Gernez Yael, Baker Mary Grace, Maglione Paul J
Division of Allergy and Immunology, Department of Pediatrics, Stanford School of Medicine, Stanford, California.
Division of Clinical Immunology, Department of Medicine, Icahn School of Medicine at Mount Sinai, New York, New York.
Transfusion. 2018 Dec;58 Suppl 3(Suppl 3):3056-3064. doi: 10.1111/trf.15020.
Primary immunodeficiency (PID) diseases result from genetic defects of the immune system that increase a patient's susceptibility to infections. The types of infections that occur in patients with PID diseases are dictated largely by the nature of the immunodeficiency, which can be defined by dysfunction of cellular or humoral defenses. An increasing number of PID diseases, including those with both cellular and humoral defects, have antibody deficiency as a major feature, and as a result can benefit from immunoglobulin replacement therapy. In fact, the most common PID diseases worldwide are antibody deficiencies and include common variable immunodeficiency, congenital agammaglobulinemia, hyper-IgM syndrome, specific antibody deficiency, and Good syndrome. Although immunoglobulin replacement therapy is the cornerstone of treatment for the majority of these conditions, a thorough understanding of the specific infections for which these patients are at increased risk can hasten diagnosis and guide additional therapies. Moreover, the infection trends in some patients with PID disease who have profound defects of cellular immunity, such as autosomal-dominant hyper-IgE syndrome (Job/Buckley syndrome) or dedicator of cytokinesis 8 (DOCK8) deficiency, suggest that select patients might benefit from immunoglobulin replacement therapy even if their immunodeficiency is not limited to antibody defects. In this review, we provide an overview of the predisposition to infections seen in PID disease that may benefit from immunoglobulin replacement therapy.
原发性免疫缺陷(PID)疾病是由免疫系统的遗传缺陷引起的,这些缺陷会增加患者对感染的易感性。PID疾病患者发生的感染类型很大程度上取决于免疫缺陷的性质,免疫缺陷可通过细胞或体液防御功能障碍来定义。越来越多的PID疾病,包括那些同时存在细胞和体液缺陷的疾病,都以抗体缺陷为主要特征,因此可从免疫球蛋白替代疗法中获益。事实上,全球最常见的PID疾病是抗体缺陷,包括常见变异型免疫缺陷、先天性无丙种球蛋白血症、高IgM综合征、特异性抗体缺陷和古德综合征。尽管免疫球蛋白替代疗法是大多数这些疾病治疗的基石,但全面了解这些患者感染风险增加的特定感染,有助于加快诊断并指导其他治疗。此外,一些细胞免疫存在严重缺陷的PID疾病患者,如常染色体显性高IgE综合征(乔布/巴克利综合征)或细胞分裂素8(DOCK8)缺陷患者的感染趋势表明,即使免疫缺陷不限于抗体缺陷,部分患者也可能从免疫球蛋白替代疗法中获益。在本综述中,我们概述了PID疾病中可能从免疫球蛋白替代疗法中获益的感染易感性。