Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal blood disorder that manifests with hemolytic anemia, bone marrow failure, and thrombosis. Many of the clinical manifestations of the disease result from complement-mediated intravascular hemolysis. Allogeneic bone marrow transplantation is the only curative therapy for PNH. Eculizumab, a monoclonal antibody that blocks terminal complement activation, is highly effective in reducing hemolysis, improving quality of life, and reducing the risk for thrombosis in PNH patients. Insights into the relevance of detecting PNH cells in PNH and other bone marrow failure disorders are highlighted, and indications for treating PNH patients with bone marrow transplantation and eculizumab are explored.
Publication types
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Research Support, N.I.H., Extramural
MeSH terms
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Antibodies, Monoclonal / therapeutic use*
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Antibodies, Monoclonal, Humanized
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Anticoagulants / administration & dosage
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Anticoagulants / therapeutic use
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Bacterial Vaccines / administration & dosage
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Blood Transfusion
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Bone Marrow / pathology
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Bone Marrow Transplantation
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Clone Cells / pathology
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Combined Modality Therapy
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Complement Activation / drug effects
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Complement C5 / antagonists & inhibitors
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Disease Management
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Female
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Folic Acid / administration & dosage
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Folic Acid / therapeutic use
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Hemoglobinuria, Paroxysmal / diagnosis
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Hemoglobinuria, Paroxysmal / genetics
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Hemoglobinuria, Paroxysmal / therapy*
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Humans
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Infant, Newborn
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Iron / administration & dosage
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Iron / therapeutic use
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Male
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Membrane Proteins / deficiency
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Membrane Proteins / genetics
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Neisseria / immunology
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Neisseriaceae Infections / prevention & control
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Pregnancy
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Pregnancy Complications, Hematologic / therapy
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Thrombosis / prevention & control
Substances
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Antibodies, Monoclonal
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Antibodies, Monoclonal, Humanized
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Anticoagulants
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Bacterial Vaccines
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Complement C5
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Membrane Proteins
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phosphatidylinositol glycan-class A protein
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Folic Acid
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eculizumab
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Iron