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

Abstract

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.

Keywords: Liver stiffness; Prognosis; Risk stratification; The GLOBE score.