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）合成异常，导致由GPI锚接在细胞膜上的一 ...

2023年7月31日 · Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease that presents clinically with a variety of symptoms, the most prevalent of which are hemolytic anemia, hemoglobinuria, and somatic symptoms including fatigue and shortness of breath. Other findings associated with PNH include thrombosis, renal insufficiency, and in the later course of the disease, even bone marrow failure. The ...

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, clonal disorder affecting hematopoiesis. It is characterized by chronic intravascular hemolysis (IVH), pronounced thrombophilia and variable cytopenia. Most patients are diagnosed in young adulthood with a median of 35–40 years.

2025年3月16日 · Paroxysmal nocturnal hemoglobinuria (PNH) is a non-malignant clonal hematopoietic disorder. There are two components to the pathogenesis of PNH: (i) a mutant stem cell, (ii) expansion of the mutant clone. Component (i) is straightforward: there is almost always an inactivating somatic mutation of the X-linked gene PIGA.

确定在阵发性睡眠性血红蛋白尿症（pnh）、自身免疫性溶血性贫血（aiha）与遗传性球形红细胞增多症（hs）鉴别诊断中有重要参考价值的溶血特征。 比较1998年1月至2017年4月108例经典型pnh患者、127例aiha患者与172例hs患者的临床表现和溶血特征。

Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, multi-systemic, progressive and life-threatening disease characterized by intravascular hemolysis, thrombotic events, serious infections and bone marrow failure.1, 2 Hemolysis in PNH is due to the action of the complement on abnormal red blood cells (RBCs). Compared with normal RBCs, PNH ...

pnh 克隆通常发生在患有骨髓衰竭（获得性 再生障碍性贫血) 且没有 pnh 相关症状。这些患者的体表面积通常较小（ < 30% 的患者患有亚临床 pnh 克隆，并且不能从补体抑制剂中获益；相反，确定性治疗是针对再生障碍性贫血的。 然而，经典 pnh 可以从再生障碍性 ...

2025年1月6日 · 这些更新对pnh的早期诊断和精准治疗有何重要意义？ 付蓉教授：在诊断方面，2024版指南更新了pnh的诊断标准和流程，完善了相关筛查指征，强调了流式细胞术在pnh诊断中的关键作用，高灵敏度流式细胞术已成为诊断pnh的“金标准”。 具体而言： 01、规范pnh克隆 ...

2024年11月5日 · In December 2023, iptacopan was approved as the first orally administered monotherapy for patients with PNH in the United States (US). Since then, two more CIs have been approved (danicopan and crovalimab). With an increasing number of CIs available for PNH, timely real-world evidence is needed to inform treatment decision-making.

2024年6月17日 · 今天，阵发性睡眠性血红蛋白尿症（pnh）创新口服单药疗法 飞赫达 ® （盐酸伊普可泮胶囊）成功开出全国首张处方， 标志着中国pnh成人患者迎来了全面控制血管内外溶血、便捷口服用药的全新治疗选择。. 作为 全球首个且目前唯一的成人pnh口服单药疗法， 盐酸伊普可泮胶囊今年4月在中国获批用于 ...