Department of Medicine, University of Pittsburgh, Pittsburgh, PA, USA.
Division of Rheumatology and Clinical Immunology, Department of Medicine, University of Pittsburgh, Pittsburgh, PA, USA.
Rheumatology (Oxford). 2020 Oct 1;59(10):2976-2981. doi: 10.1093/rheumatology/keaa076.
Idiopathic inflammatory myopathies (IIM) are a group of autoimmune diseases characterized by proximal muscle weakness. H. P. Acthar gel [repository corticotropin injection (RCI)] is a formulation of adrenocorticotropic hormone and has been approved by Food and Drug Administration for use in IIM; however, literature is limited. In this study, we report longitudinal follow-up of myositis patients treated with RCI.
Patients with refractory IIM who were enrolled in the prospective, open-label RCI trial were included in this study. The post-trial follow-up period was 6 months with assessments every 2 months, which included myositis core set measures including extra-muscular global, muscle and patient global disease activities, HAQ, and manual muscle testing.
Two patients were lost to follow-up after finalization of the trial, and the remaining eight patients were enrolled in the follow-up study. One patient remained on RCI after the trial. In the follow-up period, four of eight patients had flare at on average 4.1 months after the RCI trial. Among the patients who flared, three required an increase in prednisone. One patient was restarted on RCI at 5.5 months, but had minimal improvement after 3 months. Four patients who remained stable continued to satisfy criteria for the definition of improvement through the 6-month follow-up. However, none showed any further improvement in the primary or secondary efficacy outcomes after the initial RCI trial.
To our knowledge, this is the first study reporting the follow-up results of patients treated with standard dose and duration of Acthar. We believe that our study will provide the basis for the development of future randomized RCI trials in IIM.
特发性炎性肌病（IIM）是一组以近端肌无力为特征的自身免疫性疾病。 H. P. Acthar gel [储存促皮质素注射剂（RCI）]是促肾上腺皮质激素的一种制剂，已被美国食品和药物管理局批准用于治疗 IIM；然而，相关文献有限。在这项研究中，我们报告了接受 RCI 治疗的肌炎患者的纵向随访结果。
本研究纳入了参加前瞻性、开放性 RCI 试验的难治性 IIM 患者。试验后随访期为 6 个月，每 2 个月评估一次，评估包括肌炎核心组测量指标，包括肌肉外整体、肌肉和患者整体疾病活动、HAQ 和手动肌肉测试。
试验结束后，有 2 名患者失访，其余 8 名患者入组了随访研究。1 名患者在试验后继续接受 RCI 治疗。在随访期间，8 名患者中有 4 名在 RCI 试验后平均 4.1 个月时出现病情加重。在病情加重的患者中，有 3 名需要增加泼尼松的剂量。1 名患者在 5.5 个月时重新开始接受 RCI 治疗，但在 3 个月后仅有轻微改善。4 名病情稳定的患者继续满足定义为改善的标准，通过 6 个月的随访。然而，在初始 RCI 试验后，所有患者的主要或次要疗效结局均未显示出进一步改善。
据我们所知，这是第一项报告接受标准剂量和持续时间 Acthar 治疗的患者随访结果的研究。我们相信，我们的研究将为未来的 IIM 随机 RCI 试验提供依据。
