title: Cyclophosphamide in the treatment of focal segmental glomerulosclerosis.
abstract: Prednisone is the initial treatment of primary focal segmental glomerulosclerosis. However, when immunosuppressive agents in combination with steroids are used in the treatment of prednisone-dependent and prednisone-resistant patients the remission rate is variable. We report a long-term trial using cyclophosphamide (2.0 to 3.0 mg/kg body weight for 12 weeks) in combination with prednisone (1.0 to 2.0 mg/kg body weight), as compared with prednisone alone for the treatment of prednisone-resistant and frequently relapsing nephrotic syndrome and focal segmental glomerulosclerosis. Fifty-four patients (34 males and 20 females) with a diagnosis of idiopathic nephrotic syndrome and focal segmental glomerulosclerosis, followed-up for an average of 86.1 +/- 82.4 months, were evaluated. Complete remission occurred in 20.4% and partial remission in 14.8% of the patients treated with steroids and in 26.7 and 20.0% of the patients treated with cyclophosphamide + prednisone, respectively. Of the 24 prednisone-resistant patients treated with steroids in combination with cyclophosphamide, 33.3% obtained a complete/partial response. At the time of final evaluation, 25% of the patients treated with prednisone and 10.0% of those treated with prednisone in combination with cyclophosphamide had reached end-stage renal disease. Persistent nephrotic syndrome and progressive renal insufficiency were more frequently observed among the patients treated with prednisone alone (50.0 vs 33.3% and 33.3 vs 16.7%, respectively). The treatments were well tolerated and no patient experienced adverse reactions requiring discontinuation of medications. Although open-label and non-randomized, the present trial showed that cyclophosphamide is a reasonable choice for the treatment of primary focal segmental glomerulosclerosis and prednisone-resistant nephrotic syndrome.
authors.affiliation: Serviço de Nefrologia, Departamento de Medicina, Faculdade de Medicina, Universidade Federal da Bahia, Salvador, BA, Brasil. rm@ufba.br
authors.forename: R
authors.initials: R
authors.lastname: Martinelli
authors.forename: L J
authors.initials: LJ
authors.lastname: Pereira
authors.forename: O M M
authors.initials: OM
authors.lastname: Silva
authors.forename: A S
authors.initials: AS
authors.lastname: Okumura
authors.forename: H
authors.initials: H
authors.lastname: Rocha
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D003520:Cyclophosphamide / Q000627:therapeutic use*; D004351:Drug Resistance; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D005923:Glomerulosclerosis, Focal Segmental / Q000150:complications / Q000188:drug therapy*; D005938:Glucocorticoids / Q000627:therapeutic use*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D009404:Nephrotic Syndrome / Q000150:complications / Q000188:drug therapy*; D011241:Prednisone / Q000627:therapeutic use*; D051437:Renal Insufficiency / Q000209:etiology; D016896:Treatment Outcome
chemical_list: D005938:Glucocorticoids; D007166:Immunosuppressive Agents; D003520:Cyclophosphamide; D011241:Prednisone