Cyclophosphamide in the treatment of focal segmental glomerulosclerosis

• Published in: Brazilian journal of medical and biological research Vol. 37; no. 9; pp. 1365 - 1372
• Main Authors: Martinelli, R, Pereira, L J, Silva, O M M, Okumura, A S, Rocha, H
• Format: Journal Article
• Language: English
• Published: Brazil Associação Brasileira de Divulgação Científica 01.09.2004
• Subjects: Adolescent; Adult; BIOLOGY; Child; Cyclophosphamide; Cyclophosphamide - therapeutic use; Drug Resistance; Drug Therapy, Combination; Female; Focal segmental glomerulosclerosis; Follow-Up Studies; Glomerulosclerosis, Focal Segmental - complications; Glomerulosclerosis, Focal Segmental - drug therapy; Glucocorticoids - therapeutic use; Humans; Immunosuppressive Agents - therapeutic use; Male; MEDICINE, RESEARCH & EXPERIMENTAL; Nephrotic syndrome; Nephrotic Syndrome - complications; Nephrotic Syndrome - drug therapy; Prednisone; Prednisone - therapeutic use; Primary glomerular disease; Renal Insufficiency - etiology; Treatment Outcome; Cyclophosphamide; Prednisone; Nephrotic syndrome; Primary glomerular disease; Focal segmental glomerulosclerosis
• ISSN: 0100-879X; 1414-431X; 0100-879X; 1414-431X
• DOI: 10.1590/S0100-879X2004000900011

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.