National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD, USA.
Muscle Disease Unit, National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD, USA.
Expert Rev Clin Immunol. 2024 Feb;20(2):197-209. doi: 10.1080/1744666X.2023.2270737. Epub 2024 Jan 21.
Dermatomyositis (DM) is a rare inflammatory disease with diverse cutaneous and systemic manifestations, often associated with myositis-specific antibodies. Managing patients with refractory DM, or individuals presenting pecific complications, like calcinosis or rapidly progressive interstitial lung disease, presents unique challenges.
This review explores current and promising treatment options for DM, drawing from clinical studies, case series, and case reports that consider the underlying disease pathophysiology.
Recent advancements have improved our understanding and management of DM. The discovery of distinct DM autoantibodies and their correlation with specific clinical phenotypes has transformed patient categorization and enhanced our knowledge of the pathogenesis of the disease. Intravenous immunoglobulin, a well-established treatment in dermatomyositis, has regained prominence and a large randomized clinical trial has reaffirmed its efficacy, confirming it as an effective therapeutic option in this group of patients. Identification of the type I interferon pathway as a key pathogenic mechanism in DM has opened up new avenues for more effective treatment strategies. Blocking the JAK/STAT pathway offers potential for improved management of refractory patients and prevention of highly morbid complications. These recent advancements have significantly impacted the management and care of dermatomyositis patients, enabling tailored approaches, targeted interventions, and improved outcomes for individuals affected by this complex condition.
皮肌炎(DM)是一种罕见的炎症性疾病,具有多种皮肤和系统表现,常与肌炎特异性抗体相关。对于难治性皮肌炎患者或出现特定并发症(如钙沉积或快速进展性间质性肺病)的患者,管理上存在独特的挑战。
本综述探讨了当前和有前途的 DM 治疗选择,参考了考虑疾病病理生理学的临床研究、病例系列和病例报告。
最近的进展提高了我们对 DM 的认识和管理水平。发现独特的 DM 自身抗体及其与特定临床表型的相关性改变了患者分类,并增强了我们对疾病发病机制的了解。静脉注射免疫球蛋白是皮肌炎的一种成熟治疗方法,其重要性再次得到认可,一项大型随机临床试验再次证实了其疗效,确认其是这组患者的有效治疗选择。确定 I 型干扰素途径是 DM 的关键致病机制,为更有效的治疗策略开辟了新途径。阻断 JAK/STAT 途径为治疗难治性患者和预防高度病态并发症提供了可能。这些最近的进展极大地影响了皮肌炎患者的管理和护理,为受这种复杂疾病影响的个体提供了个性化的方法、针对性的干预和更好的结果。