Department of Neurology, University Medical Center Göttingen, Göttingen, Germany.
Curr Opin Rheumatol. 2017 Nov;29(6):632-638. doi: 10.1097/BOR.0000000000000436.
To review new advances in inclusion body myositis (IBM) and discuss them in light of current knowledge on diagnosis, pathomechanisms, and treatment perspectives.
IBM is a treatment refractory inflammatory myopathy in middle-aged patients that leads to a slow, relentlessly progressive muscle weakness, and atrophy. Recent data collections suggest that mortality in IBM patients is somewhat elevated compared with the general population. One major risk factor for death is severe dysphagia, which can now be determined by a novel real-time MRI technique. Recently, proposed diagnostic criteria with a combination of clinical and histopathological features have improved sensitivity and specificity. cytosolic 5'-nucleotidase 1A antibodies have been characterized in IBM patients and their pathophysiologic role has recently been studied. New inflammatory pathomechanisms have been identified in IBM muscle and may help to design novel treatment strategies. A broad spectrum of immunosuppressive and immunomodulatory trials have been conducted, but - so far- no effective treatment is available. Current therapeutic attempts aim to block the myostatin pathway or restore the protein homeostasis.
The expanding knowledge of the complex disease, the refinement of diagnostic criteria, and developments in diagnostic procedures are expected to foster the much needed design of new treatment approaches for future clinical trials.
回顾包涵体肌炎（IBM）的新进展，并根据目前对诊断、发病机制和治疗观点的认识对其进行讨论。
IBM 是一种中年患者中治疗抵抗性的炎症性肌病，导致缓慢、无情进展的肌肉无力和萎缩。最近的数据收集表明，与一般人群相比，IBM 患者的死亡率略高。死亡的一个主要危险因素是严重的吞咽困难，现在可以通过一种新的实时 MRI 技术来确定。最近，提出了一种结合临床和组织病理学特征的诊断标准，提高了敏感性和特异性。在 IBM 患者中已经鉴定出胞质 5'-核苷酸酶 1A 抗体，其病理生理作用最近也得到了研究。在 IBM 肌肉中已经确定了新的炎症发病机制，这可能有助于设计新的治疗策略。已经进行了广泛的免疫抑制和免疫调节试验，但到目前为止，还没有有效的治疗方法。目前的治疗尝试旨在阻断肌肉生长抑制素途径或恢复蛋白质平衡。
对复杂疾病认识的不断扩展、诊断标准的细化以及诊断程序的发展，有望促进为未来临床试验设计急需的新治疗方法。
