Mycophenolate mofetil(MMF), a potent and specific inhibitor of guanosine nu
cleotide synthesis, is a new immunosuppressive drug used to prevent rejecti
on in transplant patients. Extensive data on its utility and efficacy exist
s in adult patients but there is limited experience in pediatrics. Twenty-f
our children (14 male, 10 female,2-19 yr of age), eight of whom had receive
d living-related donor (LRD) transplants and 16 of whom had received cadave
ric donor (CD) transplants, have been treated with MMF in our institution s
ince September 1996, MMF was administered for a duration ranging from 13 we
eks to 38 months, at an average dose of 600 mg/m(2) rrange: 200-1,000 mg/do
se) every 12 h, for a total experience of 304 patient months. MMF capsules
were used in 16 patients and/or pediatric suspension in eight. Five patient
s were switched to MMF from azathioprine as a result of rejection episodes
or inability to taper prednisone, between 5 weeks and 3.5 yr post-transplan
t. All patients received prednisone, cyclosporin A (CsA), and induction the
rapy with anti-lymphocyte globulin (19 patients), anti-thymocyte globulin t
one patient) or daclizumab (four patients). In 13 patients started on MMF a
t the time of CD transplant, five (42%) had an acute rejection episode. In
seven who received a LRD transplant, one (14%,) had an acute rejection epis
ode. No patients who were converted to MMF were treated for acute rejection
following conversion to MMF. One LRD graft was lost at 19 days following i
njury to the donor artery at the time of retrieval. At the last follow-up,
the average creatinine level was 93 mu mol/L and average urea level was 8.6
mmol/L. One patient developed epigastric distress. Three patients develope
d diarrhea/abdominal pain requiring dose adjustment. Five episodes of leuko
penia and one episode of thrombocytopenia required dose adjustment. Two pat
ients developed symptomatic cytomegalovirus (CMV) infection, one while on a
cyclovir prophylaxis. No malignancy has been encountered to date. Hence, MM
F can be administered safely to children with good effect and with an accep
table side-effect profile.