DeZern Amy E, Brodsky Robert A
Division of Hematologic Malignancies, Department of Oncology, The Bunting and Blaustein Cancer Research Building, 1650 Orleans Street, Room 3M87, Baltimore, MD 21287-0013, USA.
Division of Hematology, The Johns Hopkins University School of Medicine, 720 Rutland Avenue, Ross Research Building, Room 1025, Baltimore, MD 21205, USA.
Hematol Oncol Clin North Am. 2015 Jun;29(3):479-94. doi: 10.1016/j.hoc.2015.01.005. Epub 2015 Mar 7.
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 of complement. Clinical manifestations result from the lack of specific cell surface proteins, CD55 and CD59, on PNH cells. Complement inhibition by eculizumab leads to dramatic clinical improvement. While this therapeutic approach is effective, there is residual complement activity resulting from specific clinical scenarios as well as from upstream complement components that can account for suboptimal responses in some patients. Complement inhibition strategies are an area of active research.
阵发性夜间血红蛋白尿表现为因补体激活失控导致的慢性溶血性贫血、血栓形成倾向和骨髓衰竭。溶血主要由补体替代途径介导。临床表现源于阵发性夜间血红蛋白尿（PNH）细胞缺乏特异性细胞表面蛋白CD55和CD59。依库珠单抗抑制补体可带来显著的临床改善。虽然这种治疗方法有效，但特定临床情况以及上游补体成分会导致残余补体活性，这可能是一些患者反应欠佳的原因。补体抑制策略是一个活跃的研究领域。
