Abstract：
Aetiological therapy improves liver function and may enable hepatic recompensation in decompensated cirrhosis.
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.
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.
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.
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.
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.
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.
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.
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：病因学治疗可改善肝功能，并可能使失代偿期肝硬化的肝再补偿。
目的：我们探索了失代偿性原发性胆汁性胆管炎（PBC）患者的再补偿潜力——考虑了根据Paris-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。
方法：回顾性纳入首次失代偿时的PBC患者。再代偿定义为：（i）尽管停止利尿剂/HE治疗，腹水和肝性脑病（HE）的消退，（ii）没有静脉曲张出血和（iii）持续的肝功能改善。
结果：总计，纳入42例失代偿期肝硬化患者（年龄：63.5[IQR：51.9-69.2]岁；88.1％女性；MELD-Na：13.5[IQR：11.0-15.0]），并在失代偿后随访41.9（IQR：11.0-70.9）个月。7名患者（16.7％）实现了再补偿。较低的MELD-Na（子分布危险比[SHR]：0.90；p=0.047），失代偿时的胆红素（SHR/mg/dL：0.44;p=0.005）和碱性磷酸酶（SHR/10U/L：0.67;p=0.001），以及静脉曲张出血作为失代偿事件（SHR：4.37;p=0.069），与更高的补偿概率有关。总的来说,33例患者接受UDCA治疗≥1年，12例（36%）达到巴黎II反应标准。5/12（41.7％）和2/21（9.5％）患者发生再代偿在1年内没有UDCA反应，分别。重组代偿与数字上改善的无移植存活率相关(HR:0.46;p=0.335)。尽管如此，4/7患者在发展为肝脏恶性肿瘤和/或门静脉血栓形成后出现肝脏相关并发症，2例最终死亡。
结论：在UDCA治疗下，PBC和失代偿期肝硬化患者可能实现肝再补偿。然而,由于肝脏相关的并发症在再补偿后仍然会发生，患者应保持密切随访.
目的：我们探索了失代偿性原发性胆汁性胆管炎（PBC）患者的再补偿潜力——考虑了根据Paris-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。
方法：回顾性纳入首次失代偿时的PBC患者。再代偿定义为：（i）尽管停止利尿剂/HE治疗，腹水和肝性脑病（HE）的消退，（ii）没有静脉曲张出血和（iii）持续的肝功能改善。
结果：总计，纳入42例失代偿期肝硬化患者（年龄：63.5[IQR：51.9-69.2]岁；88.1％女性；MELD-Na：13.5[IQR：11.0-15.0]），并在失代偿后随访41.9（IQR：11.0-70.9）个月。7名患者（16.7％）实现了再补偿。较低的MELD-Na（子分布危险比[SHR]：0.90；p=0.047），失代偿时的胆红素（SHR/mg/dL：0.44;p=0.005）和碱性磷酸酶（SHR/10U/L：0.67;p=0.001），以及静脉曲张出血作为失代偿事件（SHR：4.37;p=0.069），与更高的补偿概率有关。总的来说,33例患者接受UDCA治疗≥1年，12例（36%）达到巴黎II反应标准。5/12（41.7％）和2/21（9.5％）患者发生再代偿在1年内没有UDCA反应，分别。重组代偿与数字上改善的无移植存活率相关(HR:0.46;p=0.335)。尽管如此，4/7患者在发展为肝脏恶性肿瘤和/或门静脉血栓形成后出现肝脏相关并发症，2例最终死亡。
结论：在UDCA治疗下，PBC和失代偿期肝硬化患者可能实现肝再补偿。然而,由于肝脏相关的并发症在再补偿后仍然会发生，患者应保持密切随访.
