Testing for antimitochondrial
antibodies is the most useful laboratory procedure in the diagnosis of
primary biliary cirrhosis; nevertheless, 5-10% of patients with typical features of
primary biliary cirrhosis do not have detectable antimitochondrial
antibodies, their condition being referred to as antimitochondrial antibody-negative
primary biliary cirrhosis or "autoimmune
cholangitis". Uncertainty exists whether antimitochondrial antibody-positive and -negative
primary biliary cirrhosis represent distinct entities. We reviewed studies that compared: (i) the clinical, laboratory and histological characteristics of antimitochondrial antibody-positive and -negative
primary biliary cirrhosis; (ii) the response to treatment of both conditions; and (iii) the response of autoimmune
cholangitis to
ursodeoxycholic acid and immunosuppressive therapy. Antimitochondrial antibody-positive and -negative
primary biliary cirrhosis were characterized by similar clinical, laboratory and histological abnormalities,
clinical course and survival. Antimitochondrial antibody status did not seem to affect the response to
ursodeoxycholic acid. At present, the efficacy of
therapies for autoimmune
cholangitis has not been established in controlled trials. Of 52 patients with autoimmune
cholangitis treated with
ursodeoxycholic acid in 13 uncontrolled studies, 83% had serum biochemical improvement. Also, a favourable effect of immunosuppressive drugs occurred in 57% of 54 patients with autoimmune
cholangitis in 17 uncontrolled studies. Each of these trials included very few patients and most evaluated the effects of treatment on
surrogate markers of disease only. No marker that consistently distinguished patients who would respond favourably to
ursodeoxycholic acid or immunosuppression was apparent. Consequently, treatment is, at present, empirical. However,
ursodeoxycholic acid may be given when histology reveals bile duct lesions, whereas immunosuppressive therapy should probably be reserved for patients exhibiting interface
hepatitis.