Li Xiao, Golubovsky Josh, Hui-Yuen Joyce, Shah Ummara, Olech Ewa, Lomeo Rosalia, Singh Vijay, Busch Howard, Strandberg Mary Jane, Strandberg Kayla, Horowitz Leslie, Askanase Anca
Division of Adult Rheumatology, Columbia University Medical Center, New York, NY, 10032, USA.
Division of Pediatric Rheumatology, Columbia University Medical Center, New York, NY, 10032, USA.
F1000Res. 2015 Oct 23;4:1103. doi: 10.12688/f1000research.7192.2. eCollection 2015.
Acthar Gel is a long-acting formulation of adrenocorticotropic hormone (ACTH) with anti-inflammatory effects thought to be mediated in part through melanocortin receptor activation. This study was initiated to understand the role of Acthar Gel in SLE treatment in rheumatology practices.
This is a retrospective case series of nine adult female patients treated with Acthar Gel for at least six months at five academic centers. Treating physicians completed a one-page questionnaire on lupus medications, disease activity, and outcomes. Clinical response was defined using SLEDAI 2K and improvement in the clinical manifestation(s) being treated.
The most common clinical SLE manifestations/indications requiring therapy with Acthar Gel were arthritis, rash, and inability to taper corticosteroids. The mean SLEDAI 2K score at baseline was 5.8 ± 5.0 (range 0-16). Six patients were concomitantly treated with corticosteroids (mean dose 18.3mg/day). All patients were on background SLE medications including immunosuppressives. Seven of nine patients had an overall improvement, with a decrease in SLEDAI 2K from 5.8 ± 5.0 at baseline to 3.5 ± 2.7 (range 0-8); four of five patients had improvement or resolution in arthritis, and one of two patients had resolution of inflammatory rash. Four patients discontinued corticosteroids and one patient tapered below 50% of the initial dose by 3 months of treatment with Acthar Gel. No adverse events were reported.
This study suggests a role for Acthar Gel as an alternative to corticosteroids in the treatment of SLE. Acthar Gel appears to be safe and well-tolerated after 6 months of treatment, with a significant reduction in disease activity.
Acthar Gel是促肾上腺皮质激素(ACTH)的长效制剂,具有抗炎作用,其抗炎作用部分被认为是通过黑素皮质素受体激活介导的。开展本研究以了解Acthar Gel在风湿病实践中治疗系统性红斑狼疮(SLE)的作用。
这是一项回顾性病例系列研究,纳入了5个学术中心接受Acthar Gel治疗至少6个月的9例成年女性患者。治疗医生完成了一份关于狼疮用药、疾病活动度和结局的单页问卷。使用SLEDAI 2K定义临床反应以及所治疗的临床表现的改善情况。
需要使用Acthar Gel治疗的最常见SLE临床表现/指征为关节炎、皮疹以及无法减少皮质类固醇用量。基线时SLEDAI 2K评分的平均值为5.8±5.0(范围0 - 16)。6例患者同时接受皮质类固醇治疗(平均剂量18.3mg/天)。所有患者均接受包括免疫抑制剂在内的背景SLE药物治疗。9例患者中有7例整体情况改善,SLEDAI 2K评分从基线时的5.8±5.0降至3.5±2.7(范围0 - 8);5例关节炎患者中有4例病情改善或缓解,2例炎症性皮疹患者中有1例皮疹消退。4例患者停用皮质类固醇,1例患者在接受Acthar Gel治疗3个月后将皮质类固醇剂量减至初始剂量的50%以下。未报告不良事件。
本研究提示Acthar Gel在SLE治疗中可作为皮质类固醇的替代药物。治疗6个月后,Acthar Gel似乎安全且耐受性良好,疾病活动度显著降低。