Peddi Nikhil Chowdary, Vuppalapati Sravya, Sreenivasulu Himabindu, Muppalla Sudheer Kumar, Reddy Pulliahgaru Apeksha
Pediatrics, Saint Barnabas Hospital Health System, New York, USA.
General Physician, People's Education Society (PES) Institute of Medical Sciences and Research, Kuppam, IND.
Cureus. 2023 Sep 7;15(9):e44865. doi: 10.7759/cureus.44865. eCollection 2023 Sep.
Primary immunodeficiency disorders (PIDs) are a heterogeneous group of genetic conditions profoundly impacting immune function. The investigation spans various PID categories, offering insights into their distinct pathogenic mechanisms and clinical manifestations. Within the adaptive immune system, B-cell, T-cell, and combined immunodeficiencies are dissected, emphasizing their critical roles in orchestrating effective immune responses. In the realm of the innate immune system, focus is directed toward phagocytes and complement deficiencies, underscoring the pivotal roles of these components in initial defense against infections. Furthermore, the review delves into disorders of immune dysregulation, encompassing hemophagocytic lymphohistiocytosis (HLH), autoimmune lymphoproliferative syndrome (ALPS), immune dysregulation, polyendocrinopathy, enteropathy, and X-linked(IPEX), and autoimmunity polyendocrinopathy candidiasis-ectodermal dystrophy(APECED), elucidating the intricate interplay between immune tolerance and autoimmunity prevention. Diagnostic strategies for PIDs are explored, highlighting advancements in genetic and molecular techniques that enable precise identification of underlying genetic mutations and alterations in immune function. We have also outlined treatment modalities for PIDs, which often entail a multidisciplinary approach involving immunoglobulin replacement, antimicrobial prophylaxis, and, in select cases, hematopoietic stem cell transplantation. Emerging therapies, including gene therapy, hold promise for targeted interventions. In essence, this review encapsulates the complexity of PIDs, emphasizing the critical importance of early diagnosis and tailored therapeutic interventions. As research advances, a clearer understanding of these disorders emerges, fostering optimism for enhanced patient care and management in the future.
原发性免疫缺陷病(PIDs)是一组异质性的遗传疾病,对免疫功能有深远影响。该研究涵盖了各种PID类别,深入了解其独特的致病机制和临床表现。在适应性免疫系统中,对B细胞、T细胞和联合免疫缺陷进行了剖析,强调了它们在协调有效免疫反应中的关键作用。在先天性免疫系统领域,重点关注吞噬细胞和补体缺陷,突出了这些成分在抵御感染的初始防御中的关键作用。此外,该综述深入探讨了免疫失调疾病,包括噬血细胞性淋巴组织细胞增生症(HLH)、自身免疫性淋巴细胞增生综合征(ALPS)、免疫失调、多内分泌腺病、肠病和X连锁(IPEX),以及自身免疫性多内分泌腺病念珠菌病-外胚层营养不良(APECED),阐明了免疫耐受与自身免疫预防之间的复杂相互作用。探索了PID的诊断策略,强调了遗传和分子技术的进展,这些技术能够精确识别潜在的基因突变和免疫功能改变。我们还概述了PID的治疗方式,这通常需要多学科方法,包括免疫球蛋白替代、抗菌预防,以及在某些情况下的造血干细胞移植。包括基因治疗在内的新兴疗法有望实现靶向干预。本质上,这篇综述概括了PID的复杂性,强调了早期诊断和量身定制的治疗干预的至关重要性。随着研究的进展,对这些疾病的理解更加清晰,为未来改善患者护理和管理带来了希望。