Market Access and Outcomes Strategy, RTI Health Solutions, Research Triangle, NC.
Global Health Economics and Outcomes Research, Apellis Pharmaceuticals, Waltham, MA.
J Manag Care Spec Pharm. 2020 Dec;26(12-b Suppl):S14-S20. doi: 10.18553/jmcp.2020.26.12-b.s14.
The current standard of care for paroxysmal nocturnal hemoglobinuria (PNH) are the C5 inhibitors eculizumab and ravulizumab, both monoclonal antibodies designed to target the complement protein C5, thereby preventing its cleavage and the formation of the terminal attack complex. C5 inhibitors have yielded substantial improvements in the treatment of PNH and changed the mortality and morbidity, as well as health-related quality of life of patients with the disease. These treatments target underlying intravascular hemolysis; however, they do not address extravascular hemolysis, resulting in incomplete response and remaining symptoms in some patients. Therefore, despite treatment with a C5 inhibitor, some patients still experience anemia with associated fatigue, transfusion needs, and impaired health-related quality of life. This research was developed under a research contract between RTI Health Solutions and Apellis Pharmaceuticals and was funded by Apellis Pharmaceuticals. Bektas, Copley-Merriman, and Khan are employees of RTI Health Solutions. Sarda is an employee of Apellis Pharmaceuticals. Shammo consults for Apellis Pharmaceuticals.
目前阵发性睡眠性血红蛋白尿症（PNH）的标准治疗方法是 C5 抑制剂依库珠单抗和拉维珠单抗，这两种单克隆抗体都是专门针对补体蛋白 C5 设计的，从而阻止其裂解和形成末端攻击复合物。C5 抑制剂在治疗 PNH 方面取得了显著的进展，改变了疾病患者的死亡率、发病率和健康相关生活质量。这些治疗方法针对的是血管内溶血的根本原因；然而，它们并没有解决血管外溶血的问题，导致一些患者的反应不完全，仍有症状存在。因此，尽管使用了 C5 抑制剂，一些患者仍然会出现贫血，伴有疲劳、输血需求和健康相关生活质量受损。这项研究是在 RTI 健康解决方案公司和 Apellis 制药公司之间的研究合同下进行的，由 Apellis 制药公司资助。贝克塔斯、科普利-梅里曼和汗是 RTI 健康解决方案公司的员工。萨尔达是 Apellis 制药公司的员工。沙莫为 Apellis 制药公司提供咨询。
