title: Acthar gel in the treatment of nephrotic syndrome: a multicenter retrospective case series.
abstract: BACKGROUND
  Current first-line anti-proteinuric treatments for nephrotic syndrome (NS) do not produce an effective response in all patients and are not tolerated by some patients. Additional effective and tolerable treatment options in NS are strongly needed. This retrospective case series is the largest to date to examine Acthar gel (adrenocorticotropic hormone, ACTH) in patients with varied-etiology NS.
  
  
  METHODS
  This multicenter retrospective case series included adult patients with NS (N = 44) treated with Acthar gel at 6 clinical practices. NS etiologies included idiopathic focal segmental glomerulosclerosis (FSGS, 15), idiopathic membranous nephropathy (iMN, 11), IgA nephropathy (IgAN, 5), diabetic nephropathy (DN, 4), systemic lupus erythematosus class V membranous lupus nephritis (MLN, 2), minimal change disease (MCD, 2), membranoproliferative glomerulonephritis (MPGN, 1), fibrillary glomerulonephritis (FGN, 1), and unbiopsied NS (3). Proteinuria response was assessed as percent reduction from baseline and percent of patients meeting complete remission (final proteinuria <500 mg/d), partial remission (≥50 % reduction in proteinuria from baseline and final proteinuria 500-3500 mg/d), clinical response (≥30 % 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. Safety and tolerability were examined using adverse event (AE) frequency reported by patients or treating nephrologists and frequency of early discontinuation of treatment due to AEs.
  
  
  RESULTS
  68.2 % (30/44) of patients had received prior NS treatment with immunosuppressive or cytotoxic therapies. Thirty-seven patients completed Acthar gel treatment. Seven patients (15.9 %) had early termination due to AEs, including weight gain (2), hypertension (2), edema (1), fatigue (1), seizures (1) and for reasons not stated (2). Proteinuria reduction ≥30 % was shown in 81.1 % (30/37) of patients and 62.2 % (23/37) showed ≥50 % proteinuria reduction. Proteinuria responses were greatest in MCD (n = 2/2 complete remission), MLN (n = 2/2 partial remission), MPGN (n = 1/1 partial remission), FSGS (n = 12/15 [80.0 %] partial remission or clinical response), and iMN (n = 8/11 [72.7 %] complete remission, partial remission, or clinical response).
  
  
  CONCLUSIONS
  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.
authors.affiliation: Nephrology Associates of Central Florida, 3885 Oakwater Circle, Orlando, FL, 32806, USA.
authors.forename: Arvind
authors.initials: A
authors.lastname: Madan
authors.affiliation: Albany Medical College, Albany, NY, USA.
authors.forename: Snezana
authors.initials: S
authors.lastname: Mijovic-Das
authors.affiliation: HCA Inc. Physician Services, Salem, NH, USA.
authors.forename: Ana
authors.initials: A
authors.lastname: Stankovic
authors.affiliation: Lankenau Medical Center, Wynnewood, PA, USA.
authors.forename: Geoffrey
authors.initials: G
authors.lastname: Teehan
authors.affiliation: Nephrology Associates of Central Florida, 3885 Oakwater Circle, Orlando, FL, 32806, USA.
authors.forename: Amber S
authors.initials: AS
authors.lastname: Milward
authors.affiliation: Nephrology Practice, 3366 NW Expressway, Bldg D, Suite 280, Oklahoma City, OK, 73112, USA. akhastgir@khastgirmedical.com.
authors.forename: Anupa
authors.initials: A
authors.lastname: Khastgir
mesh_terms: D000324:Adrenocorticotropic Hormone / Q000627:therapeutic use*; D000328:Adult; D000368:Aged; D015331:Cohort Studies; D003928:Diabetic Nephropathies / Q000150:complications; D005260:Female; D005782:Gels; D005922:Glomerulonephritis, IGA / Q000150:complications; D015432:Glomerulonephritis, Membranoproliferative / Q000150:complications; D015433:Glomerulonephritis, Membranous / Q000150:complications; D005923:Glomerulosclerosis, Focal Segmental / Q000150:complications; D006728:Hormones / Q000627:therapeutic use*; D006801:Humans; D008181:Lupus Nephritis / Q000150:complications; D008297:Male; D008875:Middle Aged; D009402:Nephrosis, Lipoid / Q000150:complications; D009404:Nephrotic Syndrome / Q000188:drug therapy* / Q000209:etiology / Q000652:urine; D011507:Proteinuria / Q000188:drug therapy* / Q000209:etiology / Q000652:urine; D012189:Retrospective Studies; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D005782:Gels; D006728:Hormones; D000324:Adrenocorticotropic Hormone
keywords: ACTH; Acthar gel; Nephrotic syndrome; Proteinuria