Quinot Valérie, Rostasy Kevin, Höftberger Romana
Division of Neuropathology and Neurochemistry, Department of Neurology, Medical University of Vienna, 1090 Vienna, Austria.
Department of Pediatric Neurology, Children's Hospital Datteln, University Witten/Herdecke, 45711 Datteln, Germany.
J Clin Med. 2024 Sep 25;13(19):5721. doi: 10.3390/jcm13195721.
The recent discovery of pathogenic antibodies targeting cell adhesion molecules of the node of Ranvier has prompted efforts to develop a new classification for a subset of antibody-mediated peripheral neuropathies. These autoimmune nodo- and paranodopathies encompass epitopes such as neurofascin 155, neurofascin 186, contactin-1, and contactin-associated protein 1, with a high likelihood of involving additional yet unidentified proteins. So far, the investigation of this subset of patients was primarily focused on adults, with only rare reports of pediatric cases. Low awareness among pediatricians and insufficient availability of appropriate diagnostic methods in many laboratories may mask a higher pediatric incidence than currently observed. Diagnosis is made by transfected cell-based assays and ELISA to characterize the specific target antigen and antibody subclass that provides insight into the pathophysiology. Clinical features often resemble those of CIDP or GBS in adults, whilst in pediatric patients, although rare, an atypical CIDP phenotype has predominantly been reported. Yet, in contrast to classical immune-mediated neuropathies, the clinical course is usually rapidly progressive, and response to classical first-line therapy often poor. Although electrophysiological signs of demyelination are observed, segmental demyelination and inflammation are not present on pathological examination. Rather, few neuropathological reports demonstrate features of axonal neuropathy without signs of true de- or remyelination. This review aims to summarize recent findings on such nodo- and paranodoneuropathies, shining light on features of these disorders in pediatric patients, a still little-explored field with only a few reports currently present.
最近发现针对郎飞结细胞粘附分子的致病性抗体,促使人们努力为抗体介导的周围神经病的一个子集制定新的分类标准。这些自身免疫性郎飞结和旁结病包括神经束膜蛋白155、神经束膜蛋白186、接触蛋白-1和接触蛋白相关蛋白1等表位,很可能还涉及其他尚未确定的蛋白质。到目前为止,对这一子集患者的研究主要集中在成人,儿科病例报告很少。儿科医生的认识不足以及许多实验室缺乏合适的诊断方法,可能掩盖了比目前观察到的更高的儿科发病率。通过基于转染细胞的检测和酶联免疫吸附测定来诊断,以表征特定的靶抗原和抗体亚类,从而深入了解病理生理学。临床特征通常类似于成人的慢性炎症性脱髓鞘性多发性神经病(CIDP)或吉兰-巴雷综合征(GBS),而在儿科患者中,虽然罕见,但主要报告的是非典型CIDP表型。然而,与经典的免疫介导性神经病不同,临床病程通常进展迅速,对经典一线治疗的反应往往较差。虽然观察到脱髓鞘的电生理迹象,但病理检查中不存在节段性脱髓鞘和炎症。相反,很少有神经病理学报告显示轴索性神经病的特征,而没有真正的脱髓鞘或髓鞘再生迹象。这篇综述旨在总结关于此类郎飞结和旁结神经病的最新发现,揭示这些疾病在儿科患者中的特征,这一领域仍未得到充分探索,目前仅有少数报告。