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Treatment of nephrotic syndrome with adrenocorticotropic hormone (ACTH) gel.
MedLine Citation:
PMID:  21448451     Owner:  NLM     Status:  In-Data-Review    
Abstract/OtherAbstract:
PURPOSE: A synthetic adrenocorticotropin (ACTH) analog has shown efficacy in Europe as primary and secondary therapy for nephrotic syndrome, but there is no published experience using the natural, highly purified ACTH gel formulation, available in the United States, for nephrotic syndrome. We therefore investigated the use of ACTH gel for nephrotic syndrome in the United States.
PATIENTS AND METHODS: Twenty-one patients with nephrotic syndrome treated with ACTH gel outside of research settings in the United States, with initiation of therapy by December 31, 2009, allowing a minimum 6 months follow-up. We defined complete remission as stable renal function with proteinuria falling to <500 mg/day, and partial remission as stable renal function with >50% reduction in proteinuria from 500 to 3500 mg/day.
RESULTS: Twenty-one patients with nephrotic syndrome were treated: 11 with idiopathic membranous nephropathy (iMN), 4 with membranoproliferative glomerulonephritis (MPGN), 1 with focal segmental glomerulosclerosis (FSGS), 1 with minimal change disease (MCD), 1 with immunoglobulin A (IgA) nephropathy, 1 with class V systemic lupus erythematosus (SLE) glomerulonephritis, 1 with monoclonal diffuse proliferative glomerulonephritis, and 1 with unbiopsied nephrotic syndrome. ACTH was used as primary therapy for 3 patients; the remaining patients had previously failed a mean 2.3 immunosuppressive regimens. Eleven patients achieved a complete or partial remission, with 4 (19%) in complete remission. Of the 11 patients who achieved remission, 9 had iMN, 1 had FSGS, and 1 had IgA nephropathy. Of the 11 patients with iMN, 3 (27%) achieved complete remission and 6 (55%) achieved partial remission despite having previously failed a mean 2.4 therapies. Five patients reported steroid-like adverse effects, but there were no severe infections. The limitations were retrospective data analysis with short-term follow-up.
CONCLUSION: ACTH gel may be a viable treatment option for resistant nephrotic syndrome due to membranous nephropathy. Short-term data suggest that remission rates may approach 80%.
Authors:
Andrew S Bomback; James A Tumlin; Joel Baranski; James E Bourdeau; Anatole Besarab; Alice S Appel; Jai Radhakrishnan; Gerald B Appel
Publication Detail:
Type:  Journal Article     Date:  2011-03-14
Journal Detail:
Title:  Drug design, development and therapy     Volume:  5     ISSN:  1177-8881     ISO Abbreviation:  Drug Des Devel Ther     Publication Date:  2011  
Date Detail:
Created Date:  2011-03-30     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  101475745     Medline TA:  Drug Des Devel Ther     Country:  New Zealand    
Other Details:
Languages:  eng     Pagination:  147-53     Citation Subset:  IM    
Affiliation:
Department of Medicine, Division of Nephrology, Columbia University College of Physicians and Surgeons, New York, NY, USA;
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