rum. 1 In the past decade, two treatment modalities have Approximately 5% to 10% of patients with features otherbeen shown to be effective in the treatment for PBC, dewise consistent with primary biliary cirrhosis (PBC) lack antipending on the stage of the disease. For patients with less mitochondrial antibodies (AMA). Most of these patients have advanced disease, ursodeoxycholic acid (UDCA) improves other autoantibodies, a syndrome recently named ''autoimliver biochemistry and delays time to death or liver transmune cholangitis.'' We report our experience in patients with plantation. [2][3][4] Orthotopic liver transplantation (OLT) has AMA-negative PBC treated with ursodeoxycholic acid been shown not only to prolong survival but also to improve (UDCA) and/or liver transplantation (OLT). The study of rethe quality of life in patients with decompensated hepatic sponse to UDCA was performed as follows. While recruiting failure. 5-6 patients for a previously reported multicenter trial, we identified 8 patients with AMA-negative PBC. The patients were
Although the precise role of AMA in the pathogenesis of given UDCA and followed up at regular intervals. The charac-PBC has yet to be uncovered, AMA positivity constitutes an teristics of AMA-negative patients at presentation were similar important element in the diagnosis of PBC. Among patients to those of AMA-positive patients with PBC. The clinical outwith apparent PBC, however, approximately 5% to 10% do comes and sequential liver biochemistries of UDCA treatment not have AMA by indirect immunofluorescence. In 63% to were also comparable with those of AMA-positive patients. 95% of these patients, autoantibodies other than AMA, such The study of outcome of OLT was performed as follows. We as antinuclear antibody (ANA) and/or anti-smooth muscle identified OLT recipients at the Mayo Clinic who had clinical, antibody (ASMA), are positive. [7][8][9] The diagnosis of autoimradiological, and histological features compatible with PBC. mune cholangitis or cholangiopathy (AIC) is based on the Their pretransplant AMA status was determined, and each clinical constellation of chronic cholestasis, histological AMA-negative patient was paired with 2 AMA-positive pachanges of chronic nonsuppurative cholangitis, and the prestients. Of 85 OLT recipients with a diagnosis of PBC, 6 (7.1%) ence of autoantibodies other than AMA. There have been were AMA negative, including 1 who had undergone UDCA suggestions, which remain controversial, that patients with therapy. After a median of 36 months of follow-up, graft and AIC may constitute a separate entity, clinically and immunopatient survival rates and subsequent histological changes logically, from the typical AMA-positive PBC. 7,10 (disease recurrence and steroid-resistant or late rejections)
It is not well documented whether the absence of AMA were comparable in AMA-negative and -positive PBC patients. portends a different response to treatment compared to AMA-In summary, in our experience of 13 AMA-negative PBC papositive PBC. In this report, we summarize our experience tients (including 9 who met the criteria for a diagnosis of in patients with AMA-negative PBC (including those with autoimmune cholangitis), treatment with UDCA or OLT re-AIC) who have been treated with UDCA and/or OLT in comsulted in similar outcomes to those found in AMA-positive parison with AMA-positive PBC patients. For the purpose of patients. We conclude that AMA status does not affect the this report, we will use the term AMA-negative PBC for paresponse in PBC patients to treatment with UDCA or OLT. tients who are AMA-negative but otherwise have clinical fea-(HEPATOLOGY 1997;26:22-26.)
tures compatible with PBC, and the diagnosis of AIC will be reserved for the subgroup of AMA-negative PBC patients who Primary biliary cirrhosis (PBC) is characterized by clinical have positive ANA and/or ASMA. This distinction is made features of chronic cholestasis, histological changes of in part because detailed autoimmune serology profiles relymphocytic destruction of interlobular bile ducts, and the quired for making the latter diagnosis were not available in presence of antimitochondrial antibodies (AMA) in the sesome of the earlier patients.
Methods
Diagnostic Criteria.