Background Mycophenolate mofetil (MMF) has been increasingly used after liv
er transplantation (LT) in adults, We report our preliminary experience wit
h MMF as rescue therapy after pediatric LT,
Methods. A total of 19 children received MMF for 21 indications. Median age
at LT was 30 months (range 7-149), The median initial oral dose of MMF was
23 mg/kg/day (range 12-43) orally. Median follow-up after initiation of MM
F therapy was 642 days (range 229-1606),
Results, 1) Efficacy: MMF was indicated for rejection or insufficient immun
osuppression in 16 cases, with normalization of both liver function tests a
nd liver histology in 10 (62%). MMF was successfully used in one patient wi
th post-LT immune hepatitis and one patient with corticodependence. In thre
e patients with renal function impairment, MMF allowed reduction of cyclosp
orine A or tacrolimus blood levels, without subsequent rejection. 2) Tolera
nce: Six patients (32%) experienced eight side effects, mainly gastrointest
inal and hematological, which resolved after cessation of MMF in five cases
and dose reduction in three. One case of posttransplant lymphoproliferativ
e disease (PTLD) occurred under MMF therapy (5.2%). Four patients had EBV p
rimary infection, while under MMF therapy, without subsequent PTLD. Three p
atients had CMV primary infection, and five CMV reactivation, under MMF the
rapy. Seven remained asymptomatic, and one presented with CMV enteritis.
Conclusions. These preliminary results suggest that MMF is an effective and
safe immunosuppressant in pediatric LT recipients. Its use is hampered by
frequent gastrointestinal and hematological side-effects. MMF does not seem
to increase the risk of PTLD nor CMV disease.