Design and rationale for a real-world observational cohort of patients with nonalcoholic fatty liver disease: The TARGET-NASH study
Design and rationale for a real-world observational cohort of patients with nonalcoholic fatty liver disease: The TARGET-NASH study
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DOI：
10.1016/j.cct.2017.07.015
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发表时间：
2017-10-01
影响因子：
2.2
通讯作者：
Sanyal, Arun
中科院分区：
文献类型：
--
作者：
Barritt, A. S.;Gitlin, Norman;Sanyal, Arun
非酒精性脂肪性肝病（NAFLD）非常普遍，可导致肝硬化、肝细胞癌和终末期肝病。 NAFLD 包括从单纯性脂肪变性（非酒精性脂肪肝，NAFL）到伴有炎症的脂肪变性（非酒精性脂肪性肝炎，NASH）。目前推荐的 NAFLD 主要治疗方法是通过改变生活方式来减轻体重。实现这一目标的困难导致了药物治疗的强劲发展。虽然新药可能在选定的临床试验人群中显示出疗效，但其实际效果尚不清楚。 TARGET-NASH 是一项针对 NAFLD 患者的为期 5 年的纵向观察性研究，旨在评估临床实践干预措施的有效性并提供注册试验中无法获得的实用信息。 TARGET-NASH 中包含一个生物样本库，用于基因组学和疾病活动生物标志物的转化研究。患者正在代表多个专业的成人和儿科网站注册。所有接受 NAFLD 治疗的患者均符合资格，而其他 NASH 登记或临床试验中的患者将被排除在外。入组患者的年龄范围为 6 岁及以上，并将审查 3 年的临床数据。将评估患者的合并症、合并用药、疾病进展和标签外干预措施，并监测不良结果。确认 NAFLD 干预措施在儿童和成人中的使用、安全性和有效性，以及建立在现实条件下评估疾病进展的实用方法是关键的研究成果。最终，TARGET-NASH 将在学术和社区实践中建立一个大型、多样化的 NAFLD 患者登记册，用于改善健康状况并减少肝硬化和肝细胞癌的发展。
Nonalcoholic fatty liver disease (NAFLD) is highly prevalent and can lead to cirrhosis, hepatocellular carcinoma, and end-stage liver disease. NAFLD comprises the spectrum from simple steatosis (nonalcoholic fatty liver, NAFL), to steatosis with inflammation (nonalcoholic steatohepatitis, NASH). Current primary therapy recommended for NAFLD is weight loss induced by lifestyle modification. The difficulty in achieving this has led to robust pharmacological therapy development. While new drugs may show efficacy in selected phase clinical trial populations, their real-world effectiveness is unknown. TARGET-NASH is a 5-year, longitudinal, observational study of patients with NAFLD designed to evaluate the effectiveness of clinical practice interventions and provide practical information unobtainable in registration trials. A biological specimen repository is included in TARGET-NASH for translational studies of genomics and biomarkers of disease activity. Patients are enrolling at adult and pediatric sites representing multiple specialties. All patients being managed for NAFLD are eligible, whereas those in other NASH registries or clinical trials will be excluded. Enrolled patients range in age from 6 and up and will have 3 years of clinical data reviewed. Patient comorbidities, concomitant medications, disease progression and off-label interventions will be assessed, and adverse outcomes, monitored. Confirming the use, safety and effectiveness of NAFLD interventions in children and adults and establishing pragmatic methods of assessing disease progression under real-world conditions are key study outcomes. Ultimately, TARGET-NASH will establish a large, diverse registry of NAFLD patients at academic and community practices to be leveraged to improve health and reduce development of cirrhosis and hepatocellular carcinoma.