Dataset for Hepmarc: a 96 week randomised controlled feasibility trial of add-on maraviroc in people with HIV and non-alcoholic fatty liver disease

Data for paper published in PLOS ONE 14.07.2023 These files were used for the statistical analysis of the <em>hemparc feasibility trial</em> using Stata software verson 17, and are as follows, both Stata format and .csv format as appropriate. The .do file is a simple text file. <br> <strong>hepmarc_data </strong>minimum dataset: .csv, .dta: See doi:10.1136/bmjopen-2019-035596 for study protocol describing all data collected <strong>hepmarc Data dictionary </strong>.xls, .dta; description of each data fields in minimum dataset <strong>hepmarc AE listing</strong>: Adverse events listing .csv, .dta <strong>hepmarc SAP v1.0 240322_</strong>.xls .dta; description of each data fields in minimum dataset <strong>hepmarc data.do </strong>Stata .do file used to perform the analysis <br> Notes: Each particpant's age has been altered by a random amount to preserve anonymity. There are two rows for two of the participants who each reported two adverse reactions. <br> <strong>Abstract</strong> <strong>Objectives</strong> Maraviroc may reduce hepatic inflammation in people with HIV and non-alcoholic fatty liver disease (HIV-NAFLD) through CCR5-receptor antagonism, which warrants further exploration. <br> <strong>Methods</strong> We performed an open-label 96-week randomised-controlled feasibility trial of maraviroc plus optimised background therapy (OBT) versus OBT alone, in a 1:1 ratio, for people with virologically-suppressed HIV-1 and NAFLD without cirrhosis. Dosing followed recommendations for HIV therapy in the Summary of Product Characteristics for maraviroc. The primary outcomes were safety, recruitment and retention rates, adherence and data completeness. Secondary outcomes included the change in Fibroscan-assessed liver stiffness measurements (LSM), controlled attenuation parameter (CAP) and Enhanced Liver Fibrosis (ELF) scores. <br> <strong>Results</strong> Fifty-three participants (53/60, 88% of target) were recruited; 23 received maraviroc plus OBT; 89% were male; 19% had type 2 diabetes mellitus. The median baseline LSM, CAP &amp; ELF scores were 6.2 (IQR 4.6-7.8) kPa, 325 (IQR 279-351) dB/m and 9.1 (IQR 8.6-9.6) respectively. <br> Primary outcomes: all individuals eligible after screening were randomised; there was 92% (SD 6.6%) adherence to maraviroc [target &gt;90%]; 83% (95%CI 70%-92%) participant retention [target &gt;65%]; 5.5% of data were missing [target &lt;20%]. There were noo Serious Adverse Reactions ; mild-moderate intensity Adverse Reactions were reported by five participants (5/23, 22% (95%CI 5%-49%)) [target &lt;10%]. All Adverse Reactionss resolved. Secondary outcomes: no important differences were seen by treatment group for the change from baseline in LSM, CAP or ELF scores <br> <strong>Conclusions</strong> This feasibility study provides preliminary evidence of maraviroc safety amongst people with HIV-NAFLD, and acceptable recruitment, retention, and adherence rates. These data support a definitive randomised-controlled trial assessing maraviroc impact on hepatic steatosis and fibrosis. <br> Clinical trial registry: ISCRTN, registration number 31461655.

本数据集对应2023年7月14日发表于《PLOS ONE》的论文，所用文件均用于采用Stata 17版本软件开展的**hemparc可行性试验（hemparc feasibility trial）**的统计分析，涵盖Stata格式与.csv格式两类文件，具体如下：其中.do文件为纯文本文件。

**hepmarc_data**：最小数据集，包含.csv、.dta格式文件；研究方案中详述了所有采集的数据字段，详见DOI:10.1136/bmjopen-2019-035596。
**hepmarc Data dictionary**：包含.xls、.dta格式文件，用于说明最小数据集内各数据字段的含义。
**hepmarc AE listing**：不良事件清单，包含.csv、.dta格式文件。
**hepmarc SAP v1.0 240322_**：包含.xls、.dta格式文件，用于说明最小数据集内各数据字段的含义。
**hepmarc data.do**：用于执行统计分析的Stata .do脚本文件。

注：为保护受试者匿名性，所有参与者的年龄均经随机数值扰动处理；其中2名分别报告2种不良反应的受试者各对应2行数据。

**摘要**
**研究目标**：马拉韦罗（Maraviroc）可通过CCR5受体拮抗作用（CCR5-receptor antagonism），改善HIV合并非酒精性脂肪性肝病（non-alcoholic fatty liver disease, NAFLD）患者的肝脏炎症，该作用有待进一步探索。
**研究方法**：本研究针对病毒学抑制的HIV-1合并非肝硬化非酒精性脂肪性肝病患者，开展一项开放标签、96周、1:1随机对照可行性试验，对比马拉韦罗联合优化背景治疗（optimised background therapy, OBT）与单纯优化背景治疗的效果。给药方案遵循马拉韦罗产品说明书中针对HIV治疗的推荐剂量。主要结局指标为安全性、招募率、留存率、用药依从性及数据完整性；次要结局指标包括瞬时弹性成像（Fibroscan）评估的肝脏硬度测量值（liver stiffness measurements, LSM）、受控衰减参数（controlled attenuation parameter, CAP）及增强肝纤维化（Enhanced Liver Fibrosis, ELF）评分的变化情况。
**研究结果**：共招募53名受试者（占目标招募人数的88%，即53/60）；其中23名接受马拉韦罗联合OBT治疗；受试者中89%为男性，19%合并2型糖尿病。基线时受试者的LSM中位数为6.2（四分位距4.6~7.8）kPa，CAP中位数为325（四分位距279~351）dB/m，ELF评分中位数为9.1（四分位距8.6~9.6）。
主要结局指标：所有经筛查符合条件的受试者均完成随机分组；马拉韦罗用药依从率为92%（标准差6.6%），预设目标为>90%；受试者留存率为83%（95%置信区间70%~92%），预设目标为>65%；数据缺失率为5.5%，预设目标为<20%。未报告严重不良反应；5名受试者（5/23，22%，95%置信区间5%~49%）报告轻至中度不良反应，预设目标为<10%；所有不良反应均已缓解。
次要结局指标：不同治疗组间，LSM、CAP或ELF评分较基线的变化均无显著差异。
**研究结论**：本可行性研究为HIV-NAFLD患者使用马拉韦罗的安全性提供了初步证据，且招募、留存及用药依从率均符合预期。本研究数据支持开展旨在评估马拉韦罗对肝脏脂肪变性及纤维化影响的确证性随机对照试验。
临床试验注册信息：国际标准随机对照试验注册库（ISCRTN），注册号31461655。