How I treat paroxysmal nocturnal hemoglobinuria

被引:98
作者
Brodsky, Robert A. [1 ]
机构
[1] Johns Hopkins Med, Div Hematol, Ross Res Bldg,Rm 1025,720 Rutland Ave, Baltimore, MD 21205 USA
基金
美国国家卫生研究院;
关键词
D O I
10.1182/blood.2019003812
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, clonal, complement-mediated hemolytic anemia with protean manifestations. PNH can present as a hemolytic anemia, a form of bone marrow failure, a thrombophilia, or any combination of the above. Terminal complement inhibition is highly effective for treating intravascular hemolysis from PNH and virtually eliminates the risk of thrombosis, but is not effective for treating bone marrow failure. Here, I present a variety of clinical vignettes that highlight the clinical heterogeneity of PNH and the attributes and limitations of the 2 US Food and Drug Administration-approved C5 inhibitors (eculizumab and ravulizumab) to treat PNH. I review the concept of pharmacokinetic and pharmacodynamic breakthrough hemolysis and briefly discuss new complement inhibitors upstream of C5 that are in clinical development. Last, I discuss the rare indications for bone marrow transplantation in patients with PNH.
引用
收藏
页码:1304 / 1309
页数:6
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