英国基于医疗保健系统数据识别脑出血性卒中后心血管结局的准确性。

Accuracy of healthcare systems data for identifying cardiovascular outcomes after stroke due to intracerebral haemorrhage in the United Kingdom.

机构信息

Centre for Clinical Brain Sciences, The University of Edinburgh, Edinburgh, UK.

The Usher Institute, The University of Edinburgh, Edinburgh, UK.

出版信息

Trials. 2024 Nov 16;25(1):774. doi: 10.1186/s13063-024-08631-7.

DOI:10.1186/s13063-024-08631-7

PMID:39550597

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11568574/

Abstract

BACKGROUND

Healthcare systems data (HCSD) could improve the efficiency of clinical trials, but their accuracy and validity are uncertain. Our objective was to assess the accuracy of HCSD as the sole method of outcome detection in the REstart or STop Antithrombotics Randomised Trial (RESTART; ISRCTN71907627) compared with adjudicated questionnaire follow-up and compare estimates of treatment effect.

METHODS

RESTART was a prospective, open, assessor-blind, parallel-group randomised controlled trial (RCT) of antiplatelet therapy after intracerebral haemorrhage (ICH) in the UK. We included 496 (92%) of 537 RESTART participants, who were resident in England or Scotland at randomisation. Computerised randomisation incorporating minimisation allocated participants (1:1) to start or avoid antiplatelet therapy. RESTART used annual questionnaires to detect its primary outcome (recurrent ICH) and secondary outcome (a composite of haemorrhagic or ischemic major adverse cardiovascular events [MACE]) over a median of 2.0 years; an independent adjudication committee verified outcomes using medical records and brain imaging. We obtained ICD10-coded HCSD on hospital admissions and deaths in England and Scotland to identify primary and secondary outcomes. We compared HCSD with a reference standard of adjudicated outcomes. We estimated the effects of antiplatelet therapy using HCSD alone in a Cox proportional hazards model adjusted for minimisation variables.

RESULTS

In the original RESTART trial, 31 people experienced a primary outcome event. HCSD had sensitivity of 84% (95% CI 66 to 95%) and positive predictive value of 68% (51 to 82%) for recurrent ICH. HCSD estimated an effect of antiplatelet therapy (adjusted hazard ratio [aHR] 0.51, 95% CI 0.27 to 0.98; p = 0.044) that was almost identical to adjudicated outcomes (aHR 0.51, 95% CI 0.25 to 1.03; p = 0.060). HCSD had sensitivity of 84% (76 to 91%) and positive predictive value of 78% (69 to 85%) for MACE, on which HCSD estimated an effect of antiplatelet therapy (aHR 0.81, 95% CI 0.56 to 1.16; p = 0.247) that was similar to adjudicated outcomes (aHR 0.65, 95% CI 0.44 to 0.95; p = 0.025).

CONCLUSIONS

In a RCT of antiplatelet therapy for people with ICH, HCSD was reasonably accurate and provided similar estimates of treatment effect compared with adjudicated outcomes.

TRIAL REGISTRATION

ISRCTN71907627 . Registered on 25 April 2013.

摘要

背景

医疗保健系统数据（HCSD）可以提高临床试验的效率，但它们的准确性和有效性尚不确定。我们的目的是评估 HCSD 作为唯一的结果检测方法在 REstart 或 STop 抗血栓治疗随机试验（RESTART；ISRCTN71907627）中的准确性，与裁定问卷随访进行比较，并比较治疗效果的估计值。

方法

RESTART 是一项在英国进行的前瞻性、开放、评估者盲法、平行组随机对照试验（RCT），研究对象为脑出血（ICH）后的抗血小板治疗。我们纳入了 496 名（92%）随机分组时居住在英格兰或苏格兰的 537 名 RESTART 参与者。包含最小化的计算机化随机化将参与者（1:1）分配为开始或避免抗血小板治疗。RESTART 使用年度问卷来检测其主要结局（复发性 ICH）和次要结局（出血或缺血性主要不良心血管事件[MACE]的复合结局），中位随访时间为 2.0 年；一个独立的裁决委员会使用病历和脑成像来验证结局。我们在英格兰和苏格兰获得了 ICD10 编码的 HCSD 以识别主要和次要结局的住院和死亡情况。我们将 HCSD 与裁定结果的参考标准进行了比较。我们使用 Cox 比例风险模型估计了 HCSD 单独使用抗血小板治疗的效果，该模型调整了最小化变量。

结果

在原始的 RESTART 试验中，有 31 人发生了主要结局事件。HCSD 对复发性 ICH 的敏感性为 84%（95%CI 66 至 95%），阳性预测值为 68%（51 至 82%）。HCSD 估计抗血小板治疗的效果（调整后的危险比[aHR]0.51，95%CI 0.27 至 0.98；p=0.044）与裁定结果几乎相同（aHR 0.51，95%CI 0.25 至 1.03；p=0.060）。HCSD 对 MACE 的敏感性为 84%（76 至 91%），阳性预测值为 78%（69 至 85%），HCSD 估计抗血小板治疗的效果（aHR 0.81，95%CI 0.56 至 1.16；p=0.247）与裁定结果相似（aHR 0.65，95%CI 0.44 至 0.95；p=0.025）。