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免疫缺陷:原发性免疫调节紊乱疾病(PIRDs)概述。

Immunodeficiency: Overview of primary immune regulatory disorders (PIRDs).

机构信息

From the Division of Allergy and Clinical Immunology, Brigham and Women's Hospital, Boston, Massachusetts; Harvard Medical School, Boston, Massachusetts; and.

出版信息

Allergy Asthma Proc. 2024 Sep 1;45(5):332-339. doi: 10.2500/aap.2024.45.240070.

Abstract

Primary immune regulatory disorders (PIRD) comprise a heterogeneous group of monogenic disorders that impact homeostatic control of inflammation and immune tolerance. Patients with a PIRD typically present to medical care with symptoms of autoimmunity or hyperinflammation as the dominant clinical feature, symptoms that include fever, rash, lymphadenopathy, organomegaly, arthritis, and colitis are commonplace. Notably, PIRDs are a distinct entity from primary immune deficiency disorders (PIDD), which are primarily defined by a qualitative or quantitative defect in immunity, which manifests as a susceptibility to recurrent infections. PIDDs and PIRDs can be challenging to differentiate because the clinical presentations can be similar. Red flags for PIRDs include multiple autoimmune diagnoses in the same patient, chronic lymphadenopathy, hepatomegaly, and/or splenomegaly, chronic colitis, hemophagocytic lymphohistiocytosis (HLH), Epstein Barr virus (EBV) susceptibility, recurrent or persistent fever, vasculitis, and sterile inflammation. For simplicity in this brief review, we limit our discussion of PIRDs to the following categories multiple autoimmune diseases, immune dysregulation with colitis, disorders with HLH and/or EBV susceptibility, autoinflammatory syndromes, type 1 interferonopathies, and disorders of sterile inflammation. Diagnosing a PIRD requires a broad immune evaluation for both immune system deficiencies and inflammation, along with genetic testing. Given the complex nature of these diseases, treatment often requires a team of subspecialists. Treatment, depending on the specific diagnosis, may be somewhat empiric with nonspecific immune modulators, symptom-directed therapies, and, in severe cases, hematopoietic stem cell transplantation; however, with the increasing number of biologics available, we are often able to use targeted immune therapy or even gene therapy.

摘要

原发性免疫调节障碍(PIRD)包括一组异质性的单基因疾病,这些疾病影响炎症和免疫耐受的体内平衡控制。患有 PIRD 的患者通常因自身免疫或过度炎症为主要临床特征而就诊于医疗机构,这些症状包括发热、皮疹、淋巴结病、器官肿大、关节炎和结肠炎等。值得注意的是,PIRDs 与原发性免疫缺陷病(PIDD)不同,后者主要定义为免疫的定性或定量缺陷,表现为易患复发性感染。PIDD 和 PIRDs 很难区分,因为临床表现可能相似。PIRDs 的警示信号包括同一患者的多种自身免疫诊断、慢性淋巴结病、肝肿大和/或脾肿大、慢性结肠炎、噬血细胞性淋巴组织细胞增生症(HLH)、EB 病毒(EBV)易感性、反复或持续发热、血管炎和无菌性炎症。为了在本简要综述中简明扼要,我们将 PIRDs 的讨论限于以下类别:多种自身免疫性疾病、伴结肠炎的免疫失调、伴有 HLH 和/或 EBV 易感性的疾病、自身炎症综合征、1 型干扰素病和无菌性炎症。诊断 PIRD 需要对免疫系统缺陷和炎症进行广泛的免疫评估,并进行基因检测。鉴于这些疾病的复杂性质,治疗通常需要一个多学科专家团队。根据具体诊断,治疗可能是针对某些疾病的经验性治疗,包括非特异性免疫调节剂、对症治疗,在严重情况下还需要进行造血干细胞移植;然而,随着越来越多的生物制剂可用,我们通常能够使用靶向免疫治疗甚至基因治疗。

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本文引用的文献

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Immunodysregulation in immunodeficiency.免疫缺陷中的免疫失调。
Allergy Asthma Proc. 2024 Sep 1;45(5):340-346. doi: 10.2500/aap.2024.45.240058.
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Too much of a good thing: a review of primary immune regulatory disorders.过犹不及:原发性免疫调节紊乱综述。
Front Immunol. 2023 Oct 31;14:1279201. doi: 10.3389/fimmu.2023.1279201. eCollection 2023.
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Inborn Errors of Immunity.遗传性免疫缺陷病
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STAT3 gain-of-function syndrome.信号转导与转录激活因子3功能获得性综合征
Front Pediatr. 2023 Feb 9;10:770077. doi: 10.3389/fped.2022.770077. eCollection 2022.
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Towards gene therapy for IPEX syndrome.针对 IPEX 综合征的基因治疗。
Eur J Immunol. 2022 May;52(5):705-716. doi: 10.1002/eji.202149210. Epub 2022 Apr 13.

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