Efficacy of mycophenolate mofetil in patients with diffuse proliferative lupus nephritis

Background: The combination of cyclophosphamide and prednisolone is effecti ve for the treatment of severe lupus nephritis but has serious adverse effe cts. Whether mycophenolate mofetil can be substituted for cyclophosphamide is not known. Methods: In 42 patients with diffuse proliferative lupus nephritis we compa red the efficacy and side effects of a regimen of prednisolone and mycophen olate mofetil given for 12 months with those of a regimen of prednisolone a nd cyclophosphamide given for 6 months, followed by prednisolone and azathi oprine for 6 months. Complete remission was defined as a value for urinary protein excretion that was less than 0.3 g per 24 hours, with normal urinar y sediment, a normal serum albumin concentration, and values for serum crea tinine and creatinine clearance that were no more than 15 percent above the base-line values. Partial remission was defined as a value for urinary pro tein excretion that was between 0.3 and 2.9 g per 24 hours, with a serum al bumin concentration of at least 3.0 g per deciliter. Results: Eighty-one percent of the 21 patients treated with mycophenolate m ofetil and prednisolone (group 1) had a complete remission, and 14 percent had a partial remission, as compared with 76 percent and 14 percent, respec tively, of the 21 patients treated with cyclophosphamide and prednisolone f ollowed by azathioprine and prednisolone (group 2). The improvements in the degree of proteinuria and the serum albumin and creatinine concentrations were similar in the two groups. One patient in each group discontinued trea tment because of side effects. Infections were noted in 19 percent of the p atients in group 1 and in 33 percent of those in group 2 (P=0.29). Other ad verse effects occurred only in group 2; they included amenorrhea (in 23 per cent of the patients), hair loss (19 percent), leukopenia (10 percent), and death (10 percent). The rates of relapse were 15 percent and 11 percent, r espectively. Conclusions: For the treatment of diffuse proliferative lupus nephritis, th e combination of mycophenolate mofetil and prednisolone is as effective as a regimen of cyclophosphamide and prednisolone followed by azathioprine and prednisolone. (N Engl J Med 2000;343:1156-62.) (C) 2000 Massachusetts Medi cal Society.