Follow-up results of myositis patients treated with H. P. Acthar gel

Abstract

Objectives 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. Methods 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. Results 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. Conclusion 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.

<p>Treatment with Repository Corticotropin Injection in Patients with Rheumatoid Arthritis, Systemic Lupus Erythematosus, and Dermatomyositis/Polymyositis</p>

“…In addition, patients who discontinued RCI after 6 months of clinical efficacy experienced a slow increase in disease activity and disability index and a decrease in muscle strength; their first flare occurred 4 months after discontinuation of RCI. 25 Interim results from an open-label study of RCI in 9 adults with refractory cutaneous DM reported that at 3 months, 7 patients had improved Cutaneous Dermatomyositis Disease Area and Severity Index activity scores and 8 patients had improved Physician Global Assessment activity scores. 26 The current study is limited by its retrospective design and the small sample size yielded by our recruitment efforts.…”

Dry eye disease: A review of anti-inflammatory therapies

“…Furthermore, corticotropin gel treatment was also less likely to contribute to severe steroid-related adverse events in comparison to glucocorticoid treatment, while still providing anti-inflammatory benefits. [ 46 ]…”

Repository Corticotropin Injection for the Treatment of Pulmonary Sarcoidosis: A Narrative Review

“…RCI is distinct from corticosteroids, inhibitors of cell activation, proliferation, and migration (e.g., methotrexate, azathioprine, leflunomide, mycophenolate), TNF-a antagonists (e.g., adalimumab, infliximab), and anti-cluster of differentiation (CD)20 antibodies (i.e., rituximab) [56,58,59]. In other inflammatory conditions, such as systemic lupus erythematosus, idiopathic inflammatory myopathies, and rheumatoid arthritis, RCI has been found to be safe and effective for treatment of patients who were nonresponsive to corticosteroids [60][61][62]. The effectiveness of RCI in patients who are refractory to corticosteroids supports RCI's unique mechanism of action that is distinct from steroids.…”