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Rebecca Olin, MD, MS


TCT 2021: Effects of HSCT After Azacitidine Therapy for AML

By: Anna Nowogrodzki
Posted: Friday, February 19, 2021

The overall survival benefit of oral azacitidine therapy for patients with acute myeloid leukemia (AML) did not appear to change whether patients who later received hematopoietic stem cell transplantation (HSCT) were removed from the analysis or not, according to further analysis of data from the phase III QUAZAR AML-001 trial. Farhad Ravandi, MD, of MD Anderson Cancer Center, Houston, and colleagues presented their results at the 2021 Transplantation & Cellular Therapy Meetings (Abstract 140).

“These data show oral azacitidine maintenance did not prevent some patients from receiving subsequent hematopoietic stem cell transplant,” the authors wrote. Of note, “the use of oral azacitidine to ‘bridge’ patients to transplant should be considered experimental; further research is needed to clarify the risk/benefit profile.”

This study included 472 patients, aged 55 or older, with AML who were randomly assigned to receive either a placebo or 300 mg of oral azacitidine. None of the patients were eligible for HSCT at enrollment, but 47 received a transplant after the study treatment. Six patients in the azacitidine arm discontinued study treatment while in first remission to receive a stem cell transplant. The other 41 patients (9 in the azacitidine arm, 32 in the placebo arm) received a stem cell transplant after AML relapse. The trial was not designed to compare patients who did and did not receive stem cell transplants.

Median overall survival was functionally the same, regardless of whether the 47 patients receiving stem cell transplants were censored or not. With all 472 patients included, median overall survival was 24.7 months with azacitidine versus 14.8 months with a placebo (hazard ratio = 0.69). With the patients who received stem cell transplant censored, median overall survival was 24.8 months with azacitidine versus 14.8 months with a placebo (hazard ratio = 0.67).

The phase III AMADEUS trial is currently testing oral azacitidine as maintenance therapy after HSCT. 

Disclosure: The study authors’ disclosures may be found at

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