Acute gastrointestinal bleeding: proposed study outcomes for new randomised controlled trials

Area of Science:

• Gastroenterology
• Clinical Trials Methodology
• Health Outcomes Research

Background:

• Acute gastrointestinal bleeding (GIB) is a frequent cause of hospitalization.
• Inconsistent study methodologies hinder interpretation and comparison of GIB research.
• Standardized outcome measures are needed for robust GIB clinical trials.

Purpose of the Study:

• To recommend outcome measures for future randomized controlled trials (RCTs) in acute GIB.
• To propose novel RCTs for acute GIB to inform funding opportunities.
• To develop a clinically meaningful primary endpoint for acute GIB research.

Main Methods:

• Experts critically reviewed existing evidence on GIB outcomes.
• A National Institutes of Health conference focused on hemostatic outcomes in clinical trials.
• The panel aimed to propose a bleeding outcome applicable across disciplines and develop a specific GIB endpoint.

Main Results:

• A composite primary endpoint was proposed: further bleeding within 30 days requiring red blood cell transfusion, urgent intervention, or death.
• Secondary outcomes include individual components of the primary endpoint, hospitalization duration, adverse events, and healthcare resource use.
• This endpoint aims to standardize outcome assessment in acute GIB.

Conclusions:

• The proposed endpoint can advance GIB research by standardizing focus on patient-relevant outcomes.
• It will aid in designing future RCTs and determining appropriate sample sizes for GIB studies.
• Adoption of this endpoint can lead to more comparable and impactful research in acute GIB.