Use of sirolimus in the treatment of refractory autoimmune hepatitis

Am J Med. 2014 Nov;127(11):1128-1131. doi: 10.1016/j.amjmed.2014.06.016. Epub 2014 Jun 27.

Abstract

Background: Corticosteroids and azathioprine are widely accepted as the initial therapy for autoimmune hepatitis. However, the disease is refractory to steroids in about 10%-20% of patients, for whom currently there is no standardized treatment. Here we describe our experience with sirolimus in treatment of steroid refractory autoimmune hepatitis.

Methods: This is a longitudinal follow-up study. Between November 2007 and January 2014, 5 subjects with steroid refractory autoimmune hepatitis were treated with sirolimus at our institution.

Results: A response, defined as a sustained >50% fall in alanine aminotransferase (ALT) levels, was achieved in 4/5 patients. A complete response, sustained normalization of ALT levels, was achieved in 2/5 patients. The need for steroids was significantly reduced in all patients (P < .05).

Conclusions: In this small series, sirolimus appears to be useful in the treatment of patients with steroid refractory autoimmune hepatitis.

Keywords: Autoimmune hepatitis; Sirolimus; Steroid-refractory.

MeSH terms

  • Adult
  • Alanine Transaminase / blood
  • Aspartate Aminotransferases / blood
  • Azathioprine / therapeutic use
  • Bilirubin / blood
  • Drug Resistance
  • Female
  • Glucocorticoids / therapeutic use
  • Hepatitis, Autoimmune / drug therapy*
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Longitudinal Studies
  • Male
  • Middle Aged
  • Mycophenolic Acid / analogs & derivatives
  • Mycophenolic Acid / therapeutic use
  • Prednisone / therapeutic use
  • Sirolimus / therapeutic use*
  • Young Adult

Substances

  • Glucocorticoids
  • Immunosuppressive Agents
  • Aspartate Aminotransferases
  • Alanine Transaminase
  • Mycophenolic Acid
  • Azathioprine
  • Bilirubin
  • Prednisone
  • Sirolimus