BACKGROUND Ambrisentan has shown effectiveness in the treatment of Group 1 pulmonary arterial hypertension (PAH). Although portopulmonary hypertension (PoPH) is a subset of Group 1 PAH, few clinical trials have been testing PAH therapies in patients with PoPH. The objective of this study is to evaluate the efficacy and safety of ambrisentan in PoPH. METHODS This study is a prospective, multicenter, open-label trial in which treatment-naive patients with PoPH with Child-Pugh class A/B were administered with ambrisentan for 24 weeks, followed by a long-term extension (24-28 weeks). The primary end-points were change in pulmonary vascular resistance (PVR) and 6-minutes walk distance (6MWD) at 24 weeks, whereas secondary end-points included safety, World Health Organization (WHO) functional class (FC) and echocardiographic assessments. RESULTS Of the 31 patients, 23 finished 24 weeks of ambrisentan therapy and 19 finished the extension. PVR decreased significantly (mean ± SD) (7.1 ± 5 vs 3.8 ± 1.8 Wood units, p < 0.001), whereas 6MWD remained unchanged (314 ± 94 vs 336 ± 108 m). Other hemodynamic parameters such as right atrial pressure (13 ± 8 vs 9 ± 4 mm Hg, p < 0.05), mean pulmonary arterial pressure (46 ± 13 vs. 38 ± 8 mm Hg, p < 0.01), cardiac index (2.6 ± 0.6 vs. 3.5 ± 0.7 liter/min/m2, p < 0.001) showed improvement, whereas pulmonary capillary wedge pressure remained unchanged. Of the 22 patients with WHO FC assessments at baseline and 24 weeks, WHO FC improved significantly (p = 0.005). Most frequent drug-related adverse events were edema (38.7%) and headache (22.5%). One episode of leg edema resulted into the permanent discontinuation of ambrisentan. CONCLUSIONS Ambrisentan monotherapy in PoPH improves hemodynamics and FC at 24 weeks; however, it did not show any improvement in 6MWD. These preliminary outcomes should be interpreted with caution (Clinicaltrials.Gov:NCT01224210).

中文翻译：

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Ambrisentan治疗肺动脉高压：一项多中心，开放标签的试验。

背景技术Ambrisentan已显示出可有效治疗1组肺动脉高压（PAH）。尽管肺动脉高压（PoPH）是第1组PAH的一部分，但很少有临床试验测试PoPH患者的PAH疗法。这项研究的目的是评估Ambrisentan在PoPH中的疗效和安全性。方法该研究是一项前瞻性，多中心，开放标签的试验，其中对未接受过治疗的Child-Pugh A / B级PoPH的初次治疗的患者使用安贝生坦治疗24周，然后进行长期延长（24-28周） 。主要终点是24周时肺血管阻力（PVR）和6分钟步行距离（6MWD）的变化，而次要终点包括安全性，世界卫生组织（WHO）功能类别（FC）和超声心动图评估。结果31例患者中，有23例完成了伏瑞生坦治疗24周，有19例完成了扩展。PVR显着降低（平均值±SD）（7.1±5 vs 3.8±1.8木材单位，p <0.001），而6MWD保持不变（314±94 vs 336±108 m）。其他血液动力学参数，例如右心房压力（13±8 vs 9±4 mm Hg，p <0.05），平均肺动脉压（46±13 vs. 38±8 mm Hg，p <0.01），心脏指数（2.6± 0.6 vs. 3.5±0.7升/分钟/ m2，p <0.001）有所改善，而肺毛细血管楔压保持不变。在基线和第24周接受WHO FC评估的22例患者中，WHO FC显着改善（p = 0.005）。最常见的药物相关不良事件为水肿（38.7％）和头痛（22.5％）。一次腿部水肿导致安布森坦永久停用。结论PoPH中的Ambrisentan单一疗法可改善24周血流动力学和FC。但是，它的6MWD没有任何改善。这些初步结果应谨慎解释（Clinicaltrials.Gov:NCT01224210）。