Refractory autoimmune hepatitis in children: Considerations for assessment and management

Abstract

Refractory autoimmune hepatitis (AIH) is defined as intolerance of or unresponsiveness to standard immunosuppression and occurs in 10%-20% of children with AIH. Lack of response or slower than expected response to induction of remission with steroids, despite good compliance, might be the first clue to refractory AIH. Refractoriness to treatment is associated with an 11.7 times higher risk for liver transplantation or death due to liver disease. The first and foremost consideration for the management is to assess compliance with treatment. It is then important to re-evaluate the diagnosis, assess alternative aetiologies which can mimic the clinical, serological, and histological features of AIH, and address the presence of extra-hepatic co-morbidities. It is important to consider the specific clinical situations, previous therapy, and prior adverse effects before deciding on the most appropriate treatment regimen in refractory AIH. Consideration also should be given to compliance with previous therapy, need for drug level monitoring, growth potential, available formulations, route of administration of medication, and children’s and families’ preferences before deciding on the therapy. Treatment should be decided and monitored only in specialized hepatology centers.

Keywords: Autoimmune hepatitis; Refractory; Remission; Treatment; Non-compliance

Core Tip: Treatment of autoimmune hepatitis seeks to induce and maintain remission, prevent progression, and potentially reverse fibrosis. In the 10%-20% of children unresponsive to steroids and azathioprine, adherence to medication and diagnosis should be reassessed and mimicking diseases excluded. Mycophenolate mofetil is the preferred second-line agent. Further therapy should be individualized, with adjustments made only after 6-12 months.