Low-risk individuals with primary biliary cholangitis and significant liver stiffness: prognosis and treatment.

BACKGROUND

Some patients treated with ursodeoxycholic acid (UDCA) or combined fenofibrate had well-controlled biochemical parameters but high liver stiffness, and the prognosis as well as therapeutic options for these patients may be an area worthy of further exploration.

AIMS: To explore the prognosis and treatment of patients with low-risk and high liver stiffness.

METHODS

A retrospective study included 424 cases of UDCA monotherapy and 102 cases of combined fenofibrate treatment.

RESULTS

The combination of liver stiffness measurement (LSM) and the GLOBE score improved prognostic prediction for patients with UDCA monotherapy (area under the receiver operating characteristic curve [AUC] of 0.868 (0.811-0.925) for the fitted model and 0.834 (0.767-0.900) for the GLOBE score, p = 0.006). Further analyses revealed that LSM had an additive prognostic effect mainly in low-risk patients defined by GLOBE < 0.5 (AUC, 0.777 [0.724-0.825] vs 0.642 [0.583-0.699], p = 0.001). For patients in the low-risk group, the prognosis was worse when LSM > 11 kPa (7/53 [13%] vs 2/227 [1%], p = 0.001). The prognosis was consistent between patients in the "low-risk and LSM > 11 kPa" group and the medium-risk group defined by 0.5 < GLOBE < 1.8 (7/53 [13%] vs 22/121 [18%], p = 0.418). In low-risk patients treated with combined fenofibrate therapy, the prognosis was worse when LSM > 11 kPa (3/21 [14%] vs 0/47 [0%], p = 0.022). The prognosis was consistent between patients in the "low-risk and LSM > 11 kPa" and the medium-risk groups (3/21 [14%] vs 6/27 [22%], p = 0.353). Antifibrotic drugs failed to reduce the incidence of the primary outcome (5/45 [11%] vs 5/27 [19%], p = 0.598), and delayed the progression of LSM in patients with low-risk and LSM > 11 kPa at 36 months of follow-up (changes in LSM, - 3.31 [- 5.04 to - 1.52] vs - 1.74 [- 2.83 to 1.5], p = 0.046).

CONCLUSIONS

Patients with GLOBE-defined low-risk and LSM > 11 kPa had a poor prognosis, and antifibrotic therapy may slow the progression of liver stiffness in these patients.