Autoimmune hepatitis/sclerosing cholangitis overlap syndrome in childhood: a 16-year prospective study.
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.
Gregorio
G V
GV
Department of Child Health, Institute of Liver Studies, and Department of Radiology, King's College Hospital, London, England.
Portmann
B
B
Karani
J
J
Harrison
P
P
Donaldson
P T
PT
Vergani
D
D
Mieli-Vergani
G
G
D000293:Adolescent; D001323:Autoantibodies / Q000032:analysis*; D002648:Child; D002675:Child, Preschool; D002760:Cholangiopancreatography, Endoscopic Retrograde; D015209:Cholangitis, Sclerosing / Q000150:complications* / Q000175:diagnosis / Q000276:immunology* / Q000628:therapy; D005260:Female; D005500:Follow-Up Studies; D006680:HLA Antigens / Q000032:analysis / Q000145:classification; D019693:Hepatitis, Autoimmune / Q000150:complications* / Q000175:diagnosis / Q000628:therapy; D006801:Humans; D007165:Immunosuppression Therapy; D008099:Liver / Q000473:pathology; D008297:Male; D011446:Prospective Studies; D013577:Syndrome
D001323:Autoantibodies; D006680:HLA Antigens