Church Joseph A, Leibl Heinz, Stein Mark R, Melamed Isaac R, Rubinstein Arye, Schneider Lynda C, Wasserman Richard L, Pavlova Borislava G, Birthistle Karl, Mancini Marianne, Fritsch Sandor, Patrone Lisa, Moore-Perry Kerry, Ehrlich Hartmut J
Childrens Hospital Los Angeles, Los Angeles, California, USA.
J Clin Immunol. 2006 Jul;26(4):388-95. doi: 10.1007/s10875-006-9025-3. Epub 2006 May 17.
The present clinical study was designed to evaluate the efficacy, pharmacokinetics and safety of a new 10% liquid intravenous immune globulin in patients with primary immunodeficiency diseases. Sixty-one adults and children with primary immuno-deficiency diseases received doses of 300-600 mg/kg body weight every 21-28 days for 12 months. No validated acute serious bacterial infections were reported. The 95% confidence interval for the annualized rate of acute serious bacterial infections (primary endpoint) was 0-0.060. A total of four predefined validated other bacterial infections commonly occurring in primary immunodeficiency disease subjects were observed; none were serious, severe or resulted in hospitalization. The median elimination half-life of IgG was 35 days. Median total IgG trough levels varied from 9.6 to 11.2 g/L. Temporally associated adverse experiences were determined for 72 h after each infusion and the most common adverse experience was headache, which was associated with 6.9% of infusions. The study met the primary endpoint for efficacy and demonstrated excellent tolerability of the new 10% liquid intravenous imunoglobulin preparation.
本临床研究旨在评估一种新型10%液体静脉注射免疫球蛋白对原发性免疫缺陷病患者的疗效、药代动力学和安全性。61例患有原发性免疫缺陷病的成人和儿童每21 - 28天接受300 - 600mg/kg体重的剂量，持续12个月。未报告经过验证的急性严重细菌感染。急性严重细菌感染年化率（主要终点）的95%置信区间为0 - 0.060。共观察到4例原发性免疫缺陷病受试者中常见的预定义且经过验证的其他细菌感染；均不严重、不重度，也未导致住院。IgG的中位消除半衰期为35天。总IgG谷值水平中位数在9.6至11.2g/L之间。每次输注后72小时确定与时间相关的不良事件，最常见的不良事件是头痛，与6.9%的输注相关。该研究达到了疗效的主要终点，并证明了新型10%液体静脉注射免疫球蛋白制剂具有良好的耐受性。
