The clinical application of mycophenolate mofetil (MMF) has significantly widened beyond the prophylaxis of acute and chronic rejections in solid organ transplantation. MMF has been recognized as an excellent treatment option in many immunologic glomerulopathies. For children with frequently relapsing nephrotic syndrome (FRNS) or steroid-dependent nephrotic syndrome (SDNS) experiencing steroid toxicity, MMF has been recommended as a steroid-sparing drug. Uncontrolled studies in patients with FRNS and SDSN have shown that many patients can achieve sustained remission of proteinuria with MMF monotherapy. Three randomized controlled trials have similarly demonstrated that MMF is beneficial in these patients, but less effective than the calcineurin inhibitors cyclosporin A or tacrolimus. Some, but not all, patients with steroid-resistant nephrotic syndrome (SRNS) may also respond to MMF, usually given in combination with other drugs, with partial or complete remission. There are important limitations to the interpretation and comparability of these studies including study design, sample size, patient selection, clinical endpoints, carry-over effects, and duration of follow-up. In all studies, MMF had relatively few side effects, no nephrotoxicity, or no systemic toxicity. MMF is teratogenic, and contraceptive advice is required in females. There is a poor correlation between MMF dose and mycophenolic acid (MPA) exposure and significant inter- and intra-patient variability in drug pharmacokinetics. A higher estimated MPA-AUC0-12 target range than recommended for pediatric renal transplant recipients is essential to prevent relapses. Therefore, therapy should be guided by drug monitoring to avoid relapses. Further studies are needed to test the efficacy of MMF in inducing remission and, as part of a combination therapy, achieving sustained remission in patients with SRNS.
Keywords: Corticosteroids; Cyclosporin A; Idiopathic nephrotic syndrome; Mycophenolate mofetil; Mycophenolic acid; Tacrolimus.