Background Acthar Gel has been approved for use in multiple rheumatic/inflammatory conditions since the early 1950s, yet the current literature remains sparse on its real-world clinical efficacy and safety. In addition to the well-established steroid-dependent effects of Acthar Gel, research suggests it has direct steroid-independent immunomodulatory effects in a variety of tissues due to its ability to bind to multiple melanocortin receptors. Clinical reports on the real-world efficacy and safety of Acthar Gel are needed.
Objectives This is an assessment of the efficacy and tolerability of Acthar Gel among patients with systemic lupus erythematosus (SLE).
Methods This is a retrospective analysis of 4 African American female patients with SLE, all of whom had continued disease activity despite receiving standard therapy (see Table for previous treatments). They were all on chronic maintenance cortisone at an equivalent dosage of 5 mg of prednisone per day. Each patient received 80 U of Acthar Gel subcutaneously twice weekly as adjunctive treatment. Patients 1, 2, 3, and 4 were treated for 9, 1.5, 6, and 3.5 months, respectively. Outcome measures included protein/creatinine ratio, double-stranded DNA (dsDNA), lymphocyte count, white blood cell count, platelet count, and hemoglobin level.
Results The SLE patients had a mean disease duration of 7.85 years. Results are presented below (Table). There was a substantial decline in protein/creatinine ratio among 3 patients, especially patient 3, who decreased from 7628 mg/g to 1311 mg/g in approximately 11 months. The first patient had extremely elevated dsDNA, which can occur among patients with SLE, but it declined substantially after treatment initiation with Acthar Gel. Patient 4 had diffuse skin hyperpigmentation. There were no other treatment-related adverse events.
Conclusions Acthar Gel was well tolerated and reduced proteinuria in this small subset of patients. These results support those from previous SLE studies. Acthar Gel provided clinical benefits for 3 of 4 patients despite the fact that they had not responded adequately to prior treatments. The substantial improvements in protein/creatinine ratio among those 3 patients all began within the first month of initiation of 80 U Acthar Gel twice weekly and were sustained throughout the duration of treatment. Although there were only 4 subjects included in this analysis, of the 3 patients that showed improved disease activity, 2 of these patients (66%) showed improved lymphocyte counts, not unexpected in patients with autoimmune disease. Melanocortin receptors are also found on lymphocytes, and therefore it is possible that Acthar Gel provided beneficial effects on certain T cell populations among these patients with active disease who had not responded adequately to previous treatments. Additional clinical research is necessary in order to establish the optimal dosage, optimal duration, and efficacy, safety, and tolerability of therapy with Acthar Gel.
Acknowledgements Funding to support the preparation of this abstract for submission was provided by Mallinckrodt Pharmaceuticals.
Disclosure of Interest S. Pegram Speakers bureau: Compensation originally provided by Questcor Pharmaceuticals, Inc. and continued by Mallinckrodt Pharmaceuticals while serving as a speaker.
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