A phase 2 trial of azacitidine and gemtuzumab ozogamicin therapy in older patients with acute myeloid leukemia

Abstract

This trial tested the safety and efficacy of a regimen consisting of hydroxyurea followed by azacitidine, 75 mg/m(2) for 7 days, and gemtuzumab ozogamicin, 3 mg/m(2) on day 8, in older patients with newly diagnosed acute myeloid leukemia. Those achieving a complete remission received 1 consolidation treatment followed by 4 cycles of azacitidine. The patients were stratified into good-risk (age 60-69 years or performance status 0-1) and poor-risk (age ≥70 years and performance status 2 or 3) groups. Specific efficacy and safety goals were defined as being supportive of further study of the regimen. Eighty-three patients were registered in the good-risk cohort and 59 in poor-risk cohort, with median age of 71 and 75 years, respectively. In the good-risk group, 35 patients (44%) achieved a complete remission. Median relapse-free and overall survivals were 8 and 11 months, respectively. Six patients (8%) died within 30 days of registration. In the poor-risk group, 19 (35%) achieved a complete remission. Median relapse-free and overall survivals were 7 and 11 months, respectively. Seven patients (14%) died early. The results of this trial met predefined goals for efficacy and safety for the poor-risk cohort but not the good-risk group. .

Trial registration: ClinicalTrials.gov NCT00658814.

Figure 1

Overall survival, relapse-free survival, and landmark overall survival in good-risk patients. (A) Good-risk cohort, overall survival (n = 79). (B) Good-risk cohort, relapse-free survival (n = 35). (C) Good-risk cohort, landmark survival.

Figure 2

Overall survival, relapse-free survival, and landmark overall survival in poor-risk patients. (A) Poor-risk cohort, overall survival (n = 54). (B) Poor-risk cohort, relapse-free survival (n = 19). (C) Poor-risk cohort, landmark survival.

Figure 3

Relapse-free and overall survival stratified by CR vs CRi for each risk cohort. (A) Good-risk cohort, overall survival after response. (B) Good-risk cohort, relapse-free survival. (C) Poor-risk cohort, overall survival after response. (D) Poor-risk cohort, relapse-free survival.

Figure 4

Histogram of time to best response, calculated from the start of induction treatment to the date of bone marrow examination documenting the best response (CR or CRi).