Mycophenolate mofetil as rescue treatment for autoimmune liver disease in children: a 5-year follow-up.

BACKGROUND/AIM: The aim of this study was to evaluate the outcome of mycophenolate mofetil (MMF) therapy in children with autoimmune liver disease who are resistant to or intolerant of standard immunosuppression.

METHODS: (a) failure to achieve/maintain remission with prednisolone/azathioprine therapy or (b) significant treatment side-effects. Initial MMF dose was 20mg/kg/day, gradually increased to a maximum of 40 mg/kg/day. Azathioprine was stopped when MMF was commenced.

RESULTS: Twenty-six children (17 female) were recruited. Median (range) age at diagnosis was 9.9 (1.2-14.4) years. Sixteen had Type 1 autoimmune hepatitis (AIH), two Type 2 AIH, and eight had autoimmune sclerosing cholangitis (ASC). Median (range) time from diagnosis to addition of MMF was 14.9 (0.2-108.6) months. Eighteen children responded to MMF, aspartate aminotransferase (AST) normalising in 14. At median (range) follow-up of 61.5 (19.5-96.3) months, AST remained normal in 12. All 18 children were well, but two had clinical signs of portal hypertension. Eight (6 ASC) did not respond: AST remained elevated in seven, one was listed for transplant for decompensated liver disease and one had clinical signs of portal hypertension. MMF was well tolerated. Leukopenia (n=7) was the most common side-effect.

CONCLUSIONS: MMF is an effective rescue therapy for children with AIH, but not for those with ASC.