Mycophenolate mofetil (MMF) has been used to rescue liver allografts with steroid-resistant rejection (SRR). However, the long-term outcome of these patients is not known. This study evaluates the long-term outcome of MMF rescue therapy for SRR in pediatric liver allograft recipients. Twenty-six children (who received 28 liver transplants), including 16 girls, were given MMF for SRR. The median age at transplant was 1.7 (range 0.4-13.6) years. Primary immunosuppression was cyclosporine-based in 22 and tacrolimus-based in 6. All patients except one had been converted to tacrolimus prior to MMF, having already received a median of 2 (1-5) courses of high-dose intravenous methylprednisolone. The median time to MMF rescue therapy was 1.8 (0.4-35.8) months. Twenty-one of 28 episodes of SRR responded to MMF therapy. The median follow-up was 8.8 (7.7-11.5) years. In responders, there was 1 death from posttransplant lymphoproliferative disease, and no grafts were lost to chronic rejection. In the 7 nonresponders, 3 grafts were lost to chronic rejection with 2 patient deaths. Surviving children are clinically well with good liver function, and 17 remain on MMF. Three children have glomerular filtration < 80 mL/minute/1.73 m(2). Side effects of MMF were seen in 12 patients; diarrhea (n = 5) and leukopenia (n = 5) being the most common. MMF was found to be effective in treating SRR in pediatric allograft recipients, with good long-term graft function and an acceptable side-effect profile.