Endpoints for clinical trials testing treatment of acute graft-versus-host disease: a joint statement

Area of Science:

• Hematology
• Oncology
• Clinical Trial Design

Background:

• No FDA-approved agents currently exist for acute graft-versus-host disease (aGVHD) prevention or treatment.
• Lack of established precedents for comparative efficacy and safety assessments of investigational aGVHD agents.
• Need for standardized outcome measures in clinical trials for aGVHD.

Purpose of the Study:

• To reach expert consensus on terminology for a clinically meaningful primary endpoint in aGVHD treatment studies.
• To propose 'very good partial response' (VGPR) as a standardized primary endpoint for aGVHD clinical trials.

Main Methods:

• Convened a panel of experts who met on two occasions.
• Reached consensus on recommendations for terminology describing a clinically meaningful primary endpoint.
• Defined 'very good partial response' (VGPR) incorporating both diagnostic and functional criteria.

Main Results:

• Recommended terminology for 'very good partial response' (VGPR) as a primary endpoint for aGVHD studies.
• Hypothesized that the harm of overtreatment outweighs undertreatment, supporting VGPR as a balanced endpoint.
• Acknowledged VGPR's provisional status, requiring validation through clinical trial experience.

Conclusions:

• Proposed VGPR, with defined criteria, as a provisional primary endpoint for aGVHD treatment trials.
• Emphasized that VGPR must be evaluated alongside survival and safety data.
• Highlighted the need for validation of VGPR through practical application in clinical research.