From the Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand; and.
Immunology Service, Department of Laboratory Medicine, National Institutes of Health Clinical Center, National Institutes of Health, Bethesda, Maryland.
Allergy Asthma Proc. 2024 Sep 1;45(5):355-363. doi: 10.2500/aap.2024.45.240051.
Primary immunodeficiency diseases encompass a variety of genetic conditions characterized by a compromised immune system and typically results in increased susceptibility to infection. In fact, they also manifest as autoimmunity, autoinflammation, atopic diseases, and malignancy. Currently, the number of recognized monogenic primary immunodeficiency disorders is set at ∼500 different entities, owing to the exponential use of unbiased genetic testing for disease discovery. In addition, the prevalence of secondary immunodeficiency has also been on the rise due to the increased use of immunosuppressive drugs to treat diseases based on immune dysregulation, an increase in the number of individuals undergoing hematopoietic stem cell transplantation, and other chronic medical conditions, including autoimmunity. Although the clinical symptoms of immunodeficiency disorders are broad, an early diagnosis and tailored management strategies are essential to mitigate the risk of infections and prevent disease-associated morbidity. Generally, the medical history and physical examination can provide useful information that can help delineate the possibility of immune defects. In turn, this makes it feasible to select focused laboratory tests that identify immunodeficiency disorders based on the specific immune cells and their functions or products that are affected. Laboratory evaluation involves quantitative and functional classic testing (e.g., leukocyte counts, serum immunoglobulin levels, specific antibody titers in response to vaccines, and enumeration of lymphocyte subsets) as well as genetic testing (e.g., individual gene evaluation via Sanger sequencing or unbiased evaluation based on next-generation sequencing). However, in many cases, a diagnosis also requires additional advanced research techniques to validate genetic or other findings. This article updates clinicians about available laboratory tests for evaluating the immune system in patients with primary immunodeficiency disorders. It also provides a comprehensive list of testing options, organized based on different components of host defense.
原发性免疫缺陷病涵盖了多种以免疫系统受损为特征的遗传疾病,通常会导致感染易感性增加。事实上,它们还表现为自身免疫、自身炎症、特应性疾病和恶性肿瘤。目前,由于非靶向性遗传检测在疾病发现中的广泛应用,已确定的单基因原发性免疫缺陷疾病的数量约为 500 种不同的实体。此外,由于基于免疫失调的疾病治疗中免疫抑制药物的使用增加、接受造血干细胞移植的人数增加以及其他慢性医疗条件(包括自身免疫)的增加,继发性免疫缺陷的患病率也在上升。尽管免疫缺陷疾病的临床症状广泛,但早期诊断和个体化管理策略对于降低感染风险和预防与疾病相关的发病率至关重要。通常,病史和体格检查可以提供有用的信息,有助于明确免疫缺陷的可能性。反过来,这使得根据受影响的特定免疫细胞及其功能或产物选择有针对性的实验室检测来识别免疫缺陷疾病成为可能。实验室评估包括定量和功能经典检测(例如,白细胞计数、血清免疫球蛋白水平、疫苗接种后的特异性抗体滴度以及淋巴细胞亚群计数)以及遗传检测(例如,通过 Sanger 测序进行个体基因评估或基于下一代测序的非靶向评估)。然而,在许多情况下,诊断还需要额外的先进研究技术来验证遗传或其他发现。本文更新了临床医生对原发性免疫缺陷病患者免疫系统评估的可用实验室检测方法。它还提供了一个全面的测试选项列表,根据宿主防御的不同组成部分进行组织。