Table 4.

Strengths and limitations of clinical investigation supporting recent FDA-approved agents for GVHD.

Strengths

• Strong pre-clinical data demonstrating biological rationale in GVHD

• Investigation of agents with different mechanisms of actions

• Multicenter prospective clinical trials, including 2 international studies

• Use of validated diagnostic criteria and response measures in clinical trials

Limitations

• Limited use of randomized, phase III design

• No head-to-head comparison of single agents

• Some trials with small to moderate sample size

• Scarce real-world experience reported to date