Table 4.
Treatment response and secondary end points
| Azacitidine (n = 241) | CCR (n = 247) | HR | 95% CI | P | |||
|---|---|---|---|---|---|---|---|
| No. | % | No. | % | ||||
| Hematologic response* | |||||||
| CR + CRi | 67 | 27.8 | 62 | 25.1 | .5384 | ||
| CR | 47 | 19.5 | 54 | 21.9 | .5766 | ||
| CRi | 20 | 8.3 | 8 | 3.2 | .0191 | ||
| CRc-20 | 5 | 2.1 | 14 | 5.7 | .0589 | ||
| PR | 3 | 1.2 | 3 | 1.2 | 1.0 | ||
| Stable disease | 71 | 29.5 | 59 | 23.9 | .1833 | ||
| Progressive disease | 20 | 8.3 | 20 | 8.1 | 1.0 | ||
| Early death | 18 | 7.5 | 29 | 11.7 | — | ||
| No confirmed AML† | 18 | 7.5 | 12 | 4.9 | — | ||
| Not assessable‡ | 44 | 18.3 | 62 | 25.1 | — | ||
| Other secondary end points | |||||||
| EFS, mo§ | 0.87 | 0.72-1.05 | .1495 | ||||
| Median | 6.7 | 4.8 | |||||
| Min-max | 5.0-8.8 | 3.8-6.0 | |||||
| RFS, mo | 1.11 | 0.75-1.66 | .5832 | ||||
| Median | 9.3 | 10.5 | |||||
| Min-max | 6.7-12.4 | 7.3-12.3 | |||||
| Relapse after CR or CRi | 43 | 64.2 | 35 | 56.5 | .4712 | ||
| Duration of CR or CRi, mo | — | ||||||
| Median | 10.4 | 12.3 | |||||
| Min-max | 7.2-15.2 | 9.0-17.0 | |||||
| Transfusion independence|| | |||||||
| RBC | 65 | 38.5 | 45 | 27.6 | |||
| Platelets | 41 | 40.6 | 24 | 29.3 | |||
CRc-20, cytogenetic complete remission in at least 20 metaphases; EFS, event-free survival; RFS, relapse-free survival.
Hematologic response was defined by International Working Group criteria18 and was adjudicated by an independent review committee. Stable disease was defined as a clinical state that did not meet criteria for any other treatment response (eg, CR, CRi, disease progression, or treatment failure).
Patients included on the basis of local review had <30% BM blasts at central review.
Patients were missing BM aspirate or hematology assessment.
Events included treatment failure, progressive disease, relapse after CR or CRi, or death.
No transfusions for 56 consecutive days on study for patients who were transfusion dependent at baseline (≥1 transfusion in the 56 days before study start).