Björkander J, Nikoskelainen J, Leibl H, Lanbeck P, Wallvik J, Lumio J T, Braconier J H, Pavlova B G, Birthistle K, Engl W, Walter S, Ehrlich H J
SU/Sahlgrenska, Gothenburg, Sweden.
Vox Sang. 2006 May;90(4):286-93. doi: 10.1111/j.1423-0410.2006.00764.x.
The aim of this study was to evaluate the pharmacokinetics, efficacy and safety of a newly developed 10% liquid immunoglobulin preparation in patients with primary immunodeficiency diseases. This new preparation for intravenous use includes three dedicated virus clearance steps in its manufacturing process to ensure a high margin of viral safety.
This was a prospective, open-label, non-controlled, multicentre study. Twenty-two subjects with primary immunodeficiency were treated initially with three infusions of a licensed intravenous immunoglobulin to standardize the immunoglobulin G (IgG) replacement therapy of all subjects to the same intravenous product. A total of nine infusions of the new 10% liquid preparation were subsequently administered.
The median terminal half-life of total IgG following administration of the new preparation was 30.1 days. Median terminal half-lives for IgG subclasses IgG(1), IgG(2), IgG(3) and IgG(4) were 28.3, 31.3, 20.9 and 24.2 days, respectively. The median total serum IgG steady-state trough level was 8.51 g/l. No severe infection episodes started after initiation of treatment with the new preparation. The median rate of mild or moderate infection episodes was 0.48 per month. A total of 194 infusions with the new 10% liquid immunoglobulin preparation were administered. The mean dose per infusion was 0.41 g/kg body weight and the maximum infusion rates recorded were 8 ml/kg/h. Adverse experiences were mostly mild and unrelated to the study drugs. Only 4% of infusions with the new product were followed by one or more related adverse experiences.
The new 10% liquid immunoglobulin preparation was well tolerated and shown to have an excellent pharmacokinetic, efficacy and safety profile. The liquid formulation provides convenience to patients and healthcare professionals.
本研究旨在评估一种新研发的10%液体免疫球蛋白制剂在原发性免疫缺陷病患者中的药代动力学、疗效及安全性。这种新的静脉用制剂在生产过程中包含三个专门的病毒清除步骤，以确保高病毒安全性。
这是一项前瞻性、开放标签、非对照、多中心研究。22名原发性免疫缺陷患者最初接受三次已获许可的静脉免疫球蛋白输注，以使所有受试者的免疫球蛋白G（IgG）替代疗法标准化为相同的静脉产品。随后共给予九次新的10%液体制剂输注。
给予新制剂后，总IgG的中位终末半衰期为30.1天。IgG亚类IgG(1)、IgG(2)、IgG(3)和IgG(4)的中位终末半衰期分别为28.3天、31.3天、20.9天和24.2天。血清总IgG稳态谷浓度中位数为8.51 g/l。开始使用新制剂治疗后未发生严重感染事件。轻度或中度感染事件的发生率中位数为每月0.48次。共输注新的10%液体免疫球蛋白制剂194次。每次输注的平均剂量为0.41 g/kg体重，记录到的最大输注速率为8 ml/kg/h。不良事件大多轻微，且与研究药物无关。使用新产品的输注中只有4%出现一种或多种相关不良事件。
新的10%液体免疫球蛋白制剂耐受性良好，药代动力学、疗效和安全性均表现出色。液体制剂为患者和医护人员提供了便利。
