Current landscape of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitors and regulators

被引：1

作者：

Shi, Julia J. ^{[1
]}

Ozcan, Yusuf M. ^{[2
]}

Santos, Carlos I. Ayala ^{[1
]}

Patel, Hetalkumari ^{[3
]}

Shammo, Jamile ^{[4
]}

Bat, Taha ^{[1
]}

机构：

[1] Univ Texas Southwestern Med Ctr, Dept Internal Med, Div Hematol & Oncol, 5323 Harry Hines Blvd, Dallas, TX 75390 USA

[2] Univ Texas Dallas, Richardson, TX 75083 USA

[3] Univ Texas Southwestern Med Ctr, Dept Pharmacol, Dallas, TX USA

[4] Northwestern Univ, Feinberg Sch Med, Chicago, IL USA

来源：

THERAPEUTIC ADVANCES IN HEMATOLOGY | 2024年 / 15卷

关键词：

complement cascade; complement inhibitors; PNH; BREAKTHROUGH HEMOLYSIS; ECULIZUMAB; RAVULIZUMAB; MANAGEMENT; PHASE-3; PATHWAY;

D O I：

10.1177/20406207241307500

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 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.

引用

页数：12

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