Ellen & Martin Prosserman Centre for Neuromuscular Diseases, Toronto General Hospital, University Health Network, University of Toronto, Toronto, ON, Canada.
Ellen & Martin Prosserman Centre for Neuromuscular Diseases, Toronto General Hospital, University Health Network, University of Toronto, Toronto, ON, Canada.
Transfus Med Rev. 2022 Oct;36(4):220-229. doi: 10.1016/j.tmrv.2022.05.002. Epub 2022 Aug 28.
Autoimmune neuropathies are often treatable. First-line immunotherapies include intravenous immunoglobulin (IVIG), plasma exchange and corticosteroids. However, nearly 15-30% of patients are either refractory, partially responsive or chronically dependent on these first-line agents. Lack of full response leads to increased disability in addition to adverse financial implications. Consequently, there is an unmet need for more effective treatments to manage this subset of patients. There has been a remarkable increase in the knowledge about immunopathogenesis, antigenic targets, clinical phenotype correlation, and novel therapeutic agents in the last two decades. These novel agents target specific components of the immune system (humoral, cellular immunity, and complement) and have the potential to improve the management of these disorders. Unfortunately, high-quality evidence from large, controlled studies is scarce considering the relative rarity of these refractory cases, heterogeneity of clinical presentations and ethical concerns limiting the use of a placebo arm. An adaptive clinical trial design in a homogenous cohort with standardized outcomes in multiple centers and the use of historical controls will likely provide valuable scientific evidence about the efficacy and safety of these therapies. In this review, we examine the status of the newer immunotherapies in the treatment of autoimmune neuropathies based on existing data.
自身免疫性神经病通常是可治疗的。一线免疫疗法包括静脉注射免疫球蛋白（IVIG）、血浆置换和皮质类固醇。然而，近 15-30%的患者对这些一线药物具有抗药性、部分反应或长期依赖。缺乏完全反应除了带来不良的财务影响外，还会导致残疾程度增加。因此，对于这些患者亚群，存在着对更有效治疗方法的未满足需求。在过去二十年中，人们对免疫发病机制、抗原靶点、临床表型相关性以及新型治疗药物的认识有了显著提高。这些新型药物针对免疫系统的特定成分（体液免疫、细胞免疫和补体），具有改善这些疾病管理的潜力。然而，考虑到这些难治性病例相对罕见、临床表现的异质性以及限制使用安慰剂组的伦理问题，这些病例的高质量、对照研究证据非常有限。在多个中心采用标准化结局的同质队列中进行适应性临床试验设计，并使用历史对照，可能会为这些治疗方法的疗效和安全性提供有价值的科学证据。在这篇综述中，我们根据现有数据，检查了新型免疫疗法在治疗自身免疫性神经病中的现状。
