Autoimmune hepatitis/sclerosing cholangitis overlap syndrome in childhood: a 16-year prospective study

Hepatology. 2001 Mar;33(3):544-53. doi: 10.1053/jhep.2001.22131.


To investigate whether sclerosing cholangitis with an autoimmune serology characteristic of autoimmune hepatitis (AIH) and AIH are distinct entities, we studied 55 consecutive children with clinical and/or biochemical evidence of liver disease and circulating antinuclear (ANA), anti-smooth muscle (SMA), and/or liver-kidney-microsomal type 1 (LKM1) autoantibodies. They underwent liver biopsy, direct cholangiography, sigmoidoscopy, and rectal biopsy at presentation. Twenty-eight were diagnosed as AIH in the absence and 27 autoimmune sclerosing cholangitis (ASC) in the presence of radiological features of cholangiopathy. Twenty-six ASC and 20 AIH had ANA and/or SMA; 1 ASC and 8 AIH LKM1 autoantibody. Similarities between the 2 conditions included most clinical and biochemical parameters and a lower frequency of HLA DR4. Inflammatory bowel disease and histological biliary changes were more common in ASC; coagulopathy, hypoalbuminemia, lymphocytic periportal hepatitis, and HLA DR3 were more common in AIH. Histological biliary changes were observed in 65% of ASC and 31% of AIH patients. Eighty-nine percent responded to immunosuppression. Follow-up liver biopsies from 17 ASC and 18 AIH patients had similarly reduced inflammatory activity and no progression to cirrhosis. Sixteen follow-up cholangiograms from AIH patients and 9 from ASC patients were unchanged, while 8 ASC patients showed a progressive cholangiopathy. One child with AIH and ulcerative colitis developed sclerosing cholangitis 8 years after presentation. At 2 to 16 years (median, 7 years) from presentation, all patients are alive, including 4 ASC patients who underwent liver transplantation. In conclusion, ASC and AIH are similarly prevalent in childhood; cholangiography is often needed to distinguish between these 2 entities, which are likely to lie within the same disease process.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Autoantibodies / analysis*
  • Child
  • Child, Preschool
  • Cholangiopancreatography, Endoscopic Retrograde
  • Cholangitis, Sclerosing / complications*
  • Cholangitis, Sclerosing / diagnosis
  • Cholangitis, Sclerosing / immunology*
  • Cholangitis, Sclerosing / therapy
  • Female
  • Follow-Up Studies
  • HLA Antigens / analysis
  • HLA Antigens / classification
  • Hepatitis, Autoimmune / complications*
  • Hepatitis, Autoimmune / diagnosis
  • Hepatitis, Autoimmune / therapy
  • Humans
  • Immunosuppression
  • Liver / pathology
  • Male
  • Prospective Studies
  • Syndrome


  • Autoantibodies
  • HLA Antigens