Table 2.
Clinical characteristics of primary biliary cholangitis in South Korea: Result of 2 retrospective studies and a population-based study using rare intractable disease (RID) registry
| Multicenter study | Single center study | RID analysis study | |
|---|---|---|---|
| Kim KA, et al. [28] (2010) | Park Y, et al. [29] (2015) | Kim KA, et al. [25] (2016) | |
| Study design | Retrospective, multicenter | Retrospective single center | Administrative database analysis |
| Year of enrollment | 1997-2008 | 2001-2011 | 2009-2013 |
| Number of enrolled patients | 251 | 81 | 2824 (.20 year old) |
| Diagnostic criteria | Diagnostic criteria (≥ 2/3) | Paris criteria (≥ 2/3) | ICD-10 code registered in Rare intractable disease registry |
| 1) Elevated ALP with GGT | 1) ALP > 2 x UNL or GGT > 5 xUNL | ||
| 2) AMA+ (≥1:40) | 2) AMA+ | ||
| 3) Compatible histology | 3) florid bile duct lesion on liver biopsy | ||
| Age (year), mean/median | 54 | 53 | 57.4 |
| Female/male ratio | 6.6 | 5.2 | 6.2 |
| Cirrhosis at presentation | 23.5% | 32.3% | |
| Asymptomatic | 60.6% | ||
| Hepatic decompensation | 12.3% | ||
| Autoantibody positive rate | Autoantibody testing rate | ||
| ANA | 63.5% | 47% | 74% |
| AMA | 98.4% | 100% | 92.3% |
| Alkaline phosphatase, mean | 2.6 x UNL | 265 IU/L | |
| Total bilirubin (mg/dL), mean/median | 1.9 | 0.9 | |
| Immunoglobulin M >1xUNL | 74% | Mean 533 mg/dL | |
| Liver biopsy performed | 40% | 35% | |
| Comorbidities | Hyperlipidemia, 17% | ||
| Autoimmune hepatitis, 11% | |||
| Thyroid disease, 6.5% | |||
| Sjogren disease, 0.8% | |||
| Systemic lupus erythematosus, 0.5% | |||
| Systemic sclerosis, 0.3% | |||
| Rheumatoid arthritis, 0.5% | |||
| Therapy regimen | (n=244) | 2013 prescription | |
| UDCA | 86% | 100% | 93% |
| Corticosteroid/ cyclosporine/ azathioprine/ colchicine | 16%/7%/2%/0.4% | 9%/- / 5%/- | |
| 7% | |||
| UDCA response at 1yr | (n=221) | (n=65) | |
| 70% (ALP reduction >40% or normal range) | 72.3% (ALP <3 xUNL, AST < 2UNL, Bilirubin <1 mg/dL) | ||
| Overall survival rate | |||
| 5 year | 95% | 95% | |
| Case fatality rate, 2.2% during 5 years | |||
| Liver transplantation | 2.5% | ||
| Hepatocellular carcinoma at diagnosis | 1.3% |
ALP, alkaline phosphatase; UNL, upper normal limit; GGT, gamma glutamyltransferase; AMA, anti-mitochondrial antibody; ANA, anti-nuclear antibody; UDCA, ursodeoxycholic acid.