Definitions of endpoints for clinical trials in MDS
| Endpoint . | Response category . | Definition . | Point of measurement . |
|---|---|---|---|
| Overall survival | All patients | Death from any cause | Entry into trial |
| Event-free survival | All patients* | Failure or death from any cause | Entry into trial |
| Progression-free survival | All patients | Disease progression or death from MDS | Entry into trial |
| Disease-free survival | CR | Time to relapse | First documentation |
| Cause-specific death | All patients | Death related to MDS | Death |
| Endpoint . | Response category . | Definition . | Point of measurement . |
|---|---|---|---|
| Overall survival | All patients | Death from any cause | Entry into trial |
| Event-free survival | All patients* | Failure or death from any cause | Entry into trial |
| Progression-free survival | All patients | Disease progression or death from MDS | Entry into trial |
| Disease-free survival | CR | Time to relapse | First documentation |
| Cause-specific death | All patients | Death related to MDS | Death |
IPSS should be used as the primary stratification.
Complete blood counts should be evaluated at least monthly, or more often if clinically indicated, to establish the durability of responses.
Under circumstances in which presentation of event-free survival may be appropriate for responders only, this point should be clearly stated.