Department of Gastroenterology and Hepatology, University Hospitals KU Leuven, Belgium.
Best Pract Res Clin Gastroenterol. 2018 Jun-Aug;34-35:107-111. doi: 10.1016/j.bpg.2018.09.001. Epub 2018 Sep 11.
Despite near universal use of ursodeoxycholic acid (UDCA) several patients with PBC still progress to liver transplant (LT) or death. Pruritus and fatigue are the most common symptoms. Liver transplantation for pruritus is highly effective but fatigue will not disappear in the majority of the patients. In contrast to other liver diseases, portal hypertension may develop in pre-cirrhotic patients with PBC. Patients with PBC have an incidence rate of 3.4 hepatocellular carcinoma cases for every 1000 patient-years and risk factors are advanced stage of the disease and male sex. For the appropriate timing of LT the utility of prognostic models (bilirubin, Mayo risk score and MELD, in particular) and standard exception points in case of HCC are established. However, recent data from different part of the world demonstrated that PBC patients compared to patients with PSC have higher waiting-list mortality. Hyperlipidemia can be present in up to 80% of the patients but there is no evidence for an elevated cardiovascular risk, certainly not in relationship with LT. Patients transplanted for PBC suffer more frequently from acute cellular and also late cellular rejection. However, 5-year patient survival rates after LT of 80-85% is better than for most other indications. Recurrent PBC is reported in a range from 14% up to 42% after LT but in contrast to other autoimmune diseases graft loss due to recurrent disease is not a major issue. The type of immunosuppression after LT was found to be associated with the incidence of recurrence but since mediate-term impact on overall and graft survival is negligible, tacrolimus-based regimens remain standard at most centers. Observational studies suggest that long-term administration of UDCA following LT has a beneficial effect on recurrence of PBC. Therefore biomarkers after LT that may identify patients at risk for recurrence should be further explored to allows early medical intervention.
尽管熊去氧胆酸（UDCA）已被广泛应用，但仍有部分 PBC 患者需要进行肝移植（LT）或死亡。瘙痒和疲劳是最常见的症状。肝移植治疗瘙痒非常有效，但大多数患者的疲劳症状不会消失。与其他肝病不同，PBC 患者在肝硬化前可能会发生门脉高压。PBC 患者的肝细胞癌发病率为每 1000 患者年 3.4 例，危险因素为疾病晚期和男性。对于 LT 的适当时机，已经建立了预后模型（胆红素、梅奥风险评分和 MELD，特别是）和 HCC 标准例外点的效用。然而，来自世界不同地区的最新数据表明，与 PSC 患者相比，PBC 患者的等待名单死亡率更高。高达 80%的患者可能存在血脂异常，但没有证据表明心血管风险升高，当然与 LT 无关。接受 PBC 治疗的患者更频繁地发生急性和迟发性细胞排斥反应。然而，LT 后 5 年患者生存率为 80-85%，优于大多数其他适应证。LT 后报道的 PBC 复发率为 14%至 42%，但与其他自身免疫性疾病不同，由于复发性疾病导致移植物丢失不是主要问题。LT 后免疫抑制的类型与复发的发生率有关，但由于对总体和移植物存活率的中期影响可以忽略不计，因此他克莫司为基础的方案在大多数中心仍是标准方案。观察性研究表明，LT 后长期使用 UDCA 对 PBC 的复发有有益影响。因此，应该进一步探索 LT 后的生物标志物，以识别有复发风险的患者，以便早期进行医学干预。
