Dose and outcomes in primary immunodeficiency disorders
Dose and outcomes in primary immunodeficiency disorders
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DOI：
10.1111/cei.12492
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发表时间：
2014-12-01
影响因子：
4.6
通讯作者：
Bonagura, V. R.
中科院分区：
文献类型：
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作者：
Bonagura, V. R.
目前，欧洲原发性免疫缺陷疾病（PID）患者的主要治疗形式是皮下免疫球蛋白（SCIg）替代疗法。相反，直到最近，静脉注射免疫球蛋白（IVIg）替代是美国治疗PID的首选方案。PID患者比没有PID的个体更容易复发或严重感染。对2004年至2010年期间欧洲免疫缺陷学会（ESID）登记数据的回顾性分析显示，接受SCIg或IVIg替代治疗的PID患者的临床结局存在显著的区域差异[1]。在这项分析中，接受IVIg的患者似乎比接受SCIg的患者出现更严重的细菌感染，住院时间更长。此外，在一个亚组的患者中，从IVIg转换为SCIg替代治疗的患者在转换后住院天数较少[1]。然而，在决定免疫球蛋白G（IgG）给药途径时，有许多考虑因素。与静脉注射（iv）的免疫球蛋白（IG）G相比，皮下注射（sc）的免疫球蛋白G的量是有限的;因此，SC IG的给药频率必须高于IVIg。SCIg治疗方案包括每周一次、每两周一次或每日一次的推注剂量，可自行给药。静脉给药途径的典型方案是在家中或在诊所环境中每3-4周一次。与IVIg输注后血清IgG水平的峰值和谷值周期相反，SCIg方案产生的血清IgG谷值水平变化极小[2]。IVIg的全身副作用比SCIg更常见，SCIg的输注部位反应更常见，尤其是在sc IgG替代治疗的早期阶段[2，3]。促进SCIg给药的另一种方法是预输注重组透明质酸酶，然后每月一次皮下注射IgG。这
Currently, the principal form of treatment for patients with primary immunodeficiency disorders (PID) in Europe is subcutaneous immunoglobulin (SCIg) replacement therapy. Conversely, until relatively recently, intravenous immunoglobulin (IVIg) replacement was the preferred option for treatment of PID in the United States. Patients with PID are more susceptible to recurrent or severe infections than individuals without PID. A retrospective analysis of data from the European Society of Immunodeficiencies (ESID) registry, between 2004 and 2010, showed marked regional variability in the clinical outcomes of patients with PID receiving SCIg or IVIg replacement therapy [1]. In this analysis, patients receiving IVIg appeared to present with more serious bacterial infections and spend more days in hospital than patients receiving SCIg. Furthermore, in a subgroup of patients, those who switched from IVIg to SCIg replacement therapy spent fewer days in hospital after the switch [1].However, there are a number of considerations when deciding on the route of immunoglobulin G (IgG) administration. The volume of immunoglobulin (Ig) G that can be given into the subcutaneous (sc) space can be limited when compared with the volume that can be delivered intravenously (iv); thus, SCIg must be administered more frequently than IVIg. SCIg treatment regimens include weekly, bi-weekly or daily push doses that can be selfadministered. Typical regimens for the iv route of administration are once every 3–4 weeks given at home or in a clinic setting. In contrast to the cycle of peak and trough serum IgG levels following IVIg infusion, SCIg regimens produce minimal variation in trough serum IgG levels [2]. Systemic side effects are more common with IVIg than SCIg, and infusion site reactions are more common with SCIg, particularly during the early phase of sc IgG replacement therapy [2, 3]. An alternative method to facilitate SCIg administration is a pre-infusion of recombinant hyaluronidase followed by a monthly dose of sc IgG. This