title: Use of mycophenolate mofetil in steroid-dependent and -resistant nephrotic syndrome.
abstract: Cyclosporin (Cs-A) is an effective treatment for difficult cases of nephrotic syndrome (NS), but its use can be complicated by renal toxicity and a high incidence of relapses after withdrawal. We reviewed the charts of 10 Cs-A-dependent patients and 4 patients with steroid-dependent nephrotic syndrome (SDNS) not previously treated with Cs-A therapy. All patients had persistent NS, even after prior treatment with oral cyclophosphamide. Of 10 patients treated with Cs-A, 4 had surveillance renal biopsies consistent with Cs-A toxicity, and 8 of 10 had interstitial fibrosis prior to mycophenolate mofetil (MMF). Patients were treated with MMF, at 1,200 mg/m(2) per day, in an attempt to allow weaning of Cs-A and/or steroid therapy, and reduce the frequency of relapses. Overall, a significant decrease in frequency of relapses was noted after initiation of MMF therapy. In addition, 5 patients were weaned off Cs-A by 1-2 years of follow-up. One patient was weaned off Cs-A and MMF, and remained in complete remission. However, the subgroup of patients with frequently relapsing SDNS not treated with Cs-A appeared to have a reduction in the number of relapses while on MMF that did not reach statistical significance. Two patients with intractable steroid-resistant NS continued to relapse repeatedly on MMF and Cs-A therapy. We conclude that in this small, single-center, uncontrolled experience, MMF therapy in patients with Cs-A-dependent NS appears to be effective in reducing Cs-A exposure. In addition, MMF appears to significantly decrease the frequency of relapses in this patient population. Further controlled studies are warranted to better define the potential efficacy and side effects of long-term MMF therapy in this setting.
authors.affiliation: University of Michigan Health Sciences Center, Mott Children's Hospital, 1505 Simpson Road East, F6865/Box 0297, Ann Arbor, MI 48109-0297, USA. gpalombo@umich.edu
authors.forename: Gina-Marie
authors.initials: GM
authors.lastname: Barletta
authors.forename: William E
authors.initials: WE
authors.lastname: Smoyer
authors.forename: Timothy E
authors.initials: TE
authors.lastname: Bunchman
authors.forename: Joseph T
authors.initials: JT
authors.lastname: Flynn
authors.forename: David B
authors.initials: DB
authors.lastname: Kershaw
mesh_terms: D000894:Anti-Inflammatory Agents, Non-Steroidal / Q000008:administration & dosage* / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage; D004351:Drug Resistance; D004359:Drug Therapy, Combination; D005938:Glucocorticoids / Q000008:administration & dosage*; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage; D007223:Infant; D009173:Mycophenolic Acid / Q000008:administration & dosage* / Q000009:adverse effects / Q000031:analogs & derivatives*; D009404:Nephrotic Syndrome / Q000188:drug therapy*; D011241:Prednisone / Q000008:administration & dosage*; D012189:Retrospective Studies; D055502:Secondary Prevention; D016896:Treatment Outcome
chemical_list: D000894:Anti-Inflammatory Agents, Non-Steroidal; D005938:Glucocorticoids; D007166:Immunosuppressive Agents; D003520:Cyclophosphamide; D009173:Mycophenolic Acid; D011241:Prednisone