Outcome measures for clinical trials in acute myeloid leukemia
| Category . | Definition . |
|---|---|
| Early death | Death from any cause within a timeframe relevant for the therapy being investigated (eg, 30 and 60 d from commencing therapy) |
| Overall survival | Defined for all patients in a trial; measured from day 1 of randomization or day 1 of registration in non-randomized trials (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 |
| Event-free survival (EFS) | Defined for all patients in a trial; measured from day 1 of randomization or day 1 of registration in non-randomized trials to the date of treatment failure, hematologic relapse from CR/CRh/CRi or death from any cause, whichever occurs first; treatment failure is defined as not achieving either CR, CRh or CRi by a pre-defined landmark (eg, after two cycles of intensive chemotherapy or 180 d for non-intensive therapy); patients evaluable for response but not achieving either CR, CRh or CRi by the defined landmark and patients who die before the defined landmark without response assessments are considered an event at day 1 of randomization; patients alive who are non-evaluable for response should be censored at day 1 of the randomization; patients achieving either CR, CRh or CRi by the defined landmark but do not relapse or die should be censored on the date they were last assessed for response |
| Relapse-free survival (RFS)* | Defined only for patients achieving CR, CRh, or CRi; measured from the date of achievement of remission until the date of hematologic 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 known to be alive |
| Cumulative incidence of relapse (CIR) | Defined for all patients achieving CR, CRh, CRi; measured from the date of achievement of a remission until the date of hematologic relapse; patients not known to have relapsed are censored on the date they were last assessed for response; patients who died without relapse are counted as a competing cause of failure |
| Cumulative incidence of death (CID) | Defined for all patients achieving CR, CRh, CRi; measured from the date of achievement of a remission to death without prior relapse; relapse is considered as competing risk |
| If including assessment of MRD relapse | |
| EFSMRD† | Measured from day 1 of randomization or day 1 of registration in non-randomized trials to the date of failure to achieve CR, CRh or CRi by a defined landmark (eg, after two cycles of intensive chemotherapy or 180 d for non-intensive therapy), hematologic relapse, MRD relapse (for patients achieving CR, CRh or CRi without MRD) or death from any cause |
| RFSMRD† | Measured from the date of achievement of a remission (CR, CRh, or CRi) until the date of hematologic relapse, MRD relapse, or death from any cause |
| CIRMRD† | Measured from the date of achievement of a remission (CR, CRh or CRi) until the date of hematologic relapse, or molecular MRD relapse; patients who died without relapse are counted as a competing cause of failure |
| CIDMRD | Measured from the date of achievement of a remission (CR, CRh, or CRi) to death without prior relapse; morphologic or molecular MRD relapse is considered as competing risk |
| Category . | Definition . |
|---|---|
| Early death | Death from any cause within a timeframe relevant for the therapy being investigated (eg, 30 and 60 d from commencing therapy) |
| Overall survival | Defined for all patients in a trial; measured from day 1 of randomization or day 1 of registration in non-randomized trials (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 |
| Event-free survival (EFS) | Defined for all patients in a trial; measured from day 1 of randomization or day 1 of registration in non-randomized trials to the date of treatment failure, hematologic relapse from CR/CRh/CRi or death from any cause, whichever occurs first; treatment failure is defined as not achieving either CR, CRh or CRi by a pre-defined landmark (eg, after two cycles of intensive chemotherapy or 180 d for non-intensive therapy); patients evaluable for response but not achieving either CR, CRh or CRi by the defined landmark and patients who die before the defined landmark without response assessments are considered an event at day 1 of randomization; patients alive who are non-evaluable for response should be censored at day 1 of the randomization; patients achieving either CR, CRh or CRi by the defined landmark but do not relapse or die should be censored on the date they were last assessed for response |
| Relapse-free survival (RFS)* | Defined only for patients achieving CR, CRh, or CRi; measured from the date of achievement of remission until the date of hematologic 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 known to be alive |
| Cumulative incidence of relapse (CIR) | Defined for all patients achieving CR, CRh, CRi; measured from the date of achievement of a remission until the date of hematologic relapse; patients not known to have relapsed are censored on the date they were last assessed for response; patients who died without relapse are counted as a competing cause of failure |
| Cumulative incidence of death (CID) | Defined for all patients achieving CR, CRh, CRi; measured from the date of achievement of a remission to death without prior relapse; relapse is considered as competing risk |
| If including assessment of MRD relapse | |
| EFSMRD† | Measured from day 1 of randomization or day 1 of registration in non-randomized trials to the date of failure to achieve CR, CRh or CRi by a defined landmark (eg, after two cycles of intensive chemotherapy or 180 d for non-intensive therapy), hematologic relapse, MRD relapse (for patients achieving CR, CRh or CRi without MRD) or death from any cause |
| RFSMRD† | Measured from the date of achievement of a remission (CR, CRh, or CRi) until the date of hematologic relapse, MRD relapse, or death from any cause |
| CIRMRD† | Measured from the date of achievement of a remission (CR, CRh or CRi) until the date of hematologic relapse, or molecular MRD relapse; patients who died without relapse are counted as a competing cause of failure |
| CIDMRD | Measured from the date of achievement of a remission (CR, CRh, or CRi) to death without prior relapse; morphologic or molecular MRD relapse is considered as competing risk |