Young Neal S, Meyers Gabrielle, Schrezenmeier Hubert, Hillmen Peter, Hill Anita
Washington, DC.
Semin Hematol. 2009 Jan;46(1 Suppl 1):S1-S16. doi: 10.1053/j.seminhematol.2008.11.004.
PNH is a rare clonal hematopoietic stem disorder clinically characterized by the triad of chronic complement-mediated hemolysis, thrombosis, and bone marrow failure. While median survival has improved when historical data are compared to more recent data, thrombosis, the major cause of death in PNH, is still observed in approximately 40% of patients. The symptoms associated with this disorder–including fatigue, pain, esophageal spasm, and erectile dysfunction–are often severe and disabling. While PNH may be a curiosity to the physician, it forces the majority of patients to significantly modify their lives. Transplantation represents a curative option; however, the risks associated with this option are not insignificant. Eculizumab has been shown to significantly reduce hemolysis, improve anemia, reduce transfusion requirements, and significantly improve fatigue and other QoL scores. Clearly, targeted complement inhibition by eculizumab has the promise to significantly improve the lives of patients with PNH.
阵发性睡眠性血红蛋白尿症(PNH)是一种罕见的克隆性造血干细胞疾病,临床特征为慢性补体介导的溶血、血栓形成和骨髓衰竭三联征。尽管将历史数据与近期数据相比较时,患者的中位生存期有所改善,但血栓形成作为PNH的主要死因,仍见于约40%的患者中。与该疾病相关的症状,包括疲劳、疼痛、食管痉挛和勃起功能障碍,往往很严重且使人丧失能力。虽然PNH对医生来说可能只是一种罕见病,但它迫使大多数患者对自己的生活做出重大调整。移植是一种治愈性选择;然而,该选择所带来的风险也不容小觑。已证明依库珠单抗可显著减少溶血、改善贫血、减少输血需求,并显著改善疲劳及其他生活质量评分。显然,依库珠单抗靶向抑制补体有望显著改善PNH患者的生活。