Division of Rheumatology, Allergy, and Immunology, Department of Medicine, University of North Carolina School of Medicine, Chapel Hill, NC, 27599, USA,
Curr Allergy Asthma Rep. 2014 Sep;14(9):462. doi: 10.1007/s11882-014-0462-4.
Autoimmune lymphoproliferative syndrome (ALPS) is characterized by immune dysregulation due to a defect in lymphocyte apoptosis. The clinical manifestations may be noted in multiple family members and include lymphadenopathy, splenomegaly, increased risk of lymphoma, and autoimmune disease, which typically involves hematopoietic cell lines manifesting as multilineage cytopenias. Since the disease was first characterized in the early 1990s, there have been many advances in the diagnosis and management of this syndrome. The inherited genetic defect of many ALPS patients has involved (FAS) pathway signaling proteins, but there remain those patients who carry undefined genetic defects. Despite ALPS having historically been considered a primary immune defect presenting in early childhood, adult onset presentation is increasingly becoming recognized and more so in genetically undefined patients and those with somatic FAS mutations. Thus, future research may identify novel pathways and/or regulatory proteins important in lymphocyte activation and apoptosis.
自身免疫性淋巴组织增生综合征(ALPS)的特征是由于淋巴细胞凋亡缺陷导致的免疫失调。临床表现可在多个家庭成员中观察到,包括淋巴结病、脾肿大、淋巴瘤风险增加和自身免疫性疾病,这些疾病通常涉及造血细胞系,表现为多谱系血细胞减少症。自 20 世纪 90 年代初首次描述该疾病以来,在该综合征的诊断和治疗方面取得了许多进展。许多 ALPS 患者的遗传性遗传缺陷涉及(FAS)途径信号蛋白,但仍有一些患者携带未定义的遗传缺陷。尽管 ALPS 历史上被认为是一种在儿童早期出现的原发性免疫缺陷,但越来越多的成年发病病例得到了认识,尤其是在遗传上未定义的患者和具有体细胞 FAS 突变的患者中。因此,未来的研究可能会确定在淋巴细胞激活和凋亡中起重要作用的新途径和/或调节蛋白。