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Long-term effectiveness and safety of ravulizumab in paroxysmal nocturnal hemoglobinuria: a plain language summary. [PDF]
Arrais C, Carnelutto N, Reyes J.
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Paroxysmal Nocturnal Hemoglobinuria in Children
Paroxysmal nocturnal hemoglobinuria (PNH), an acquired hematologic disorder characterized by intravascular hemolysis, nocturnal hemoglobinuria, thrombotic events, serious infections, and bone marrow failure, is very rare in children. PNH is caused by a somatic mutation of the phosphatidylinositol glycan (GPI) complementation class A (PIGA) gene ...
Heuvel-Eibrink, M.M. (Marry) van den +1 more
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Pregnancy and paroxysmal nocturnal hemoglobinuria
A patient is described who developed symptoms of paroxysmal nocturnal hemoglobinuria (PNH) in her first pregnancy. This was uneventful except for a spontaneous preterm delivery. The second pregnancy was complicated by severe anemia and a hemolytic crisis with Budd-Chiari syndrome at 31 weeks' amenorrhoea.
Bais, J. +3 more
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Paroxysmal Cold Hemoglobinuria
Hematology/Oncology Clinics of North America, 2015Paroxysmal cold hemoglobinuria is a rare cause of autoimmune hemolytic anemia predominantly seen as an acute form in young children after viral illnesses and in a chronic form in some hematological malignancies and tertiary syphilis. It is a complement mediated intravascular hemolytic anemia associated with a biphasic antibody against the P antigen on ...
Satish, Shanbhag, Jerry, Spivak
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Pegcetacoplan for paroxysmal nocturnal hemoglobinuria
Blood, 2022AbstractApproximately a third of patients with paroxysmal nocturnal hemoglobinuria (PNH) remain transfusion dependent or have symptomatic anemia despite treatment with a C5 inhibitor. Pegcetacoplan inhibits complement proximally at the level of C3 and is highly effective in treating persistent anemia resulting from C3-mediated extravascular hemolysis ...
Gloria F. Gerber, Robert A. Brodsky
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Paroxysmal nocturnal hemoglobinuria
Hématologie, 2013Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder of hematopoietic stem cells. PNH is related to a somatic mutation in the phosphatidylinositol glycan class A (PIG-A), X-linked gene, responsible for a deficiency in glycosylphosphatidylinositol-anchored proteins (GPI-AP).
R, Peffault de Latour +2 more
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