Mycophenolate mofetil: Experience in two adolescents with systemic lupus erythematosus and Wegener's granulomatosis and severe renal involvement

Introduction: Therapy of autoimmune diseases in childhood may be difficult due to prolonged requirement of toxic immunosuppressive agents. To induce remission, intravenous cyclophosphamide (CYC) or methylprednisolone are common. Infections and gonadal toxicity are major risks of CYC. In recent years new therapeutic agents have been developed. Reports on the use of mycophenolate mofetil (MMF, Cellcept®) in children with autoimmune diseases are rare. We report on our experience with MMF in severe renal disease in patients with systemic lupus erythematosus (SLE) and Wegener's granulomatosis. Case reports: Patient 1 Wegener's granulomatosis is a necrotising, granulomatous vasculitis affecting respiratory tract and kidneys. At the age of 12 years Wegener's granulomatosis was diagnosed, the boy suffered from arthritis and necrotising glomerulonephritis. Serum contained circulating antineutrophil cycoplasmatic antibodies (c-ANCA). Despite six pulses of CYC, oral CYC and methylprednisolone pulses, remission failed. Cumulative toxic dosage of 250mg CYC/kg was exceeded. At the age of 13 1/2 years therapy with MMF (17mg/kg b.w.) was started. CYC therapy was stopped, oral prednisolone was reduced. c-ANCA decreased significantly and toxic effects of CYC therapy are reduced. Patient 2 Since the age of 13 1/2 years the girl suffers from SLE with mesangioproliferative glomerulonephritis (WHO type IIIa), proteinuria, and hypertension. Immunosuppressive therapy consisted of CYC bolus, oral CYC, azathioprine, prednisolone and methylprednisolone. Remission of nephritis failed and two years after onset of SLE immunsuppressive treatment was changed to MMF (20mg/kg b.w.) and oral prednisolone. Five months later disease activity allowed low prednisolone dose and MMF. Conclusions: Clinical trials in adults have demonstrated effiency of MMF in lupus nephritis. MMF seems to be a promising agent in young patients with autoimmune diseases who do not respond to high doses of cyclophosphamide or azathioprine or who have already received a cumulative toxic dosage of cyclophosphamide. Long-term efficiency and benefit have to be proven.