Acthar gel (adrenocorticotropic hormone) may be an option for treating nephrotic syndrome (NS), new findings suggest.

Arvind Madan, MD, of Nephrology Associates of Central Florida in Orlando, and colleagues conducted a multicenter retrospective case series that included 44 adult NS patients treated with Acthar gel at 6 clinical practices. Of these, 30 (68.2%) had received prior treatment with immunosuppressive or cytotoxic therapies. NS etiologies included idiopathic focal segmental glomerulosclerosis (FSGS, 15 patients), idiopathic membranous nephropathy (iMN, 11), IgA nephropathy (IgAN, 5), diabetic nephropathy (DN, 4), systemic lupus erythrematosus class V membranous lupus nephritis (MLN, 2), minimal change disease (MCD, 2), membranoproliferative glomerulonephritis (MPGN, 1), fibrillary glomerulonephritis (FGN, 1), and unbiopsied NS (3).

The investigators assessed proteinuria response as percent reduction from baseline and percent of patients who achieve complete remission (final proteinuria less than 500 mg/day), partial remission (50% or greater reduction in proteinuria from baseline and final proteinuria 500–3,500 mg/day), clinical response (30% or greater reduction in proteinuria from baseline that did not meet criteria for complete or partial remission), and no response (failed to meet remission or clinical response criteria) following Acthar gel therapy.

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A total of 37 patients completed Acthar gel treatment. Seven patients (15.9%) ended the treatment early because of adverse effects. Thirty patients (81.1%) experienced a 30% or greater reduction in proteinuria and 23 patients (62.2%) experienced a 50% or greater reduction in proteinuria, the researchers reported in BMC Nephrology (2016 17:37). Complete remission occurred in both MCD patients and partial remission occurred in both MLN patients and the patients with MPGN. Partial remission or clinical response was observed in 12 (80%) of the 15 FSGS patients. Complete or partial remission or clinical response occurred in 8 (72.7%) of the 11 iMN patients.

“Acthar gel may meet an important treatment need in patients with treatment-resistant NS in response to first-line therapies, patients unable to tolerate first-line therapies, and in patients with advanced disease,” the authors concluded.