Background: Current first-line anti-proteinuric treatments for nephrotic syndrome (NS) do not produce an effective\nresponse in all patients and are not tolerated by some patients. Additional effective and tolerable treatment\noptions in NS are strongly needed. This retrospective case series is the largest to date to examine Acthar gel\n(adrenocorticotropic hormone, ACTH) in patients with varied-etiology NS.\nMethods: This multicenter retrospective case series included adult patients with NS (N = 44) treated with\nActhar gel at 6 clinical practices. NS etiologies included idiopathic focal segmental glomerulosclerosis (FSGS,\n15), idiopathic membranous nephropathy (iMN, 11), IgA nephropathy (IgAN, 5), diabetic nephropathy (DN, 4),\nsystemic lupus erythematosus class V membranous lupus nephritis (MLN, 2), minimal change disease (MCD, 2),\nmembranoproliferative glomerulonephritis (MPGN, 1), fibrillary glomerulonephritis (FGN, 1), and unbiopsied NS\n(3). Proteinuria response was assessed as percent reduction from baseline and percent of patients meeting\ncomplete remission (final proteinuria <500 mg/d), partial remission (ââ?°Â¥50 % reduction in proteinuria from\nbaseline and final proteinuria 500ââ?¬â??3500 mg/d), clinical response (ââ?°Â¥30 % reduction in proteinuria from baseline\nthat did not meet criteria for complete or partial remission), and no response (failed to meet remission or\nclinical response criteria) following Acthar gel therapy. Safety and tolerability were examined using adverse\nevent (AE) frequency reported by patients or treating nephrologists and frequency of early discontinuation\nof treatment due to AEs.\nResults: 68.2 % (30/44) of patients had received prior NS treatment with immunosuppressive or cytotoxic\ntherapies. Thirty-seven patients completed Acthar gel treatment. Seven patients (15.9 %) had early termination\ndue to AEs, including weight gain (2), hypertension (2), edema (1), fatigue (1), seizures (1) and for reasons not\nstated (2). Proteinuria reduction ââ?°Â¥30 % was shown in 81.1 % (30/37) of patients and 62.2 % (23/37) showed\nââ?°Â¥50 % proteinuria reduction. Proteinuria responses were greatest in MCD (n = 2/2 complete remission), MLN\n(n = 2/2 partial remission), MPGN (n = 1/1 partial remission), FSGS (n = 12/15 [80.0 %] partial remission or\nclinical response), and iMN (n = 8/11 [72.7 %] complete remission, partial remission, or clinical response).\nConclusions: Acthar gel may meet an important treatment need in patients with treatment-resistant NS in\nresponse to first-line therapies, patients unable to tolerate first-line therapies, and in patients with advanced\ndisease.
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