Safety and efficacy of self-administered subcutaneous immunoglobulin in patients with primary immunodeficiency diseases
Safety and efficacy of self-administered subcutaneous immunoglobulin in patients with primary immunodeficiency diseases
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DOI：
10.1007/s10875-006-9021-7
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发表时间：
2006-05-01
影响因子：
9.1
通讯作者：
Berger, Melvin
中科院分区：
文献类型：
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作者：
Ochs, Hans D.;Gupta, Sudhir;Berger, Melvin
静脉注射免疫球蛋白（IVIg），间隔3-4周，目前是美国原发性免疫缺陷疾病（PIDD）患者的标准治疗方法。为了评估免疫球蛋白给药的替代模式，我们设计了一项开放标签研究，以调查PIDD患者皮下注射免疫球蛋白制剂（16% IgG）的有效性和安全性。在最后一次IVIg输注后，65名患者进入了为期3个月的洗入/洗出阶段，旨在通过皮下注射免疫球蛋白使患者达到稳定状态。随后是12个月的每周SCIg输注，剂量在药代动力学亚研究中确定，以提供非劣等血管内暴露。这导致平均每周剂量为158 mg/kg，相当于以前静脉注射剂量的137%。2例（4%）患者各报告1次严重细菌感染（肺炎），年发病率为0.04例/患者年。每名患者每年有4.43例感染。在研究期间，平均谷血清IgG水平从786 mg/dL增加到1040 mg/dL，平均增加39%。最常见的治疗相关不良事件是输液反应，91%的患者报告；这主要是轻度或中度的，发病率随着时间的推移而下降。治疗相关的严重不良事件未见报道。我们的结论是，皮下注射16%的SCIg是一种安全有效的替代IVIg替代治疗PIDD。
Intravenous immunoglobulin (IVIg) infusions at 3-4 week intervals are currently standard therapy in the United States for patients with primary immune deficiency diseases (PIDD). To evaluate alternative modes of immunoglobulin administration we have designed an open-label study to investigate the efficacy and safety of a subcutaneously administered immunoglobulin preparation (16% IgG) in patients with PIDD. After their final IVIg infusion, 65 patients entered a 3-month, wash-in/wash-out phase, designed to bring patients to steady-state with subcutaneously administered immunoglobulin. This was followed by 12 months of weekly SCIg infusions, at a dose determined in a pharmacokinetic substudy to provide noninferior intravascular exposure. This resulted in a mean weekly dose of 158 mg/kg, calculated to equal 137% of the previous intravenous dose. Two patients (4%) each reported 1 serious bacterial infection (pneumonia), an annual rate of 0.04 per patient-year. There were 4.43 infections of any type per patient-year. Mean trough serum IgG levels increased from 786 to 1040 mg/dL during the study, a mean increase of 39%. The most frequent treatment-related adverse event was infusion-site reaction, reported by 91% of patients; this was predominantly mild or moderate, and the incidence decreased over time. No treatment-related serious adverse events were reported. We conclude that subcutaneous administration of 16% SCIg is a safe and effective alternative to IVIg for replacement therapy of PIDD.