Waheed Aiman, Gul Muhammad Hamza, Wardak Abdul Baseer, Shabbir Muhammad Usama Bin, Touseef Sabahat, Zafar Fatima, Hussaini Helai
Rawalpindi Medical College, Rawalpindi, Pakistan.
Hayatabad Medical Complex, Peshawar, Pakistan.
Ann Med Surg (Lond). 2025 Jan 30;87(2):451-453. doi: 10.1097/MS9.0000000000002775. eCollection 2025 Feb.
Crovalimab, a new promising drug for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) has emerged. This marks a significant advancement in the treatment of PNH and potentially other complement-mediated disorders. PNH is characterized by complement-mediated hemolysis, thrombosis, and bone marrow failure, leading to severe morbidity and mortality in affected individuals. PNH arises from a somatic mutation in the PIGA gene, leading to the loss of glycosylphosphatidylinositol (GPI)-anchored proteins on blood cells, making them vulnerable to complement-mediated destruction. Crovalimab is a new anti-C5 recycling antibody that offers a promising treatment by being administered subcutaneously every 4 weeks at a low volume. Clinical trials such as COMMODORE 3 have shown crovalimab's effectiveness and high tolerability in PNH patients who have not previously used a C5 inhibitor. Crovalimab has been proven effective in maintaining hemoglobin levels, reducing the need for transfusions, and improving patient outcomes by inhibiting terminal complement activation.
一种用于治疗阵发性夜间血红蛋白尿(PNH)的新的有前景的药物——克罗瓦利单抗已出现。这标志着PNH治疗以及潜在的其他补体介导疾病的治疗取得了重大进展。PNH的特征是补体介导的溶血、血栓形成和骨髓衰竭,导致受影响个体出现严重的发病率和死亡率。PNH由PIGA基因的体细胞突变引起,导致血细胞上糖基磷脂酰肌醇(GPI)锚定蛋白的缺失,使其易受补体介导的破坏。克罗瓦利单抗是一种新型抗C5循环抗体,通过每4周皮下注射小剂量给药,提供了一种有前景的治疗方法。诸如COMMODORE 3等临床试验已表明,克罗瓦利单抗在未曾使用过C5抑制剂的PNH患者中具有有效性和高耐受性。通过抑制终末补体激活,克罗瓦利单抗已被证明在维持血红蛋白水平、减少输血需求以及改善患者预后方面有效。