Shi Julia J, Ozcan Yusuf M, Santos Carlos I Ayala, Patel Hetalkumari, Shammo Jamile, Bat Taha
Division of Hematology and Oncology, Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, TX, USA.
University of Texas at Dallas, Richardson, TX, USA.
Ther Adv Hematol. 2024 Dec 23;15:20406207241307500. doi: 10.1177/20406207241307500. eCollection 2024.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder which is caused by mutations in phosphatidylinositol glycan class A leading to hemolysis of red blood cells via complement inhibition. The first treatment for PNH, eculizumab, was FDA approved in 2007. Since then, many new treatment options for PNH have arisen. This critical review will examine all medications available for PNH on the US market, highlight several major medications in development, and discuss the risks and treatment considerations associated with each option. It is not intended to address PNH clonal dynamics, disease presentation, or discussions on when to initiate treatment.
阵发性睡眠性血红蛋白尿症(PNH)是一种罕见的血液疾病,由磷脂酰肌醇聚糖A类基因突变引起,通过补体抑制导致红细胞溶血。PNH的首个治疗药物依库珠单抗于2007年获得美国食品药品监督管理局(FDA)批准。从那时起,出现了许多针对PNH的新治疗选择。这篇批判性综述将审视美国市场上可用于治疗PNH的所有药物,重点介绍几种正在研发的主要药物,并讨论与每种选择相关的风险和治疗注意事项。本文无意探讨PNH的克隆动力学、疾病表现或何时开始治疗的相关讨论。