AB0382 A single-site, investigator initiated, open-label trial of the adrenocorticotropic hormone (acth) analogue h.p. acthar gel (repository corticotropin injection) among subjects with moderately to severely active systemic lupus erythematosus (sle)

Background Systemic lupus erythematosus (SLE) is a serious systemic disease which left untreated or inadequately treated may have a substantial effect on the morbidity and mortality of the individuals who are afflicted. Conventional treatments include immunosuppressive medications and corticosteroids. However, there is a need for alternative therapeutic options, particularly in SLE patients who may not be adequately controlled with or who are intolerant to traditional therapies. Acthar is known to have steroidogenic mechanisms of action through endogenous production of cortisol and is also reported to have immunomodulatory properties facilitated through the interactions with melanocortin receptors found in immune effector cells and other tissues. Self-administered via subcutaneous injection, Acthar may represent a potential treatment option for those patients. Objectives This study was designed to evaluate the efficacy of Acthar for reducing the severity of active lupus flares among patients with SLE who inadequately responded to other treatments. Methods Seven female patients (mean age=48.3 years, mean duration of disease=7.1 years) who had not, in the clinical judgment of the treating physician, responded adequately to previous treatments were included in the study. Patients received, through self-administration, 1 mL (80 U/mL) of Acthar gel for 7-15 days. Over a period of 28 days, all patients were assessed weekly. Outcome measurements included Medical Doctor Global Assessment (MDGA), Patient Global Assessment (PGA), Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K), erythrocyte sedimentation rate (ESR), and C-reactive protein (CRP). Student’s t-test was conducted to compare data obtained at Day 28 with those from baseline. Results There were statistically significant improvements in both SLEDAI-2K (P=.025) and MDGA (P=.020), as well as a trend toward improvement in PGA (P=.064), indicative of a reduction in the severity of lupus flares. Acthar was well-tolerated; however, bilateral edema was present in the legs/ankles of one patient. No treatment-related serious or unexpected adverse events were observed. Conclusions Lupus flares usually require treatment with prednisone in varying doses that depend upon the severity. The results obtained during this trial reveal that, when treated with Acthar, patients who had not responded adequately to other therapeutic agents can have significant improvements in scores that indicate reduction of