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[皮下注射丙种球蛋白治疗常见变异型免疫缺陷病。西班牙的首次经验]

[Subcutaneous gammaglobulin in common variable immunodeficiency. First experience in Spain].

作者信息

Maroto Hernando M, Soler Palacín P, Martin Nalda N, Oliveras Arenas M, Español Boren T, Figueras Nadal C

机构信息

Sección de Cardiología Infantil, Hospital Infantil La Fe, Valencia, España.

出版信息

An Pediatr (Barc). 2009 Feb;70(2):111-9. doi: 10.1016/j.anpedi.2008.11.011. Epub 2009 Feb 3.

Abstract

INTRODUCTION AND AIM

Weekly home-based subcutaneous immunoglobulin (SCIg) therapy is an alternative to intravenous immunoglobulin (IVIg) in the treatment of patients with primary antibody deficiencies. The objective of this study was to investigate the efficacy, safety, related quality of life and cost effectiveness of SCIg in our area.

MATERIALS AND METHODS

Observational and descriptive study including paediatric patients with common variable immunodeficiency (CVID) receiving SCIg in our hospital (November 2006 to April 2008). Obtained data were compared with those from the last year with IVIg.

RESULTS

Eleven patients with CVID were included. Median age was 15 years. The median trough serum IgG level was 622 mg/dl with IVIg. In patients in whom the SCIg dose was maintained or reduced compared to IVIg, the median trough serum IgG level was 850 mg/dl (p < 0.0005). Annual rate of infection was 2.22 per patient-year, without significant differences to IVIg (p = 0.212). There were 58 treatment-related adverse events (AE) reported with SCIg (45 local AE and 13 systemic AE). The most frequent treatment-related adverse event was infusion-site reaction. Switching to home-based subcutaneous IgG treatment led to significant improvements in quality of life and substantial cost savings.

CONCLUSIONS

We conclude that subcutaneous administration of 16% SCIg is a safe and cost-effective alternative to IVIg for replacement therapy of primary antibody deficiencies. Median trough serum IgG levels were higher with SCIg. Local AE were common but mild and the incidence decreased over time. Quality of life is significantly improved.

摘要

引言与目的

对于原发性抗体缺陷患者的治疗,每周居家皮下注射免疫球蛋白(SCIg)疗法是静脉注射免疫球蛋白(IVIg)的一种替代方案。本研究的目的是调查SCIg在我们地区的疗效、安全性、相关生活质量及成本效益。

材料与方法

一项观察性和描述性研究,纳入了2006年11月至2008年4月在我院接受SCIg治疗的患有常见变异型免疫缺陷(CVID)的儿科患者。将获得的数据与上一年接受IVIg治疗的数据进行比较。

结果

纳入了11例CVID患者。中位年龄为15岁。使用IVIg时血清IgG谷值水平中位数为622mg/dl。与IVIg相比,SCIg剂量维持不变或减少的患者,血清IgG谷值水平中位数为850mg/dl(p<0.0005)。每位患者每年的感染率为2.22次,与IVIg相比无显著差异(p = 0.212)。SCIg报告了58例与治疗相关的不良事件(AE)(45例局部AE和13例全身AE)。最常见的与治疗相关的不良事件是输注部位反应。改用居家皮下注射IgG治疗可显著改善生活质量并大幅节省成本。

结论

我们得出结论,皮下注射16%的SCIg是用于原发性抗体缺陷替代治疗的一种安全且具有成本效益的IVIg替代方案。SCIg的血清IgG谷值水平中位数更高。局部AE常见但轻微,且发生率随时间下降。生活质量得到显著改善。

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