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原发性胆汁性胆管炎失代偿期肝硬化患者潜在肝脏补偿标准的评估。

Evaluation of potential hepatic recompensation criteria in patients with PBC and decompensated cirrhosis.

机构信息

Division of Gastroenterology and Hepatology, Department of Medicine III, Medical University of Vienna, Vienna, Austria.

Vienna Hepatic Hemodynamic Lab, Division of Gastroenterology and Hepatology, Department of Medicine III, Medical University of Vienna, Vienna, Austria.

出版信息

Aliment Pharmacol Ther. 2024 Apr;59(8):962-972. doi: 10.1111/apt.17908. Epub 2024 Feb 26.

Abstract

BACKGROUND

Aetiological therapy improves liver function and may enable hepatic recompensation in decompensated cirrhosis.

AIMS

We explored the potential for recompensation in patients with decompensated primary biliary cholangitis (PBC) - considering a biochemical response to ursodeoxycholic acid (UDCA) according to Paris-II criteria as a surrogate for successful aetiological treatment.

METHODS

Patients with PBC were retrospectively included at the time of first decompensation. Recompensation was defined as (i) resolution of ascites and hepatic encephalopathy (HE) despite discontinuation of diuretic/HE therapy, (ii) absence of variceal bleeding and (iii) sustained liver function improvement.

RESULTS

In total, 42 patients with PBC with decompensated cirrhosis (age: 63.5 [IQR: 51.9-69.2] years; 88.1% female; MELD-Na: 13.5 [IQR: 11.0-15.0]) were included and followed for 41.9 (IQR: 11.0-70.9) months after decompensation. Seven patients (16.7%) achieved recompensation. Lower MELD-Na (subdistribution hazard ratio [SHR]: 0.90; p = 0.047), bilirubin (SHR per mg/dL: 0.44; p = 0.005) and alkaline phosphatase (SHR per 10 U/L: 0.67; p = 0.001) at decompensation, as well as variceal bleeding as decompensating event (SHR: 4.37; p = 0.069), were linked to a higher probability of recompensation. Overall, 33 patients were treated with UDCA for ≥1 year and 12 (36%) achieved Paris-II response criteria. Recompensation occurred in 5/12 (41.7%) and in 2/21 (9.5%) patients with vs. without UDCA response at 1 year, respectively. Recompensation was linked to a numerically improved transplant-free survival (HR: 0.46; p = 0.335). Nonetheless, 4/7 recompensated patients presented with liver-related complications after developing hepatic malignancy and/or portal vein thrombosis and 2 eventually died.

CONCLUSIONS

Patients with PBC and decompensated cirrhosis may achieve hepatic recompensation under UDCA therapy. However, since liver-related complications still occur after recompensation, patients should remain under close follow-up.

摘要

背景

病因治疗可改善肝功能,并可能使失代偿性肝硬化恢复代偿。

目的

我们探讨了原发性胆汁性胆管炎(PBC)失代偿患者恢复代偿的潜力 - 考虑根据巴黎 II 标准对熊去氧胆酸(UDCA)的生化反应作为成功病因治疗的替代指标。

方法

回顾性纳入失代偿时的 PBC 患者。恢复代偿定义为:(i)尽管停止使用利尿剂/HE 治疗,腹水和肝性脑病(HE)仍得到解决,(ii)无静脉曲张出血,(iii)肝功能持续改善。

结果

共纳入 42 例 PBC 失代偿性肝硬化患者(年龄:63.5 [IQR:51.9-69.2] 岁;88.1%为女性;MELD-Na:13.5 [IQR:11.0-15.0]),失代偿后随访 41.9(IQR:11.0-70.9)个月。7 例患者(16.7%)达到恢复代偿。较低的 MELD-Na(亚分布风险比 [SHR]:0.90;p=0.047)、胆红素(每毫克/分升 SHR:0.44;p=0.005)和碱性磷酸酶(每 10 U/L SHR:0.67;p=0.001)在失代偿时,以及静脉曲张出血作为失代偿事件(SHR:4.37;p=0.069),与更高的恢复代偿概率相关。总体而言,33 例患者接受 UDCA 治疗≥1 年,12 例(36%)达到巴黎 II 反应标准。在 1 年内,12 例患者中有 5 例(41.7%)和 21 例患者中有 2 例(9.5%)达到 UDCA 反应标准,出现恢复代偿。恢复代偿与移植无相关生存率的改善相关(HR:0.46;p=0.335)。尽管如此,在发生肝恶性肿瘤和/或门静脉血栓形成后,7 例恢复代偿的患者中有 4 例出现与肝脏相关的并发症,其中 2 例最终死亡。

结论

在 UDCA 治疗下,PBC 失代偿性肝硬化患者可能恢复代偿。然而,由于恢复代偿后仍会发生肝脏相关并发症,因此患者仍应密切随访。

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