Safety and efficacy data are presented on the use of mycophenolate mofetil (MMF) in 26 children and adolescents with lupus. Data include therapy before and 12 months after starting MMF. 18 of 26 patients had biopsy-proved lupus nephritis. Group 1 were commenced on MMF induction and/or maintenance therapy (n=14), group 2 converted from azathioprine because of inadequate disease control (n=12). 73% of all (10 (71%) group 1 and 10 (83%) group 2) patients experienced a significant improvement in British Isles Lupus Assessment Group score (from median 9.0 to 3.0). Children with hypocomplementaemia increased their C3 significantly in both groups (0.53–1.15 for group 1 and 0.63–1.2 g/l for group 2, p=0.001), and C4 level only in group 1 (0.08–0.17, p=0.01). Renal function and albuminuria improved in those with active nephritis (p≤0.01). Significant improvements were seen in both groups in haemoglobin, erythrocyte sedimentation rate and lymphocyte counts. Prednisolone dose was weaned in both groups, p<0.05. Side-effects were seen in four patients, but none was judged to be severe enough to discontinue treatment. MMF treatment in this cohort of children with lupus seemed to be safe, well tolerated and effective.
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