Wan George J, Niewoehner John, Hayes Kyle
Mallinckrodt Pharmaceuticals, Bridgewater, NJ, USA.
Clinicoecon Outcomes Res. 2023 Jun 26;15:499-512. doi: 10.2147/CEOR.S410082. eCollection 2023.
Acthar Gel (repository corticotropin injection [RCI]) is a naturally sourced complex mixture of adrenocorticotropic hormone analogs and other pituitary peptides used to treat patients with serious and rare inflammatory and autoimmune conditions. This narrative review summarizes the key clinical and economic findings among 9 indications: infantile spasms (IS), multiple sclerosis (MS) relapses, rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), dermatomyositis and polymyositis (DM/PM), ocular inflammatory diseases (primarily uveitis and severe keratitis), symptomatic sarcoidosis, and proteinuria in nephrotic syndrome (NS). Key studies of clinical efficacy and healthcare resource utilization and cost from 1956 to 2022 are discussed. Evidence supports the efficacy of RCI across all 9 indications. RCI is recommended as first-line treatment for IS and is associated with improved outcomes for the other 8 indications, including increased recovery rates in MS relapse; improved disease control in RA, SLE, and DM/PM; real-world effectiveness in patients with uveitis and severe keratitis; improved lung function and reduced corticosteroid use in symptomatic sarcoidosis; and increased rates of partial remission of proteinuria in NS. For many indications, RCI may improve clinical outcomes during exacerbations or when conventional treatments have failed to show a benefit. RCI is also associated with a reduction in the use of biologics, corticosteroids, and disease-modifying antirheumatic drugs. Economic data suggest RCI is a cost-effective, value-based treatment option for MS relapse, RA, and SLE. Other economic benefits have been demonstrated for IS, MS relapses, RA, SLE, and DM/PM, including reduced hospitalizations, lengths of stay, inpatient and outpatient services, and emergency department visits. RCI is considered safe and effective and features economic benefits for numerous indications. Its ability to control relapse and disease activity makes RCI an important nonsteroid treatment option that could help preserve functioning and well-being among patients with inflammatory and autoimmune conditions.
促肾上腺皮质激素凝胶（长效促肾上腺皮质激素注射液 [RCI]）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复杂混合物，用于治疗严重和罕见的炎症及自身免疫性疾病患者。本叙述性综述总结了9种适应症的关键临床和经济研究结果：婴儿痉挛症（IS）、多发性硬化症（MS）复发、类风湿性关节炎（RA）、系统性红斑狼疮（SLE）、皮肌炎和多发性肌炎（DM/PM）、眼部炎症性疾病（主要是葡萄膜炎和严重角膜炎）、症状性结节病以及肾病综合征（NS）中的蛋白尿。讨论了1956年至2022年临床疗效、医疗资源利用和成本的关键研究。证据支持RCI在所有9种适应症中的疗效。RCI被推荐作为IS的一线治疗药物，并且与其他8种适应症的改善结果相关，包括MS复发时恢复率提高；RA、SLE和DM/PM疾病控制改善；葡萄膜炎和严重角膜炎患者的真实世界疗效；症状性结节病患者肺功能改善和皮质类固醇使用减少；以及NS中蛋白尿部分缓解率提高。对于许多适应症，RCI可能在病情加重期间或传统治疗未显示益处时改善临床结果。RCI还与生物制剂、皮质类固醇和改善病情抗风湿药的使用减少有关。经济数据表明，RCI是MS复发、RA和SLE具有成本效益、基于价值的治疗选择。IS、MS复发、RA、SLE和DM/PM也显示出其他经济效益，包括住院次数、住院时间、住院和门诊服务以及急诊科就诊次数减少。RCI被认为是安全有效的，并且在许多适应症中具有经济效益。其控制复发和疾病活动的能力使RCI成为一种重要的非甾体治疗选择，有助于维持炎症和自身免疫性疾病患者的功能和健康。
