Best Practices in Large Database Clinical Epidemiology Research in Hepatology: Barriers and Opportunities

Abstract

With advances in computing and information technology, large health care research databases are becoming increasingly accessible to investigators across the world. These rich, population-level data sources can serve many purposes, such as to generate "real-world evidence," to enhance disease phenotyping, or to identify unmet clinical needs, among others. This is of particular relevance to the study of patients with end-stage liver disease (ESLD), a socioeconomically and clinically heterogeneous population that is frequently under-represented in clinical trials. This review describes the recommended "best practices" in the execution, reporting, and interpretation of large database clinical epidemiology research in hepatology. The advantages and limitations of selected data sources are reviewed, as well as important concepts on data linkages. The appropriate classification of exposures and outcomes is addressed, and the strategies needed to overcome limitations of the data and minimize bias are explained as they pertain to patients with ESLD and/or liver transplantation (LT) recipients. Lastly, selected statistical concepts are reviewed, from model building to analytic decision making and hypothesis testing. The purpose of this review is to provide the practical insights and knowledge needed to ensure successful and impactful research using large clinical databases in the modern era and advance the study of ESLD and LT.

Recipient and Center Factors Associated With Immunosuppression Practice Beyond the First Year After Liver Transplantation and Impact on Outcomes

“…As such, the linkage of UNOS and other data sources (eg, other registries and healthcare claims data) creates a uniquely detailed resource for pharmacoepidemiology research. 12,36 Importantly, we observed similar trends in the use of individual drugs at 1-y post-LT in our linked cohort as described in OPTN reports. 20,22 In determining the optimal immunosuppression strategy post-LT, data from multiple types of studies should be taken into consideration (eg, clinical trials, meta-analyses and large observational cohorts).…”

Prevalence, mortality, and resource utilization of Staphylococcus aureus bacteremia in liver transplant recipients: A 2012–2016 nationwide analysis

“…Considering that the objective of this study was to frame the economic burden of SAB in LT recipients, the NIS database contains the element most pertinent to the exposures and outcomes considered. 9 A total of 26,415 LT recipients from 2012 to 2016 were identified. The inpatient prevalence of SAB among this LT population was 1.3% (180 MSSA, 160 MRSA).…”

“Beyond MELD” – Emerging strategies and technologies for improving mortality prediction, organ allocation and outcomes in liver transplantation

“…Historically, the predominant forms of large clinical research databases in the United States and Europe have been based on either patient registries, such as the Scientific Registry of Transplant Recipients or Eurotransplant databases, 11,59,60 multicentre curated cohorts, or administrative claims databases. [61][62][63] Beyond these large databases, there has been a growing movement towards aggregation of longitudinal electronic health records (EHRs) across multiple institutions and health systems. [64][65][66] In the United States and the European Union, EHRs now have greater than 96% penetration in acute care hospital and physicians' offices.…”