Mycophenolate mofetil treatment of severe renal disease in pediatric onsetsystemic lupus erythematosus

Objective. To report the first clinical experience with mycophenolate mofet il (MMF, CellCept(R)) 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, az athioprine, and/or cyclophosphamide. Treatment outcome was monitored throug h assessment of Systemic Lupus Erythematosus Disease Activity Index (SLEDAI ) score, renal function, and serologic markets such as complement and anti dsDNA antibodies. Results. While renal function normalized in 4/4 patients with membranous gl omerulonephritis, 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 6/1 1 patients (55%) without deterioration of renal function. Adverse events, o bserved in 8 patients (73%), were not dose dependent, and included infectio ns, leukopenia, nausea, pruritus, headache, and fatigue. Conclusion. MMF may represent a valuable alternative to traditional cytotox ic 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 serol ogic disease activity in pediatric onset SLE. Adverse events such as infect ions may limit its use and remain a concern.