Department of Pediatric Nephrology, Radboud University Medical Center, Radboud Institute for Molecular Life Sciences, Amalia Children's Hospital, PO Box 9101, 6500 HB, Nijmegen, The Netherlands.
Department of Pharmacy, Radboud University Medical Center, Nijmegen, The Netherlands.
Clin Pharmacokinet. 2019 Jul;58(7):859-874. doi: 10.1007/s40262-019-00742-8.
Eculizumab is the first drug approved for the treatment of complement-mediated diseases, and current dosage schedules result in large interindividual drug concentrations. This review provides insight into the pharmacokinetic and pharmacodynamic properties of eculizumab, both for reported on-label (paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, generalized myasthenia gravis) and off-label (hematopoietic stem cell transplantation-associated thrombotic microangiopathy) indications. Furthermore, we discuss the potential of therapeutic drug monitoring to individualize treatment and reduce costs.
依库珠单抗是首个获批用于治疗补体介导疾病的药物,目前的给药方案导致个体间药物浓度差异较大。本综述深入探讨了依库珠单抗的药代动力学和药效学特性,包括已批准的适应证(阵发性睡眠性血红蛋白尿症、非典型溶血尿毒综合征、全身性重症肌无力)和超适应证(造血干细胞移植相关血栓性微血管病)。此外,我们还讨论了治疗药物监测在实现个体化治疗和降低成本方面的潜力。