Chandra Tanya, Aggarwal Rohit
Georgetown University, Washington, DC, USA.
University of Pittsburgh, 3601 5th Avenue, Suite 2B, Pittsburgh, PA, 15261, USA.
Rheumatol Ther. 2023 Jun;10(3):523-537. doi: 10.1007/s40744-023-00545-1. Epub 2023 Mar 26.
Idiopathic inflammatory myopathies (IIMs) are autoimmune disorders characterized by symmetric proximal muscle weakness and chronic inflammation, with an increased risk of morbidity and mortality. The current standard of care includes traditional immunosuppressive pharmacotherapies; however, some patients cannot tolerate or do not adequately respond to these therapies, highlighting the need for alternative treatments for refractory disease. Acthar Gel (repository corticotropin injection) is a naturally sourced mixture of adrenocorticotropic hormone analogs and other pituitary peptides that has been approved by the US Food and Drug Administration since 1952 for use in patients with two subgroups of IIMs, dermatomyositis (DM) and polymyositis (PM). However, it has not been routinely used in the treatment of IIMs. While Acthar may induce steroidogenesis, it also has a steroid-independent mechanism of action by exerting immunomodulatory effects through the activation of melanocortin receptors on immune cells, such as macrophages, B cells, and T cells. Recent clinical trials, retrospective analyses, and case reports add to the growing evidence suggesting that Acthar may be effective in patients with DM and PM. Here we review the current evidence supporting the safety and efficacy of Acthar for the treatment of refractory DM and PM.
特发性炎性肌病（IIMs）是一种自身免疫性疾病，其特征为对称性近端肌无力和慢性炎症，发病和死亡风险增加。当前的治疗标准包括传统免疫抑制药物疗法；然而，一些患者无法耐受这些疗法或对其反应不佳，这凸显了对难治性疾病进行替代治疗的必要性。促皮质素凝胶（长效促肾上腺皮质激素注射液）是一种天然来源的促肾上腺皮质激素类似物和其他垂体肽的混合物，自1952年以来已被美国食品药品监督管理局批准用于IIMs的两个亚组，即皮肌炎（DM）和多发性肌炎（PM）的患者。然而，它尚未常规用于IIMs的治疗。虽然促皮质素可能诱导类固醇生成，但它也具有不依赖类固醇的作用机制，通过激活免疫细胞（如巨噬细胞、B细胞和T细胞）上的黑皮质素受体发挥免疫调节作用。最近的临床试验、回顾性分析和病例报告进一步证明，促皮质素可能对DM和PM患者有效。在此，我们综述了支持促皮质素治疗难治性DM和PM的安全性和有效性的现有证据。
