In > 80% of patients with
autoimmune hepatitis,
steroid therapy alone or in combination with
azathioprine results in disease remission. Treatment response results in reversal of
fibrosis and excellent long-term survival in many patients, whereas untreated patients may expect a 10-year survival of < 30%. The use of
azathioprine monotherapy (2 mg/kg/day) has gained widespread acceptance in maintaining remission in clinical practice. Although all patients with
autoimmune hepatitis may not need treatment, particularly those with mild disease, alternative strategies are required in patients who have failed to achieve remission on standard
therapy of
steroids with or without
azathioprine, or patients with
azathioprine-induced
drug toxicity. In such circumstances, the use of
salvage therapy in the form of
ciclosporin,
tacrolimus or
mycophenolate mofetil may be warranted.
Liver transplantation is the treatment of choice for patients who present with subacute
liver failure or decompensated
cirrhosis.
Salvage therapy results in an exponential rise in cost with each increment in therapeutic escalation. As an alternative to standard
therapy, it has also been suggested that novel
therapies such as
ciclosporin,
tacrolimus or
mycophenolate mofetil be initiated to achieve remission. However, a > 10-fold cost differential exists between the charges associated with more potent immunosuppression and standard
therapy. Therefore, in evaluating novel immunosuppression in
autoimmune hepatitis, it behoves clinicians not only to consider end points pertaining to efficacy, but also end points pertaining to cost-effectiveness. Moreover, the exact role of pharmacogenomics and genotyping of
thiopurine methyltransferase in patients with
autoimmune hepatitis needs to be fully defined.