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

IF 3.4 3区 医学 Q2 HEMATOLOGY
Therapeutic Advances in Hematology Pub Date : 2024-12-23 eCollection Date: 2024-01-01 DOI:10.1177/20406207241307500
Julia J Shi, Yusuf M Ozcan, Carlos I Ayala Santos, Hetalkumari Patel, Jamile Shammo, Taha Bat
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引用次数: 0

Abstract

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.

补体抑制剂和调节剂时代阵发性夜间血红蛋白尿的现状。
阵发性夜间血红蛋白尿(PNH)是一种罕见的血液疾病,是由磷脂酰肌醇聚糖a类突变引起的,通过补体抑制导致红细胞溶血。首个治疗PNH的药物eculizumab于2007年获得FDA批准。从那时起,出现了许多新的PNH治疗方案。这篇重要的综述将审查美国市场上所有可用于PNH的药物,重点介绍几种正在开发的主要药物,并讨论每种选择的风险和治疗考虑。它不打算解决PNH克隆动力学,疾病表现,或何时开始治疗的讨论。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
4.30
自引率
0.00%
发文量
54
审稿时长
7 weeks
期刊介绍: Therapeutic Advances in Hematology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of hematology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in hematology, providing a forum in print and online for publishing the highest quality articles in this area.
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