title: Mycophenolate mofetil in steroid/cyclosporine-dependent/resistant nephrotic syndrome.
abstract: Attempts to minimize the effects of prolonged steroid use in steroid-dependent nephrotic syndrome (SDNS) and the need to overcome steroid resistance (SRNS) justifies immunosuppressant therapy. We report our experience in a cohort of patients with SD/SRNS during the administration of mycophenolate mofetil (MMF) in a prospective protocol initiated in January 2001. Twenty-six children with idiopathic nephrotic syndrome were included (21 steroid dependent and 5 steroid resistant), whose response did not change after sequential treatment with cyclophosphamide (CPM) and cyclosporine (CsA). Histopathologic patterns were: 11 minimal change disease (MCD), 1 diffuse mesangial proliferation (DMP), 13 focal segmental glomerulosclerosis (FSGS) and membranous 1 glomerulonephritis (MGN). The median age of onset of NS was 2.8 years (range 1.2-12.5), and treatment with MMF was performed at a median age of 11.4 years (range 5-17) with an initial dose of 600 mg/m(2)/12 h, adjusted to maintain levels of mycophenolic acid (MPA) at 2.5-5 mcg/ml. The planned duration of study to assess treatment efficacy was 6 months. The mean MMF dose required was 624 (SD=136) mg/m(2)/12 h (range 415-970), which maintained mean C(0)-MPA levels of 2.9 (SD=1.17) mcg/ml (range 1.2-5.9 mcg/ml). In the five patients with SRNS, only one achieved complete remission. In the patients with SDNS, steroid sparing was achieved in 15 and 9 remained in remission on MMF monotherapy. Withdrawal of MMF resulted in immediate relapse in 47%. In our study, MMF was a useful immunosuppressant due to its fewer undesirable effects and similar efficacy to other drugs used. It appears effective for the maintenance of remission in SDNS patients, with a response similar to that of CsA.
authors.affiliation: Department of Pediatric Nephrology, La Fe Hospital, Avda. Campanar 21, 46009 Valencia, Spain. mendizabal_san@gva.es
authors.forename: S
authors.initials: S
authors.lastname: Mendizábal
authors.forename: I
authors.initials: I
authors.lastname: Zamora
authors.forename: O
authors.initials: O
authors.lastname: Berbel
authors.forename: M J
authors.initials: MJ
authors.lastname: Sanahuja
authors.forename: J
authors.initials: J
authors.lastname: Fuentes
authors.forename: J
authors.initials: J
authors.lastname: Simon
mesh_terms: D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D003520:Cyclophosphamide / Q000627:therapeutic use; D016572:Cyclosporine / Q000627:therapeutic use*; D004305:Dose-Response Relationship, Drug; D004351:Drug Resistance; D005938:Glucocorticoids / Q000008:administration & dosage / Q000627:therapeutic use; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D009173:Mycophenolic Acid / Q000031:analogs & derivatives* / Q000627:therapeutic use; D009404:Nephrotic Syndrome / Q000188:drug therapy*; D011241:Prednisone / Q000008:administration & dosage / Q000627:therapeutic use; D011446:Prospective Studies; D013256:Steroids / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D005938:Glucocorticoids; D007166:Immunosuppressive Agents; D013256:Steroids; D016572:Cyclosporine; D003520:Cyclophosphamide; D009173:Mycophenolic Acid; D011241:Prednisone