Utility of routinely collected electronic health records data to support effectiveness evaluations in inflammatory bowel disease: a pilot study of tofacitinib.
Utility of routinely collected electronic health records data to support effectiveness evaluations in inflammatory bowel disease: a pilot study of tofacitinib.
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DOI：
10.1136/bmjhci-2021-100337
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发表时间：
2021-05
影响因子：
4.1
通讯作者：
Butte AJ
中科院分区：
文献类型：
--
作者：
Rudrapatna VA;Glicksberg BS;Butte AJ
电子健康记录（EHR）作为现实世界证据的潜在来源，正受到监管机构、生物制药公司和支付者越来越多的关注。然而，它们是否适合研究具有复杂活动措施的疾病尚不清楚。我们试图评估使用EHR数据估计炎症性肠病（IBD）的治疗效果，使用托法替尼作为用例。查询了加州大学旧金山分校（2012年6月至2019年4月）的记录，以识别接受托法替尼治疗的IBD患者。弗朗西斯科。根据预先登记的方案手动提取基线和随访时的疾病活动变量。评估了符合近期溃疡性结肠炎（UC）和克罗恩病（CD）随机试验终点的患者比例。86例患者开始接受托法替尼治疗。真实世界和试验队列的基线特征相似，除了前者肿瘤坏死因子抑制剂的普遍失败。54%（UC）和62%（CD）的患者在基线（第-6至0个月）时完全捕获疾病活动，而只有32%（UC）和69%（CD）的患者有完整的随访数据（第2至8个月）。使用数据插补，我们估计达到试验主要终点的比例与UC（16%，p值=0.5）和CD（38%，p值=0.8）的已发表估计值相似。尽管托法替布用于不同队列，但该初步研究重现了基于试验的托法替布疗效估计值，但发现常规收集的数据存在大量缺失。未来的工作需要加强EHR数据，并在IBD等复杂疾病中提供真实证据。
Electronic health records (EHR) are receiving growing attention from regulators, biopharmaceuticals and payors as a potential source of real-world evidence. However, their suitability for the study of diseases with complex activity measures is unclear. We sought to evaluate the use of EHR data for estimating treatment effectiveness in inflammatory bowel disease (IBD), using tofacitinib as a use case. Records from the University of California, San Francisco (6/2012 to 4/2019) were queried to identify tofacitinib-treated IBD patients. Disease activity variables at baseline and follow-up were manually abstracted according to a preregistered protocol. The proportion of patients meeting the endpoints of recent randomised trials in ulcerative colitis (UC) and Crohn’s disease (CD) was assessed. 86 patients initiated tofacitinib. Baseline characteristics of the real-world and trial cohorts were similar, except for universal failure of tumour necrosis factor inhibitors in the former. 54% (UC) and 62% (CD) of patients had complete capture of disease activity at baseline (month −6 to 0), while only 32% (UC) and 69% (CD) of patients had complete follow-up data (month 2 to 8). Using data imputation, we estimated the proportion achieving the trial primary endpoints as being similar to the published estimates for both UC (16%, p value=0.5) and CD (38%, p-value=0.8). This pilot study reproduced trial-based estimates of tofacitinib efficacy despite its use in a different cohort but revealed substantial missingness in routinely collected data. Future work is needed to strengthen EHR data and enable real-world evidence in complex diseases like IBD.
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影响因子：
158.5
作者：
Feagan, B. G.;Sandborn, W. J.;Rutgeerts, P.
通讯作者：
Rutgeerts, P.
影响因子：
12.6
作者：
Ha, Christina;Ullman, Thomas A.;Kornbluth, Asher
通讯作者：
Kornbluth, Asher
影响因子：
6.1
作者：
Zoccali, Carmine;Blankestijn, Peter J.;Wanner, Christoph
通讯作者：
Wanner, Christoph
影响因子：
24.5
作者：
Panés J;Sandborn WJ;Schreiber S;Sands BE;Vermeire S;D'Haens G;Panaccione R;Higgins PDR;Colombel JF;Feagan BG;Chan G;Moscariello M;Wang W;Niezychowski W;Marren A;Healey P;Maller E
通讯作者：
Maller E
影响因子：
13.8
作者：
Norgeot, Beau;Glicksberg, Benjamin S.;Butte, Atul J.
通讯作者：
Butte, Atul J.