Weinberg Ethan, Stonelake Aimee, Borges Kelly, Toal Emily, Aghayeva Sevda, Rossman Milton, Ligon Colin, Reddy K Rajender
Division of Gastroenterology and Hepatology, University of Pennsylvania Perelman School of Medicine, USA.
Division of Pulmonary, Allergy and Critical Care, University of Pennsylvania, USA.
Clin Exp Hepatol. 2024 Sep;10(3):194-196. doi: 10.5114/ceh.2024.141732. Epub 2024 Jul 25.
Sarcoidosis is characterized by noncaseating granulomas that can affect multiple organs. Due to the lack of prospective studies regarding treatment of hepatic sarcoidosis with ursodeoxycholic acid (UDCA), we set out to evaluate its effects in a single-center, open-label, prospective, pre-post study.
A total of 10 patients were screened from August 2018 to July 2020; seven met the criteria and were enrolled. The study was terminated prior to achieving target enrollment of 10 patients due to the difficulty in recruitment around the COVID-19 pandemic. Most patients were women (4/7; 57.1%) and African American (5/7; 71.4%). One patient dropped out during the first month of observation due to a new diagnosis of esophageal cancer. Six completed the 6-month observation and UDCA treatment periods. One patient stopped UDCA within the first month of active treatment due to the side effect of nausea.
There was a decrease in ALP and GGT after six months of UDCA treatment compared to six months of observation (ALP - 257.6 to 202.2, = 0.23; GGT - 302.5 to 111.8, = 0.059), but this did not reach statistical significance. There were also decreases in all key secondary endpoints (ALT - 50.8 to 29.8, = NS; AST - 40.3 to 31.2, = NS, VCTE kPa - 8.3 to 6.3, = NS). As with the primary endpoints, none of the key secondary endpoints reached statistical significance.
There is significant potential for UDCA as first-line treatment of hepatic sarcoid. Multi-center, ideally prospective, studies of longer duration are needed.
结节病的特征是形成非干酪样肉芽肿，可累及多个器官。由于缺乏关于熊去氧胆酸（UDCA）治疗肝结节病的前瞻性研究，我们开展了一项单中心、开放标签、前瞻性、前后对照研究，以评估其疗效。
2018年8月至2020年7月共筛查了10例患者；7例符合标准并纳入研究。由于在新冠疫情期间招募困难，该研究在未达到10例患者的目标入组人数之前就终止了。大多数患者为女性（4/7；57.1%）且为非裔美国人（5/7；71.4%）。1例患者在观察的第一个月因新诊断出食管癌而退出。6例完成了6个月的观察和UDCA治疗期。1例患者在积极治疗的第一个月内由于恶心的副作用而停用了UDCA。
与6个月的观察期相比，UDCA治疗6个月后碱性磷酸酶（ALP）和γ-谷氨酰转移酶（GGT）有所下降（ALP - 从257.6降至202.2，P = 0.23；GGT - 从302.5降至111.8，P = 0.059），但未达到统计学显著性。所有关键次要终点指标也有所下降（谷丙转氨酶（ALT） - 从50.8降至29.8，P = 无统计学意义；谷草转氨酶（AST） - 从40.3降至31.2，P = 无统计学意义，瞬时弹性成像（VCTE）kPa - 从8.3降至6.3，P = 无统计学意义）。与主要终点指标一样，关键次要终点指标均未达到统计学显著性。
UDCA作为肝结节病一线治疗具有显著潜力。需要开展多中心、理想情况下为前瞻性的、持续时间更长的研究。
