Results 21 to 30 of about 15,382 (226)

Baseline characteristics and disease burden in patients in the International Paroxysmal Nocturnal Hemoglobinuria Registry

Haematologica, 2014
Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease associated with hemolytic anemia, bone marrow failure, thrombosis, and, frequently, poor quality of life.
Hubert Schrezenmeier, Petra Muus, Gérard Socié, Jeffrey Szer, Alvaro Urbano-Ispizua, Jaroslaw P. Maciejewski, Robert A. Brodsky, Monica Bessler, Yuzuru Kanakura, Wendell Rosse, Gus Khursigara, Camille Bedrosian, Peter Hillmen   +12 more
doaj   +1 more source

Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label proof-of-concept study

Blood Advances, 2022
Iptacopan (LNP023) is a novel, oral selective inhibitor of complement factor B under clinical development for paroxysmal nocturnal hemoglobinuria (PNH).
J. Jang, Lily Wong Lee Lee, B. Ko, S. Yoon, Katie Li, Irina Baltcheva, P. Nidamarthy, Raghav Chawla, G. Junge, E. Yap   +9 more
semanticscholar   +1 more source

Paroxysmal Nocturnal Hemoglobinuria [PDF]

Blood, 1950
Abstract 1. Paroxysmal noctural hemoglobinuria is believed to be an acquired disease of the hematopoietic system in which abnormal red cells, white cells, and platelets are produced. The lesion of the cells probably involves the stromal proteins in such a fashion that they are susceptible to the proteolytic effect of a sytem of normal ...
openaire   +2 more sources

The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey

Annals of Hematology, 2022
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening disease with symptoms of hemolysis and thrombosis. Current therapies for this complement-mediated disease rely predominantly on inhibition of the C5 complement protein.
D. Dingli, J. E. Matos, K. Lehrhaupt, S. Krishnan, M. Yeh, J. Fishman, S. Sarda, S. Baver   +7 more
semanticscholar   +1 more source

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

Paroxysmal Nocturnal Hemoglobinuria

Current Opinion in Hematology, 2012
The aim is to report on recent observations related to the natural history of paroxysmal nocturnal hemoglobinuria (PNH) and to review new therapeutic strategies for controlling the hemolysis of PNH.This review focuses on studies designed to characterize the long-term outcome of patients with PNH treated with eculizumab and to define the relationship ...
  +9 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

How I Treat Paroxysmal nocturnal hemoglobinuria.

Blood, 2021
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, clonal, complement-mediated hemolytic anemia with protean manifestations. PNH can present as a hemolytic anemia, a form of bone marrow failure, a thrombophilia, or any combination of the above ...
R. Brodsky
semanticscholar   +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

Long‐term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2‐year results from two pivotal phase 3 studies

European Journal of Haematology, 2022
The complement component 5 (C5) inhibitor ravulizumab demonstrated non‐inferiority to eculizumab following 26 weeks of treatment in complement inhibitor‐naïve and complement inhibitor‐experienced patients with paroxysmal nocturnal hemoglobinuria (PNH ...
A. Kulasekararaj, M. Griffin, Saskia M C Langemeijer, K. Usuki, A. Kulagin, M. Ogawa, Ji Yu, A. Mujeebuddin, J. Nishimura, J. W. Lee, R. Peffault de Latour   +10 more
semanticscholar   +1 more source