Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder that happens when part of your immune system attacks and damages your red blood cells and platelets. Left untreated, PNH can cause hemolytic anemia, chronic kidney disease or thrombosis (blood clots). Healthcare providers treat PNH with medication that prevents blood cell damage.

陣發性夜間血紅素尿症 （英語： paroxysmal nocturnal hemoglobinuria，縮寫為 PNH）是一種罕見、複雜且為後天造成的致命性 血液 疾病 [3]，被發現在 19世紀，大約每百萬人會有一到二人罹患此疾病 [4][5]，而確診後5年內的存活率只有約65% [6]。

阵发性睡眠性血红蛋白尿症（paroxysmal nocturnal hemoglobinuria, PNH）是一种由于1个或几个造血干细胞经获得性体细胞PIG-A基因（phosphotidyl inositol glycan complementation group A）突变造成的非恶性的克隆性疾病，PIG-A突变造成糖基磷脂酰肌醇（glycosyl phosphatidyl inositol，GPI）合成 ...

阵发性睡眠性血红蛋白尿 ( paroxysmal nocturnal hemoglobinuria，PNH)是一种少见的获得性克隆性造血干细胞疾病，表现为溶血性贫血、血栓和骨髓衰竭[1~4]。PNH是由造血干细胞出现磷脂酰肌醇糖苷A (phosphatidylinositol glycan class A，PIGA)基因一个或多个突变导致的。

2000年1月1日 · The first certain description of PNH is that of William Gull (1816-1890), a leading physician at Guy's Hospital, London in the mid-and late-19th century. He wrote a case report notable for its length and completeness and for its lack of any references [2].

2024年9月23日 · Soliris (Eculizumab) 是一种针对补体蛋白 C5 的单克隆抗体，该蛋白参与 PNH 患者红细胞的破坏。 它于 2007 年获得美国食品和药物管理局 (FDA) 批准，是目前 PNH 治疗的护理标准。 2023年，全球Soliris市场估值约为15亿美元，预计未来几年将大幅增长。 Ultomiris（Ravulizumab）是一种长效单克隆抗体，也针对C5。 它于2018年获得FDA批准，并被证明比Soliris更有效、更方便，且给药间隔更长。 预计到 2024 年，全球 Ultomiris 市场将达到 …

2024年3月1日 · Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder caused by complement-mediated hemolysis and thrombosis through the alternative pathway. The most common symptom of PNH is fatigue due to chronic anemia, which can negatively impact quality of life (QoL) and affect overall well-being.

2016年2月12日 · The history of the development of the understanding of the natural history of paroxysmal nocturnal hemoglobinuria (PNH) illustrates the interaction between basic science and clinical problems. Through application of the knowledge obtained in the laboratory, a better understanding of the clinical problems has been achieved.

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic stem cell disease caused by abnormal expression of glycosylphosphatidylinositol (GPI) on the cell membrane due to mutations in the phosphatidylinositol glycan class A (PIGA) gene.

2023年1月3日 · For the last 20 years, therapy of paroxysmal nocturnal hemoglobinuria (PNH) relied-up until recently-on antibody based terminal complement inhibitionon. PNH pathophysiology-a mutational defect leading to partial or complete absence of complement-regulatory proteins on blood cells-leads to intravascu …