Effectiveness of Immunoglobulin Replacement Therapy on Clinical Outcome in Patients with Primary Antibody Deficiencies: Results from a Multicenter Prospective Cohort Study
Effectiveness of Immunoglobulin Replacement Therapy on Clinical Outcome in Patients with Primary Antibody Deficiencies: Results from a Multicenter Prospective Cohort Study
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DOI：
10.1007/s10875-011-9511-0
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发表时间：
2011-06-01
影响因子：
9.1
通讯作者：
Fiorilli, Massimo
中科院分区：
文献类型：
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作者：
Quinti, Isabella;Soresina, Annarosa;Fiorilli, Massimo
对201例常见可变免疫缺陷患者和101例X连锁无丙种球蛋白血症患者进行了一项为期5年的多中心前瞻性研究，累积随访时间为1，365患者年，以确定相关临床共病的预后标志物和风险因素，长期免疫球蛋白治疗的效果和随着时间的推移维持IgG谷水平，以最大限度地降低感染风险。总体而言，21%的常见可变免疫缺陷患者和24%的X连锁无丙种球蛋白血症患者在研究期间保持无感染。在开始IG替代治疗后，观察到肺炎发作减少。在观察期间，肺炎发生率保持较低水平，并随时间推移保持恒定。肺炎患者的IgG谷水平并不明显低于非肺炎患者，但IgG谷水平持续< 400 mg/dL的患者除外。在X连锁无丙种球蛋白血症中，通过最终多变量模型确定的肺炎的唯一共病风险因素是支气管扩张的存在。在常见的可变免疫缺陷中，我们的数据使我们能够确定以高肺炎风险为特征的临床表型：诊断时IgG和伊加水平低的患者;伊加水平< 7 mg/dL且患有支气管扩张的患者。替代剂量免疫球蛋白治疗非感染性共病（自身免疫、淋巴细胞增生和肠病）的效果仍有待确定。由于肺部疾病进展在X连锁无丙种球蛋白血症和常见可变免疫缺陷患者亚组中发挥的主要作用，抗体缺乏患者中独特的一般保护性IgG谷水平仍不确定。
A 5-years multicenter prospective study on 201 patients with common variable immunodeficiencies and 101 patients with X-linked agammaglobulinemia over a cumulative follow-up period of 1,365 patient-years was conducted to identify prognostic markers and risk factors for associated clinical co-morbidities, the effects of long-term immunoglobulin treatment and the IgG trough level to be maintained over time required to minimise infection risk. Overall, 21% of the patients with common variable immunodeficiencies and 24% of patients with X-linked agammaglobulinemia remained infection free during the study. A reduction of pneumonia episodes has been observed after initiation of Ig replacement. During the observation time, pneumonia incidence remained low and constant over time. Patients with pneumonia did not have significant lower IgG trough levels than patients without pneumonia, with the exception of patients whose IgG trough levels were persistently < 400 mg/dL. In X-linked agammaglobulinemia, the only co-morbidity risk factor identified for pneumonia by the final multivariable model was the presence of bronchiectasis. In common variable immunodeficiencies, our data allowed us to identify a clinical phenotype characterised by a high pneumonia risk: patients with low IgG and IgA levels at diagnosis; patients who had IgA level < 7 mg/dL and who had bronchiectasis. The effect of therapy with immunoglobulins at replacement dosage for non-infectious co-morbidities (autoimmunity, lymphocytic hyperplasia and enteropathy) remains to be established. A unique general protective trough IgG level in antibody deficiency patients will remain undefined because of the major role played by the progression of lung disease in X-linked agammaglobulinemia and in a subset of patients with common variable immunodeficiencies.