Current and Future Pharmacologic Complement Inhibitors
The availability of anticomplement therapies has been a major achievement for medicine in the last decade. Indeed, eculizumab has changed the treatment paradigm of paroxysmal nocturnal hemoglobinuria and atypical hemolytic…
Thrombotic Microangiopathy
Thrombotic microangiopathies (TMA) such as atypical hemolytic uremic syndrome (aHUS) have evolved from rare, fulminant childhood afflictions to uncommon diseases with acute and chronic phases involving both children and adults….
Shiga Toxin Associated Hemolytic Uremic Syndrome
Shiga toxin associated hemolytic uremic syndrome (Stx HUS), a thrombotic microangiopathy, is the most common cause of pediatric acute kidney injury but has no direct treatment. A better understanding of…
Congenital CD59 Deficiency
The severe clinical symptoms of inherited CD59 deficiency confirm the importance of CD59 as essential complement regulatory protein for protection of cells against complement attack, in particular protection of hematopoietic…
Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria is manifests with a chronic hemolytic anemia from uncontrolled complement activation, a propensity for thrombosis and marrow failure. The hemolysis is largely mediated by the alternative pathway…
Ultralarge Von Willebrand Factor–Induced Platelet Clumping and Activation of the Alternative Complement Pathway in Thrombotic Thrombocytopenic Purpura and the Hemolytic-Uremic Syndromes
The molecular linkage between ultralarge (UL) von Willebrand factor (VWF) multimers and the alternative complement pathway (AP) has recently been described. Endothelial cell (EC)-secreted and anchored ULVWF multimers (in long…
Paroxysmal Cold Hemoglobinuria
Paroxysmal 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…
Cold Agglutinin-Mediated Autoimmune Hemolytic Anemia
Cold antibody types account for about 25% of autoimmune hemolytic anemias. Primary chronic cold agglutinin disease (CAD) is characterized by a clonal lymphoproliferative disorder. Secondary cold agglutinin syndrome (CAS) complicates…
Hemolysis from ABO Incompatibility
ABO incompatibility of red blood cells leads to brisk complement-mediated lysis, particularly in the setting of red cell transfusion. The ABO blood group is the most clinically significant blood group…
