Shoaib Eisha, Imam Filzah, Khan Mahnoor, Jaber Amin Mohammed H
Karachi Medical and Dental College, Karachi, Pakistan.
Faculty of Medicine, Alzaiem Alazhari University, Khartoum, Sudan.
Ann Med Surg (Lond). 2024 Nov 13;86(12):6921-6922. doi: 10.1097/MS9.0000000000002706. eCollection 2024 Dec.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening hematologic disease that is characterized by the destruction of red blood cells, leading to a range of severe symptoms and complications. Recent advancements in drug therapies have significantly improved the prognosis for PNH patients. This editorial comprises the impact of PNH drugs, focusing on eculizumab and ravulizumab and comparing them to the recently approved complement inhibitor, crovalimab, which targets the complement system to prevent hemolysis. The discussion includes an analysis of clinical trial data and patient outcomes. The editorial mainly addresses emerging therapies, like crovalimab, that promise to offer more comprehensive, complete blockage of the complement system and low-dose solutions, reducing the treatment hassle while simultaneously appealing to a wider range of patients.
阵发性夜间血红蛋白尿(PNH)是一种罕见的、危及生命的血液疾病,其特征是红细胞遭到破坏,从而导致一系列严重症状和并发症。药物治疗方面的最新进展显著改善了PNH患者的预后。这篇社论阐述了PNH药物的影响,重点介绍了依库珠单抗和ravulizumab,并将它们与最近获批的补体抑制剂crovalimab进行比较,后者通过作用于补体系统来防止溶血。讨论内容包括对临床试验数据和患者治疗结果的分析。这篇社论主要探讨了诸如crovalimab等新兴疗法,这些疗法有望更全面、彻底地阻断补体系统,并采用低剂量方案,在减少治疗麻烦的同时吸引更多患者。