Mastaglia F L
Centre for Neuromuscular and Neurological Disorders, University of Western Australia, Queen Elizabeth II Medical Centre, Nedlands 6009, Australia.
Neurol India. 2008 Jul-Sep;56(3):263-70. doi: 10.4103/0028-3886.43444.
The three major immune-mediated inflammatory myopathies, dermatomyositis (DM), polymyositis (PM) and inclusion body myositis (IBM), each have their own distinctive clinical features, underlying pathogenetic mechanisms and patterns of muscle gene expression. In DM a complement-dependent humoral process thought to be initiated by antibodies to endothelial cells results in a microangiopathy with secondary ischemic changes in muscles. On the other hand, in PM and IBM there is a T-cell response with invasion of muscle fibers by CD8+ lymphocytes and perforin-mediated cytotoxic necrosis. In IBM degenerative changes are also a feature and comprise autophagia with rimmed vacuole formation and inclusions containing beta-amyloid and other proteins whose accumulation may be linked to impaired proteasomal function. The relationship between the inflammatory and degenerative component remains unclear, as does the basis for the selective vulnerability of certain muscles and the resistance to conventional forms of immunotherapy in most cases of IBM. Patients with DM or PM usually respond to treatment with glucocorticoids and immunosuppressive agents but their use remains largely empirical. Intravenous immunoglobulin therapy can be used to achieve disease control in patients with severe weakness or dysphagia, or in patients with immunodeficiency, but its use is limited by expense. Emerging therapies for resistant cases include TNFalpha inhibitors (etanercept, infliximab) and monoclonal antibodies (rituximab, alemtuzumab). However, experience with these therapies is still limited and there is a need for randomized trials to test their efficacy and establish guidelines for their use in clinical practice.
三种主要的免疫介导性炎性肌病,即皮肌炎(DM)、多发性肌炎(PM)和包涵体肌炎(IBM),各自具有独特的临床特征、潜在发病机制和肌肉基因表达模式。在DM中,一种由抗内皮细胞抗体引发的补体依赖性体液过程导致微血管病,并继发肌肉缺血性改变。另一方面,在PM和IBM中,存在T细胞反应,CD8+淋巴细胞侵入肌纤维并通过穿孔素介导细胞毒性坏死。在IBM中,退行性改变也是一个特征,包括自噬伴镶边空泡形成以及含有β-淀粉样蛋白和其他蛋白质的包涵体,这些蛋白质的积累可能与蛋白酶体功能受损有关。炎症和退行性成分之间的关系仍不清楚,某些肌肉选择性易损性的基础以及大多数IBM病例对传统免疫疗法耐药的原因也不清楚。DM或PM患者通常对糖皮质激素和免疫抑制剂治疗有反应,但这些药物的使用很大程度上仍基于经验。静脉注射免疫球蛋白疗法可用于控制严重肌无力或吞咽困难患者或免疫缺陷患者的病情,但其使用受到费用限制。针对耐药病例的新兴疗法包括肿瘤坏死因子α抑制剂(依那西普、英夫利昔单抗)和单克隆抗体(利妥昔单抗、阿仑单抗)。然而,这些疗法的经验仍然有限,需要进行随机试验以测试其疗效并制定临床实践中使用的指南。