Histological Features of Primary Biliary Cirrhosis Predict Biological Response to Therapy and Outcome
Yuko Kakuda, Kenichi Harada, Yasunori Sato, Motoko Sasaki, Yasuni Nakanuma. Kanazawa University Graduate School of Medicine, Kanazawa, Japan
Background: Ursodeoxycholic acid (UDCA) is the standard treatment for primary biliary cirrhosis. UDCA nonresponders have a poorer prognosis and need additional therapies. We retrospectively compared the histological features of nonresponders and responders.
Design: Sixty-nine pretreatment liver biopsy specimens and clinical data were analyzed. Cases were divided into 3 groups: advanced [n = 10, with cirrhosis-related conditions; histologically proven cirrhosis or cirrhosis-related complications and/or symptoms, i.e., ascites, ruptured and/or endoscopically treated gastroesophageal varices, hepatic encephalopathy, hyperbilirubinemia (≥2.0 mg/dL), or hepatocellular carcinoma]; nonresponders (n = 16, with subsequent cirrhosis-related conditions and/or histological stage progression); and responders [n = 43, without these conditions or stage progression during at least 5 (11 ± 5) years of follow-up]. Histological evaluations were performed as per a new system proposed recently, i.e., staging was evaluated as per 3 histological components (fibrosis, bile duct loss, and orcein-positive granule deposition).
Results: Stage distribution of the 3 groups was as follows: stage 1/2/3/4 = 0/2/2/6 in advanced cases, 0/9/5/2 in nonresponders, and 9/31/3/0 in responders. All cases with a fibrosis score of 0 were responders. Almost all cases with an orcein-positive granule deposition score of 2–3 were advanced and nonresponders (except 1 receiving immunosuppressants for rheumatoid arthritis). Orcein-positive granule deposition scores of ≥2, ≥4, and ≥5, sum of orcein-positive granule deposition + fibrosis scores, and sum of bile duct loss + orcein-positive granule deposition + fibrosis scores showed 58% sensitivity and 98% specificity for distinguishing advanced and nonresponders from responders.
Conclusions: Our results showed the association between liver biopsy features and response to therapies and patient outcome. Disease progression risk may be evaluated within 1 year as follows:
Tuesday, March 5, 2013 1:00 PM
Poster Session IV # 156, Tuesday Afternoon