Related Experiment Videos

Weighting composite endpoints in clinical trials: essential evidence for the heart team

Betty C Tong1, Joel C Huber, Deborah D Ascheim

  • 1Division of Thoracic and Cardiovascular Surgery, Duke University Medical Center, Durham, North Carolina 27710, USA. betty.tong@duke.edu

The Annals of Thoracic Surgery
|July 17, 2012
PubMed

Insights

Patients value different outcomes in coronary revascularization trials differently. Risk of death, stroke, and longevity are prioritized over repeat procedures, suggesting a need for weighted composite endpoints in trial analysis.

Area of Science:

  • Cardiovascular Medicine
  • Clinical Trial Design
  • Health Economics

Background:

  • Coronary revascularization trials commonly use composite endpoints for major adverse cardiac and cerebrovascular events (MACCE).
  • Current analysis methods assign equal importance to all MACCE elements, potentially introducing bias.
  • Patient perspectives on the relative importance of MACCE components are not well-established.

Purpose of the Study:

  • To determine the relative importance of individual Major Adverse Cardiac and Cerebrovascular Events (MACCE) elements from a patient's viewpoint.
  • To assess the impact of patient-weighted endpoints on the interpretation of coronary revascularization trial results.
  • To investigate patient bias related to procedural labeling in revascularization decisions.

Main Methods:

  • A discrete choice experiment was conducted with 224 survey respondents.
  • Respondents evaluated hypothetical scenarios based on the Synergy Between Percutaneous Coronary Intervention With Taxus and Cardiac Surgery (SYNTAX) trial criteria.
  • Conjoint analysis was used to derive relative weights for MACCE, longevity, and recovery time.

Main Results:

  • Patient-weighted importance: death (0.23), stroke (0.18), longevity/recovery (0.17), myocardial infarction (0.14), repeat revascularization (0.11).
  • Weighted analysis showed a decreased, but persistent, MACCE advantage for coronary artery bypass graft surgery over percutaneous coronary intervention.
  • Procedural labels significantly influenced patient choice (87% preferred bypass surgery when unlabeled, 73% when labeled).

Conclusions:

  • Major Adverse Cardiac and Cerebrovascular Events (MACCE) elements hold unequal weight for patients.
  • Incorporating patient-derived weights enhances the validity of composite endpoints in clinical trials.
  • Labeling of procedures introduces bias, affecting patient decision-making in coronary revascularization.
Abstract

Related Concept Videos

Clinical Trials: Overview01:11

Clinical Trials: Overview

Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
Clinical Trials01:16

Clinical Trials

Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches01:23

Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches

Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast, controlled...
Heart Failure V: Medical Management01:30

Heart Failure V: Medical Management

Medical Management of Acute Decompensated Heart Failure (ADHF)The primary goals of therapy for patients hospitalized with acute decompensated heart failure (ADHF) include:Relieving symptomsOptimizing volume statusSupporting oxygenation and ventilationMaintaining cardiac output (CO) and end-organ perfusionIdentifying and addressing the cause of ADHFPreventing complicationsProviding patient education on factors precipitating HF exacerbationPlanning for dischargeOngoing monitoring and assessment...
Blood Studies for Cardiovascular System I: Cardiac Biomarkers01:20

Blood Studies for Cardiovascular System I: Cardiac Biomarkers

Cardiac biomarkers are enzymes, proteins, and hormones released into the blood when cardiac cells are injured. They are powerful tools for triaging.
The essential diagnostic tools for detecting myocardial necrosis and monitoring individuals suspected of having acute coronary syndrome (ACS) include:
Troponins
Troponins, particularly cardiac troponins I and T, are the most precise and sensitive markers of myocardial injury. They are detectable within 4-6 hours of myocardial injury and remain...
Acute Coronary Syndrome III: Diagnostic Studies01:30

Acute Coronary Syndrome III: Diagnostic Studies

Diagnosing acute coronary syndrome or ACS begins with a thorough patient history. Notable symptoms include central, crushing chest pain radiating to the left arm, neck, jaw, or back, along with shortness of breath, sweating (diaphoresis), nausea, vomiting, dizziness, and palpitations.It is crucial to note any history of cardiac illnesses and assess risk factors, including age, gender, smoking, hypertension, diabetes, hyperlipidemia, and a sedentary lifestyle.During physical examination, vital...