Mycophenolate Mofetil in Children with Frequently Relapsing Nephrotic Syndrome

Abstract

Children with frequently relapsing nephrotic syndrome (FRNS) often develop adverse effects from prednisone. Attempts to induce long-term remission in such patients have had varying levels of success. In this multicenter, prospective, open-label study, 14 centers enrolled 33 patients with FRNS, all of whom were in remission at the time of entry. Six of the patients were steroid dependent. The patients received mycophenolate mofetil (MMF) 600 mg/m 2 twice daily (maximum 1 g twice daily) for 6 mo. A tapering dosage of alternate-day prednisone was given to each patient during the first 16 wk of MMF therapy. Patients were monitored for relapses of NS during and after MMF therapy. Treatment failure was defined as a relapse of NS. The patients had the following features at study entry: Age 6.8 ؎ 2.7 yr (range 2 to 15 yr); 56% male, 44% female; and 50% white; 25% black, and 25% other. Estimated GFR at entry was 138 ؎ 42 ml/min per 1.73 m 2 . Twenty-four (75%) of 32 patients stayed in remission throughout the 6 mo of MMF therapy. The relapse rate in these patients improved from one episode every 2 mo before MMF to one every 14.7 mo after MMF. Eight patients stayed in remission during the post-MMF period, for periods of 18 to 30 mo, whereas 16 relapsed after stopping MMF. Eight (25%) of 32 patients relapsed while taking MMF. It is concluded that MMF is effective for maintaining remission in patients who have FRNS and receive treatment for at least 6 mo and is associated with a low incidence of adverse events.

Mycophenolate mofetil for steroid-dependent nephrotic syndrome: a phase II Bayesian trial

“…Subsequently, numerous reports showed that in patients treated with cyclosporine, changing to MMF allowed a similar control of relapse rate with the benefit of improved glomerular filtration rate and hypertrichosis reversal [6][7][8][9][10]. To date, only five prospective studies of MMF in pediatric relapsing NS are available [14][15][16][17][18]. However, in these studies, patients had different past histories, as some had SDNS but others had frequently relapsing NS [16], a situation not as severe as SDNS.…”

“…Prospective studies on MMF efficacy in pediatric SDNS are limited [14][15][16][17][18]. Demonstrating a statistically significant effect of a new therapy is difficult in rare diseases, as long duration of the inclusion period may be necessary.…”

Mycophenolate Mofetil versus Cyclosporin A in Children with Frequently Relapsing Nephrotic Syndrome

“…Several small studies with limited statistical power have shown that MMF has steroid-sparing effects and reduces relapse rates in patients with FR-SSNS, albeit with varying efficacy. [11][12][13][14][15][16][17][18] We studied efficacy and safety of MMF in patients with FR-SSNS in comparison with CsA in a prospective randomized multicenter open-label crossover trial.…”

Mycophenolate-Associated Alopecia in an Adolescent Girl Mycophenolate and Alopecia

“…Mycophenolate mofetil (MMF) is an immunosuppressive agent more recently added to the therapeutic armamentarium against steroid resistant (SRNS), frequently relapsing (FRNS) and steroid or cyclosporine dependent nephrotic syndrome (SDNS, CsADNS) (1)(2)(3)(4)(5)(6)(7). It has emerged as a new agent without nephrotoxicity and gonadal toxicity (3).…”

“…It has emerged as a new agent without nephrotoxicity and gonadal toxicity (3). The most common side effects are gastrointestinal (GI) complaints (gastritis, nausea, vomiting, dyspepsia, constipation, diarrhea, abdominal distention, abdominal pain), anemia, leukopenia, and a predisposition to infection and malignancies (2,5).…”