Muscle Immunobiology Group, Neuromuscular Center, Department of Neurology, University Medical Center Göttingen, Göttingen, Germany.
Department of Neurology and Pain Treatment, Immanuel Klinik Rüdersdorf, University Hospital of the Brandenburg Medical School Theodor Fontane, Rüdersdorf bei Berlin, Germany.
Neurotherapeutics. 2022 Apr;19(3):911-921. doi: 10.1007/s13311-022-01220-z. Epub 2022 Apr 8.
Inflammatory myopathies, including polymyositis (PM), dermatomyositis (DM), inclusion body myositis (IBM), necrotizing myopathy (NM), antisynthetase syndrome (ASS) and overlap myositis (OM), in short myositis, are rare diseases. All forms of myositis have progressive muscle weakness in common, with each subtype characterized by different autoantibody profiles, histological findings and extramuscular manifestations. Due to better understanding of the pathogenesis of the muscle inflammation in myositis, new molecular pathways for targeted therapy have been discovered. Current therapies aim at different components of the innate or the adaptive immune response. Additionally, non-inflammatory mechanisms in myositis have come into focus as possible treatment targets. The use of therapeutical antibodies in myositis has been examined in various clinical studies, several of them randomized controlled ones: Depletion of B-cells by rituximab has been established as treatment of refractory myositis. IVIG, an antibody therapy in the wider sense, has now been licensed for DM following a recent positive clinical trial. Negative study results were reported in randomized trials with infliximab, sifalimumab and bimagrumab. Studies on basiliximab and eculizumab are currently underway, and are expected to yield results in a couple of years. Despite some promising results of clinical studies with antibody therapy in myositis, further research is crucial to optimize the treatment for this debilitating disease and to find treatment alternatives for treatment-refractory patients.
炎性肌病,包括多发性肌炎(PM)、皮肌炎(DM)、包涵体肌炎(IBM)、坏死性肌病(NM)、抗合成酶综合征(ASS)和重叠性肌炎(OM),简称肌炎,是罕见疾病。所有形式的肌炎都有进行性肌无力的共同特征,每种亚型都有不同的自身抗体谱、组织学发现和肌肉外表现。由于对肌炎肌肉炎症发病机制的更好理解,已经发现了新的靶向治疗分子途径。目前的治疗方法针对固有或适应性免疫反应的不同成分。此外,肌炎中的非炎症机制已成为可能的治疗靶点。在各种临床研究中已经检查了治疗性抗体在肌炎中的应用,其中一些是随机对照研究:利妥昔单抗对 B 细胞的耗竭已被确立为治疗难治性肌炎的方法。静脉注射免疫球蛋白(IVIG),广义上的抗体治疗,在最近的一项阳性临床试验后已被批准用于 DM。在随机试验中报告了英夫利昔单抗、西法利单抗和比马鲁单抗的阴性研究结果。巴利昔单抗和依库珠单抗的研究正在进行中,预计在几年内会有结果。尽管肌炎抗体治疗的临床研究有一些有希望的结果,但进一步的研究对于优化这种使人衰弱的疾病的治疗以及为治疗抵抗的患者寻找治疗替代方案至关重要。