The Treatment of Primary Immune Deficiencies: Lessons Learned and Future Opportunities.

IF 8.4 2区 医学 Q1 ALLERGY Clinical Reviews in Allergy & Immunology Pub Date : 2023-08-01 Epub Date: 2022-07-01 DOI:10.1007/s12016-022-08950-0
Kenneth Paris, Luke A Wall
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Abstract

Primary immunodeficiency is a group of disorders associated with susceptibility to infectious agents and the development of various comorbidities. Many primary immunodeficiencies are complicated by immune dysregulation, autoinflammation, or autoimmunity which impacts multiple organ systems. Major advances in the treatment of these disorders have occurred over the last half-century, and deeper molecular understanding of many disorders combined with clinically available genetic testing is allowing for use of precision therapy for several primary immunodeficiencies. Patients with antibody deficiencies who rely on immunoglobulin replacement therapy now have many treatment options with products that are much safer and better tolerated compared to the past. Newborn screening for severe combined immunodeficiency, now implemented throughout the USA and in many countries worldwide, has lowered the age at which many patients are diagnosed with these diseases. Early diagnosis of severe combined immunodeficiency allows infants to proceed to definitive therapy such as stem cell transplantation or gene therapy prior to facing potentially life-threatening infections. While stem cell transplantation continues to carry significant risks, knowledge gained over recent decades is allowing for improved survival with less toxicity and less graft versus host disease.

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原发性免疫缺陷的治疗:经验教训与未来机遇。
原发性免疫缺陷是一组与易感染传染病病原体和出现各种合并症有关的疾病。许多原发性免疫缺陷并发免疫失调、自身炎症或自身免疫,影响多个器官系统。在过去的半个世纪中,这些疾病的治疗取得了重大进展,对许多疾病更深入的分子认识与临床上可用的基因检测相结合,使精准治疗在一些原发性免疫缺陷症中得以应用。依靠免疫球蛋白替代疗法的抗体缺乏症患者现在有了许多治疗选择,与过去相比,这些产品更加安全,耐受性更好。新生儿重症联合免疫缺陷筛查目前已在美国和世界许多国家实施,降低了许多患者被诊断出这些疾病的年龄。重症联合免疫缺陷症的早期诊断可以让婴儿在面临可能危及生命的感染之前,接受干细胞移植或基因治疗等明确的治疗。虽然干细胞移植仍有很大风险,但近几十年来获得的知识使移植后的存活率提高,毒性降低,移植物抗宿主疾病减少。
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来源期刊
CiteScore
22.30
自引率
1.10%
发文量
58
审稿时长
6-12 weeks
期刊介绍: Clinical Reviews in Allergy & Immunology is a scholarly journal that focuses on the advancement of clinical management in allergic and immunologic diseases. The journal publishes both scholarly reviews and experimental papers that address the current state of managing these diseases, placing new data into perspective. Each issue of the journal is dedicated to a specific theme of critical importance to allergists and immunologists, aiming to provide a comprehensive understanding of the subject matter for a wide readership. The journal is particularly helpful in explaining how novel data impacts clinical management, along with advancements such as standardized protocols for allergy skin testing and challenge procedures, as well as improved understanding of cell biology. Ultimately, the journal aims to contribute to the improvement of care and management for patients with immune-mediated diseases.
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