Table 7.

Outcome measures for clinical trials in AML

Category	Definition
Overall survival	Defined for all patients of a trial; measured from the date of entry into a clinical trial or from the date of diagnosis (eg, for correlative science studies) to the date of death from any cause; patients not known to have died at last follow-up are censored on the date they were last known to be alive
Relapse-free survival (RFS)*,†	Defined only for patients achieving CR, or CRi; measured from the date of achievement of a remission until the date of relapse or death from any cause; patients not known to have relapsed or died at last follow-up are censored on the date they were last examined
Event-free survival (EFS)†	Defined for all patients of a trial; measured from the date of entry into a study to the date of primary refractory disease, or relapse from CR, or CRi, or death from any cause; patients not known to have any of these events are censored on the date they were last examined
Cumulative incidence of relapse (CIR)†,‡	Defined for all patients achieving CR, CRi; measured from the date of achievement of a remission until the date of relapse; patients not known to have relapsed are censored on the date they were last examined; patients who died without relapse are counted as a competing cause of failure

CID, cumulative incidence of death; CIR, cumulative incidence of relapse.

^*RFS and disease-free survival have been used with the same definition.

^†In clinical trials in which the response criterion CR_MRD− is used, consideration should be given to include molecular relapse as assessed by RT-qPCR or MFC as a criterion for relapse; similarly, for analysis of EFS, no achievement of CR_MRD− may be regarded as an event. The definitions of RFS, EFS, and CIR must be clearly defined within each protocol.

^‡It is important to provide estimates of CID as well because just considering the results of CIR may be misleading if, for instance, CIR is lower for 1 group but CID is actually higher for that same group.