Response to cyclophosphamide in steroid-resistant focal segmental glomerulosclerosis: a reappraisal.
The response to and clinical outcome of cyclophosphamide therapy were retrospectively assessed in 29 steroid-resistant patients with idiopathic nephrotic syndrome and focal segmental glomerulosclerosis (FSGS) to determine whether a partial response to this drug was associated with long-term clinical benefits. Twenty of the patients were nephrotic when cyclophosphamide was started and 9 were not. Three of the nephrotic patients had a complete response (i.e., sustained remission of disease) to cyclophosphamide. Nine nephrotic patients had partial responses. Of these, 8 have residual proteinuria and one has progressed to end-stage renal disease (ESRD). In contrast, of the 8 nephrotic patients who were resistant to cyclophosphamide, only one has residual proteinuria, while 7 have chronic renal failure (CRF) or ESRD. The incidence of CRF or ESRD in patients with a partial response to cyclophosphamide (1 of 9) was significantly lower (p = 0.004) than that in patients who were resistant to cyclophosphamide (7 of 8). The benefit of cyclophosphamide in patients who were not overtly nephrotic was less certain. This study indicates that a partial response to cyclophosphamide leads to improvement in the clinical outcome of many steroid-resistant nephrotic patients with FSGS.
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use*; D005921:Glomerulonephritis / Q000188:drug therapy*; D005923:Glomerulosclerosis, Focal Segmental / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D007223:Infant; D007676:Kidney Failure, Chronic / Q000517:prevention & control; D009404:Nephrotic Syndrome / Q000188:drug therapy* / Q000209:etiology; D011241:Prednisone / Q000627:therapeutic use; D011379:Prognosis; D011507:Proteinuria / Q000209:etiology; D012189:Retrospective Studies
D003520:Cyclophosphamide; D011241:Prednisone