Mycophenolate mofetil treatment of severe renal disease in pediatric onset systemic lupus erythematosus

Objective. To report the first clinical experience with mycophenolate mofetil (MMF, CellCept®) in children with lupus nephritis. Methods. Eleven children with various forms of lupus nephritis were treated with oral MMF at a mean dose of 22 mg/kg/day (range 17-42) for a mean of 9.8 months (range 3-17). All children received concomitant prednisone and 7/11 were taking concomitant hydroxychloroquine. Indications for MMF included treatment refractory nephritis despite high dose oral or IV prednisone, azathioprine, and/or cyclophosphamide: Treatment outcome was monitored through assessment of Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) score, renal function, and serologic markers such as complement and anti dsDNA antibodies. Results. While renal function normalized in 4/4 patients with membranous glomerulonephritis, little effect was observed in children with proliferative glomerulonephritis. Ten children experienced a marked reduction in SLEDAI score. Anti-dsDNA antibody and serum complement levels improved or remained stable in 80% of the children. Concomitant prednisone was decreased in 6/11 patients (55%) without deterioration of renal function. Adverse events, observed in 8 patients (73%), were not dose dependent, and included infections, leukopenia, natisea, pruritus, headache, and fatigue. Conclusion. MMF may represent a valuable alternative to traditional cytotoxic agents for children With class v lupus nephritis, but was less effective in attenuating disease progression in class IV glomerulonephritis. MMF had a steroid sparing effect and appeared to be effective in controlling serologic disease activity in pediatric onset SLE. Adverse events such as infections may limit its use and remain a concern.