Toward complement inhibition 2.0: Next generation anticomplement agents for paroxysmal nocturnal hemoglobinuria
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DOI: 10.1002/ajh.25016
Abstract: Therapeutic complement inhibition by eculizumab has revolutionized the treatment of paroxysmal nocturnal hemoglobinuria (PNH) with a major impact on its natural history. Nevertheless, emerging unmet clinical needs may benefit from the development of novel complement… read more here.
Keywords: paroxysmal nocturnal; inhibition; nocturnal hemoglobinuria; complement inhibition ... See more keywords
Iron overload after complement inhibitor treatment of Paroxysmal Nocturnal Hemoglobinuria
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DOI: 10.1002/ajh.26187
Abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is an uncommon bone marrow disorder associated with hemolytic anemia, thrombosis, and cytopenias. Prior to 2007, treatment for PNH was limited and patients with PNH were treated with corticosteroids, anticoagulation for… read more here.
Keywords: treatment; paroxysmal nocturnal; iron overload; nocturnal hemoglobinuria ... See more keywords
Paroxysmal nocturnal hemoglobinuria: Where we stand
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DOI: 10.1002/ajh.26832
Abstract: For the last 20 years, therapy of paroxysmal nocturnal hemoglobinuria (PNH) relied—up until recently—on antibody based terminal complement inhibitionon. PNH pathophysiology—a mutational defect leading to partial or complete absence of complement‐regulatory proteins on blood cells—leads to… read more here.
Keywords: hemoglobinuria stand; complement; nocturnal hemoglobinuria; paroxysmal nocturnal ... See more keywords
Paroxysmal nocturnal hemoglobinuria: Where are we going
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DOI: 10.1002/ajh.26882
Abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare nonmalignant clonal hematological disorder that is characterized by a deficiency of the GPI‐linked complement regulators on the membrane of hematopoietic cells, which renders them susceptible to complement‐mediated damage.… read more here.
Keywords: hemoglobinuria going; complement; nocturnal hemoglobinuria; paroxysmal nocturnal ... See more keywords
Nationwide study of paroxysmal nocturnal hemoglobinuria in South Korea: paradox of eculizumab
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DOI: 10.1007/s00277-020-04133-z
Abstract: Eculizumab is effective in managing patients with paroxysmal nocturnal hemoglobinuria (PNH). In South Korea, the financial support for eculizumab therapy is extended by the National Health Insurance Services (NHIS) only to patients with high-risk PNH… read more here.
Keywords: group; paroxysmal nocturnal; eculizumab; nocturnal hemoglobinuria ... See more keywords
Battle of the clones: paroxysmal nocturnal hemoglobinuria vs myelodysplastic syndrome
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DOI: 10.1007/s00277-020-04134-y
Abstract: Dear Editor, Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disorder of hematopoietic stem cell characterized by mutations in the PIGA gene leading to complementmediated lysis [1]. Approved in 2009, eculizumab is the first specific… read more here.
Keywords: analysis; paroxysmal nocturnal; myelodysplastic syndrome; nocturnal hemoglobinuria ... See more keywords
Novel targeted C3 inhibitor pegcetacoplan for paroxysmal nocturnal hemoglobinuria
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DOI: 10.1007/s10238-022-00830-3
Abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening acquired blood disease characterized by chronic complement-mediated hemolysis and thrombosis. On May 14, 2021, the US FDA approved a new targeted C3 therapy Empaveli (pegcetacoplan), once called… read more here.
Keywords: paroxysmal nocturnal; nocturnal hemoglobinuria; novel targeted; pegcetacoplan ... See more keywords
Screening for paroxysmal nocturnal hemoglobinuria (PNH) in patients presenting with cerebral sinovenous thrombosis (CSVT): Results of a FLAER based flowcytometry study in Indian patients
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DOI: 10.1007/s11239-019-01982-y
Abstract: Patients with paroxysmal nocturnal hemoglobinuria (PNH) may present with thrombosis at unusual sites, of which cerebral sinovenous thrombosis (CSVT) is one and screening for PNH is recommended in this condition. Though many patients diagnosed with… read more here.
Keywords: flowcytometry; pnh; paroxysmal nocturnal; nocturnal hemoglobinuria ... See more keywords
Effects of eculizumab treatment on quality of life in patients with paroxysmal nocturnal hemoglobinuria in Japan
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DOI: 10.1007/s12185-018-2409-3
Abstract: In paroxysmal nocturnal hemoglobinuria (PNH), various symptoms due to intravascular hemolysis exert a negative impact on patients’ quality of life (QOL). To determine clinical factors related with improvements in QOL in PNH patients treated, we… read more here.
Keywords: eculizumab treatment; treatment; paroxysmal nocturnal; nocturnal hemoglobinuria ... See more keywords
Literature Review of Fatigue Scales and Association with Clinically Meaningful Improvements in Outcomes Among Patients With and Without Paroxysmal Nocturnal Hemoglobinuria
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DOI: 10.1007/s12325-022-02111-7
Abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by anemia and debilitating fatigue. Limited evidence characterizes the association between hemoglobin, an indicator of anemia and disease activity, and patient-reported fatigue scales. This review… read more here.
Keywords: patients without; meaningful improvements; clinically meaningful; among patients ... See more keywords
Treatment of paroxysmal nocturnal hemoglobinuria in pregnancy with eculizumab: A case report
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DOI: 10.1016/j.crwh.2021.e00294
Abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease caused by mutations in hematopoietic stem cells leading to pancytopenia and a predisposition for thromboembolic events. In pregnancy, these manifestations can be amplified, leading to increased neonatal… read more here.
Keywords: pregnancy; treatment; case; paroxysmal nocturnal ... See more keywords