ObjectiveTo systematically review the efficacy and safety of mycophenolate mofetil (MMF) for Henoch-Schonlein purpura nephritis (HSPN). MethodsDatabases such as PubMed, EMbase, CENTRAL, VIP, CNKI, CBM and WanFang Data were electronically searched for comprehensively collecting the randomized controlled trials (RCTs) on the efficacy and safety of MMF for HSPN from inception to December, 2013. Two reviewers independently screened studies according to the inclusion and exclusion criteria, extracted data and evaluated the methodological quality of the included studies. Then meta-analysis was performed using RevMan 5.1 software. ResultsA total of 10 RCTs involving 426 patients (231 in the trial group and 195 in the control group) were included. The trial group was treated with MMF and corticosteroids, and the control group was treated with corticosteroids monotherapy or combined with cyclophosphamide (CTX), leflunomide (LEF), or azathioprine (AZA). The results of meta-analysis showed that, as for efficacy, no significant difference was found between the two groups after six-mouth treatment (OR=1.36, 95%CI 0.67 to 2.73, P=0.85), while after twelve-mouth treatment, MMF was superior to CTX with a significant difference (OR=6.58, 95%CI 2.45 to 17.33, P=0.002). In addition, the efficacy of MMF was still superior to the azathioprine group, but not better than either LEF or prednisone monotherapy. Lower incidence of side effects were found in the MMF group, compared with the CTX group (OR=0.25, 95%CI 0.13 to 0.45, P < 0.000 01) and the prednisone monotherapy group (OR=0.26, 95%CI, 0.09 to 0.79, P=0.02), while there was no significant difference between the MMF group and the LEF group in side effects. ConclusionBased on the current evidence, the efficacy of MMF for HSPN is better than CTX, and its side effects are less than those of CTX and prednisone.