Characteristics | Adjusted HR (95% CI)* | P value HR | Untreated LT-free survival5y (95% CI) | NNT5y (95% CI)†¶ | Untreated LT-free survival10y (95% CI) | NNT10y (95% CI)†¶ |
Sex | ||||||
Male | 0.52 (0.35 to 0.77) | 0.0011 | 0.68 (0.60 to 0.76) | 8 (5 to 21) | 0.55 (0.46 to 0.64) | 6 (4 to 15) |
Female | 0.44 (0.38 to 0.52) | <0.0001 | 0.82 (0.80 to 0.84) | 11 (9 to 14) | 0.62 (0.59 to 0.64) | 6 (5 to 7) |
Age (years) | ||||||
≤46.0 | 0.33 (0.24 to 0.46) | <0.0001 | 0.83 (0.79 to 0.86) | 9 (7 to 14) | 0.60 (0.55 to 0.66) | 5 (3 to 6) |
46.0–62.7 | 0.46 (0.37 to 0.56) | <0.0001 | 0.80 (0.78 to 0.83) | 10 (8 to 14) | 0.67 (0.64 to 0.71) | 7 (5 to 9) |
>62.7 | 0.60 (0.48 to 0.76) | <0.0001 | 0.81 (0.77 to 0.84) | 14 (9 to 28) | 0.52 (0.47 to 0.58) | 7 (5 to 13) |
Cirrhosis‡ | ||||||
No | 0.32 (0.24 to 0.42) | <0.0001 | 0.92 (0.90 to 0.95) | 20 (14 to 34) | 0.71 (0.66 to 0.76) | 6 (5 to 8) |
Yes | 0.31 (0.24 to 0.40) | <0.0001 | 0.48 (0.42 to 0.54) | 4 (3 to 5) | 0.33 (0.27 to 0.39) | 3 (3 to 4) |
Disease stage§ | ||||||
Early | 0.37 (0.30 to 0.47) | <0.0001 | 0.92 (0.91 to 0.94) | 22 (17 to 32) | 0.78 (0.75 to 0.80) | 8 (6 to 11) |
Intermediate | 0.32 (0.25 to 0.40) | <0.0001 | 0.62 (0.57 to 0.67) | 5 (4 to 6) | 0.22 (0.17 to 0.28) | 3 (3 to 4) |
Advanced | 0.50 (0.37 to 0.70) | 0.0001 | 0.26 (0.20 to 0.34) | 5 (3 to 8) | 0.14 (0.92 to 0.20) | 5 (4 to 9) |
ALP | ||||||
≤2× ULN | 0.61 (0.45 to 0.82) | 0.0014 | 0.90 (0.87 to 0.92) | 26 (15 to 70) | 0.79 (0.75 to 0.82) | 13 (8 to 35) |
2–4× ULN | 0.46 (0.36 to 0.59) | <0.0001 | 0.82 (0.79 to 0.85) | 11 (8 to 17) | 0.59 (0.56 to 0.64) | 6 (4 to 8) |
>4× ULN | 0.36 (0.25 to 0.52) | <0.0001 | 0.66 (0.62 to 0.70) | 5 (4 to 8) | 0.41 (0.36 to 0.46) | 4 (3 to 5) |
Bilirubin | ||||||
≤ULN | 0.39 (0.32 to 0.48) | <0.0001 | 0.91 (0.90 to 0.92) | 19 (15 to 27) | 0.75 (0.72 to 0.78) | 7 (6 to 10) |
>ULN | 0.40 (0.33 to 0.48) | <0.0001 | 0.49 (0.45 to 0.53) | 4 (4 to 5) | 0.20 (0.16 to 0.25) | 4 (3 to 4) |
Albumin | ||||||
<LLN | 0.32 (0.24 to 0.43) | <0.0001 | 0.35 (0.29 to 0.41) | 3 (3 to 4) | 0.15 (0.11 to 0.21) | 3 (3 to 4) |
≥LLN | 0.46 (0.40 to 0.54) | <0.0001 | 0.87 (0.86 to 0.89) | 16 (13 to 21) | 0.68 (0.66 to 0.71) | 7 (6 to 9) |
Platelet count | ||||||
<150×109/L | 0.48 (0.35 to 0.46) | 0.0007 | 0.52 (0.47 to 0.58) | 5 (4 to 9) | 0.27 (0.22 to 0.34) | 4 (3 to 7) |
≥150×109/L | 0.44 (0.37 to 0.52) | <0.0001 | 0.86 (0.84 to 0.87) | 14 (11 to 18) | 0.68 (0.65 to 0.70) | 7 (5 to 8) |
*HRs were adjusted for sex, age, year of diagnosis, albumin, platelet count, bilirubin, alkaline phosphatase, aspartate aminotransferase and alanine aminotransferase;.
†The 95% CI of the NNT was based on both the 95% CI of the adjusted HR of UDCA as well as on the 95% CI of the cumulative 5-year LT-free survival in patients without UDCA therapy.
‡Baseline histological data were available for 2173 patients;.
§Biochemical disease stage according to Rotterdam criteria.29
¶As the number needed to treat always needs to be rounded up, decimal differences in absolute clinical efficacy cannot be presented.
ALP, alkaline phosphatase; LLN, lower limit of normal; LT, liver transplantation; NNT, number needed to treat; PBC, primary biliary cholangitis; UDCA, ursodeoxycholic acid; ULN, upper limit of normal; 5y, 5 years; 10y, 10 years.