting.
openaire   +3 more sources

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

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   +1 more source