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A History of Research of PNH: Defining a Disease
Pages 1-8
This volume reviews the fundamental understanding of this potentially life-threatening disease and the advances in treatment that have been achieved with the use of the monoclonal antibody eculizumab. Although the PIGA gene has been known for many years, the mechanism of clonal dominance in paroxysmal nocturnal hemoglobinuria is still largely unknown. This book, Paroxysmal Nocturnal Hemoglobinuria, discusses the direction of continuing research in this area, as well as the potential for the development of management guidelines. It serves as a valuable source of information for both basic scientists and physicians, especially immunologists targeting GPI-anchored proteins and complements, and hematologists specializing in bone marrow failure.
A History of Research of PNH: Defining a Disease
Pages 1-8
Synthesis, Genetics, and Congenital Diseases of GPI-Anchored Proteins
Pages 11-54
Animal Models of Paroxysmal Nocturnal Hemoglobinuria
Pages 55-65
Complement and PNH
Pages 67-95
Epidemiology in PNH: The PNH Global Registry
Pages 99-107
Epidemiology in PNH: Clinical Epidemiological Studies in Korea
Pages 109-120
Haemolysis in PNH: Depletion of Nitric Oxide
Pages 121-135
Bone Marrow Failure in PNH
Pages 137-151
Thrombophilia in PNH
Pages 153-172
Diagnosis and Classification of PNH
Pages 173-183
Clinical Significance of a Small Population of Glycosylphosphatidylinositol-Anchored Membrane Proteins (GPI-APs)-Deficient Cells in the Management of Bone Marrow Failure
Pages 185-195
Clonal Origin and Clonal Selection in PNH
Pages 197-213
Pathogenesis of Clonal Dominance in PNH: Selection Mechanisms in PNH
Pages 215-227
Pathogenesis of Clonal Dominance in PNH: Growth Advantage in PNH
Pages 229-251
Clinical Management in PNH
Pages 253-269
Clinical Effects of Eculizumab in PNH
Pages 271-282
Clinical Effects of Eculizumab in PNH: Extravascular Hemolysis After Eculizumab Treatment
Pages 283-295
Clinical Effects of Eculizumab in PNH: Poor Responders to Eculizumab
Pages 297-306
Hematopoietic Stem Cell Transplantation in PNH
Pages 307-317
Future Strategies of Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria
Pages 319-346
Pregnancy in Paroxysmal Nocturnal Hemoglobinuria
Pages 347-358
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