PRIMARY IMMUNODEFICIENCY DISEASE

原发性免疫缺陷病

• 批准号: 7952135
• 负责人: J. ANDREW GRANT
• 金额: $ 1.31万
• 依托单位: UNIVERSITY OF TEXAS MED BR GALVESTON
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-04-01 至 2009-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7952135
• 关键词: 12 year old; Adverse reactions; Antibody Formation; Bacterial Infections; Clinical Research; Computer Retrieval of Information on Scientific Projects Database; Country; Disease; Drug Kinetics; Enrollment; Funding; Grant; Immunoglobulin G; Immunoglobulins; Immunologic Deficiency Syndromes; Individual; Infection; Institution; Intravenous; Intravenous Immunoglobulins; Intravenous infusion procedures; Licensing; Methods; Muscle; Patients; Predisposition; Preparation; Replacement Therapy; Research; Research Personnel; Resources; Risk; Route; Safety; Serum; Severities; Source; United States; United States National Institutes of Health; aged; intravenous administration; prevent; subcutaneous

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Defective antibody formation is the most common abnormality in the majority of primary immunodeficiency (PID) diseases. It is most often reflected by a decrease in serum immunoglobulins, which in turn leads to increased susceptibility to bacterial infections especially of the sinopulmonary tract. Individuals with these diseases require replacement therapy with immune globulins to prevent or reduce the severity of infections. For many years, immune globulin replacement therapy was given by the muscular and then by the intravenous (IV) route. Currently, the vast majority of immune globulins in the United States are licensed for IV administration. During the last few years, subcutaneous (SC) application of immune globulin preparations was introduced in many countries worldwide. This method of IgG replacement therapy is considered to be effective, safe and also highly appreciated by the patients as it has a low risk of adverse reactions and leads to higher trough serum IgG concentrations compared to monthly IV infusions. The purpose of the study is to evaluate the tolerability of IGIV, 10% given subcutaneously and the pharmacokinetics of immunoglobulin G (IgG) following subcutaneous (SC) treatment with IGIV, 10% in subjects with primary immunodeficiency (PID) disorders. Approximately 23 subjects with PID aged 12 years and older who had previously been treated with intravenous (IV) immunoglobulins and approximately 12 subjects with PID aged 2 to <12 years who also had received previous treatment with IV immunoglobulins will be enrolled and treated. The study will help assess the safety and efficacy of this mode of treatment.

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 缺陷性抗体形成是大多数原发性免疫缺陷（PID）疾病中最常见的异常。它最常见的反映是血清免疫球蛋白的减少，这反过来又导致对细菌感染的易感性增加，特别是鼻窦感染。患有这些疾病的个体需要用免疫球蛋白进行替代治疗，以预防或减轻感染的严重程度。多年来，免疫球蛋白替代疗法是通过肌肉注射，然后通过静脉注射（IV）途径。目前，美国绝大多数免疫球蛋白都被许可用于IV给药。在过去的几年里，世界上许多国家都引入了免疫球蛋白制剂的皮下（SC）应用。这种IgG替代疗法被认为是有效、安全的，并且受到患者的高度赞赏，因为与每月IV输注相比，它具有较低的不良反应风险，并且导致较高的血清IgG谷浓度。 本研究的目的是在原发性免疫缺陷（PID）疾病受试者中评价IGIV，10%皮下给药的耐受性和IGIV，10%皮下（SC）治疗后免疫球蛋白G（IgG）的药代动力学。将入组约23例既往接受过静脉注射（IV）免疫球蛋白治疗的12岁及以上PID受试者和约12例既往也接受过IV免疫球蛋白治疗的2至<12岁PID受试者并进行治疗。这项研究将有助于评估这种治疗模式的安全性和有效性。