A recent study (AN Zizzo et al. JPGN 2017; 65: 6-15) performed a systematic review and meta-analysis of pediatric autoimmune hepatitis (AIH) studies.
The most remarkable finding was that there were only 76 patients from 15 qualifying studies.
- Response to mycophenolate mofetil (MMF) with 34 patients was 36% (according to abstract) at 6 months (discrepancy in article –results state 38% response)
- Response to cyclosporine with 15 patients was 83% (discrepancy in article –results state 86% response)
- Response to tacrolimus with 4 patients was 50%
- Adverse effects were very common, particularly with cyclosporine (64% noted at least 1 adverse effect)
The article has an associated editorial (N Kerkar, pg 2-3). “The adverse event profile of cyclosporine with gingival hyperplasia, hypertrichosis, nephrotoxicity, and neurotoxicity made it challenging for long-term use in children.” Besides the small number of patients, “the studies that were included were largely “observational”‘ which limits their findings as well. The study authors recommend MMF as the preferred option for 2nd-line therapy.
My take: Fortunately, most patients with autoimmune hepatitis respond to first line therapy with azathioprine/steroids. It is unclear what is the optimal 2nd-line treatment for refractory patients.
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