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 prednisoneresistant 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.

Focal Segmental Glomerulosclerosis in Paediatric Population of South Punjab Pakistan: A Tertiary Care Hospital Experience

“…14 Complete remission among 33.3% children was noted by Brazilian researchers using a combination of steroid and cyclophosphamide. 19 It was also noted in the present study that 53.3% of the cases using this combination therapy reported relapse within two years of stopping this protocol and they were started on cyclosporine and achieved sustained remission. Previous local data also found 36% of the patients using cyclophosphamide and steroid to report relapse later and were shifted to cyclosporine who achieved sustained remission.…”

Therapies for steroid-resistant nephrotic syndrome

“…In the two trials comparing cyclophosphamide with prednisone [27, 28], 35% of 34 children achieved remission with prednisone alone. Similarly, a large observational study [29] found that 27% of children treated with oral cyclophosphamide achieved remission but that this rate did not differ from children given prednisone alone.…”

Long-term therapy with cyclosporine and alternate-day prednisone in patients with idiopathic Steroid Resistant Nephrotic Syndrome (SRNS)

“…Prolonged corticosteroid for 6-12 months may have a remission rate of 28% with increased rate of iatrogenic complications [5]. A partial or complete remission was reported with oral cyclospsphamide with a remission rate close to continued steroid therapy alone [31]. One trial compared oral cyclophosphamide with IV cyclophosphamide and reported no significant difference [32].…”