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阵发性夜间血红蛋白尿的抗体治疗

[Antibody therapy for paroxysmal nocturnal hemoglobinuria].

作者信息

Nishimura Jun-Ichi

机构信息

Osaka University Graduate School of Medicine, Department of Hematology and Oncology.

出版信息

Rinsho Ketsueki. 2020;61(8):929-936. doi: 10.11406/rinketsu.61.929.

Abstract

Treatment with eculizumab (Soliris), a humanized anti-C5 monoclonal antibody improves the quality of life of patients with paroxysmal nocturnal hemoglobinuria (PNH), remarkably reduces hemolysis, improves symptoms associated with hemolysis, and prevents thrombosis. Because eculizumab therapy is not a curative treatment, it is necessary to continue infusion every two weeks, which has been an issue from the viewpoint of convenience. In recent years, an improved version of eculizumab, ravulizumab (Ultomiris), which relies on the technology of recycling antibodies has been developed and can be administered every 8 weeks. Crovalimab (SKY59), which can be administered subcutaneously every four weeks, is also under development, and therefore, the convenience for patients with PNH is improving. However, many issues still persist, and several new anti-complement drugs are currently under development. Hopefully, a better drug will be developed by thorough examination of what drug is best for the patient by considering not only its efficacy and safety but also its convenience.

摘要

使用人源化抗C5单克隆抗体依库珠单抗(Soliris)进行治疗可改善阵发性夜间血红蛋白尿(PNH)患者的生活质量,显著减少溶血,改善与溶血相关的症状,并预防血栓形成。由于依库珠单抗疗法并非治愈性治疗,因此有必要每两周持续输注一次,从便利性角度来看这一直是个问题。近年来,已开发出依库珠单抗的改进版本ravulizumab(Ultomiris),其依赖抗体循环技术,可每8周给药一次。每四周可皮下给药的crovalimab(SKY59)也在研发中,因此,PNH患者的便利性正在提高。然而,许多问题仍然存在,目前还有几种新型抗补体药物正在研发中。有望通过全面考量哪种药物对患者最有利,不仅考虑其疗效和安全性,还考虑其便利性,从而研发出更好的药物。

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