Institute of Pathology, University of Würzburg, Würzburg, Germany; Department of Pathology, Chang Gung Memorial Hospital, Chang Gung University College of Medicine, Taoyuan, Taiwan.
Institute of Pathology, University of Würzburg, Würzburg, Germany; Institute of Pathology, University Medical Centre Mannheim, University of Heidelberg, Theodor-Kutzer-Ufer 1-3, D-68135 Mannheim, Germany.
J Autoimmun. 2014 Aug;52:122-9. doi: 10.1016/j.jaut.2013.12.006. Epub 2013 Dec 24.
Late-onset myasthenia gravis (LOMG) has become the largest MG subgroup, but the underlying pathogenetic mechanisms remain mysterious. Among the few etiological clues are the almost unique serologic parallels between LOMG and thymoma-associated MG (TAMG), notably autoantibodies against acetylcholine receptors, titin, ryanodine receptor, type I interferons or IL-12. This is why we checked LOMG patients for two further peculiar features of TAMG - its associations with the CTLA4(high/gain-of-function) +49A/A genotype and with increased thymic export of naïve T cells into the blood, possibly after defective negative selection in AIRE-deficient thymomas. We analyzed genomic DNA from 116 Caucasian LOMG patients for CTLA4 alleles by PCR/restriction fragment length polymorphism, and blood mononuclear cells for recent thymic emigrants by quantitative PCR for T cell receptor excision circles. In sharp contrast with TAMG, we now find that: i) CTLA4(low) +49G(+) genotypes were more frequent (p = 0.0029) among the 69 LOMG patients with age at onset ≥60 years compared with 172 healthy controls; ii) thymic export of naïve T cells from the non-neoplastic thymuses of 36 LOMG patients was lower (p = 0.0058) at diagnosis than in 77 age-matched controls. These new findings are important because they suggest distinct initiating mechanisms in TAMG and LOMG and hint at aberrant immuno-regulation in the periphery in LOMG. We therefore propose alternate defects in central thymic or peripheral tolerance induction in TAMG and LOMG converging on similar final outcomes. In addition, our data support a 60-year-threshold for onset of 'true LOMG' and an LOMG/early-onset MG overlapping group of patients between 40 and 60.
迟发性重症肌无力 (LOMG) 已成为最大的 MG 亚组,但潜在的发病机制仍不清楚。为数不多的病因线索之一是 LOMG 与胸腺瘤相关重症肌无力 (TAMG) 之间几乎独特的血清学相似性,特别是针对乙酰胆碱受体、titin、ryanodine 受体、I 型干扰素或 IL-12 的自身抗体。这就是为什么我们在 LOMG 患者中检查了 TAMG 的另外两个特殊特征 - 其与 CTLA4(高/功能获得) +49A/A 基因型的关联,以及幼稚 T 细胞从胸腺向血液中的过度输出,可能是在 AIRE 缺陷性胸腺瘤中阴性选择缺陷后。我们通过聚合酶链反应/限制性片段长度多态性分析了 116 名高加索 LOMG 患者的基因组 DNA 中的 CTLA4 等位基因,并通过定量聚合酶链反应分析了血液单核细胞中的近期胸腺迁出细胞。与 TAMG 形成鲜明对比的是,我们现在发现:i)与 172 名健康对照相比,发病年龄≥60 岁的 69 名 LOMG 患者中 CTLA4(低) +49G(+) 基因型更为常见 (p=0.0029);ii)36 名 LOMG 患者的非肿瘤性胸腺中的幼稚 T 细胞从胸腺的输出在诊断时比 77 名年龄匹配的对照更低 (p=0.0058)。这些新发现很重要,因为它们表明 TAMG 和 LOMG 中存在不同的起始机制,并暗示 LOMG 中周围免疫调节异常。因此,我们提出 TAMG 和 LOMG 中中枢性或外周性免疫耐受诱导的交替缺陷,导致类似的最终结果。此外,我们的数据支持 60 岁作为“真正的 LOMG”发病的阈值,以及 40 至 60 岁之间的 LOMG/早发性 MG 重叠患者组。