Study Finds Oral Azacitidine as AML Maintenance Treatment Prolongs Survival


Patients with AML that has gone into remission following initial chemotherapy were found to remain in remission longer and have improved OS if they took a pill form of azacitidine as a maintenance treatment.

Patients with acute myeloid leukemia (AML) that has gone into remission following initial chemotherapy were found to remain in remission longer and have improved overall survival (OS) if they took a pill form of azacitidine as a maintenance treatment, according to a randomized, international phase 3 clinical trial. Since this is the first time an AML maintenance treatment was found to have such a strong benefit for patients, it has already been adopted in practices as a standard part of care.

Published in December 2020 in the New England Journal of Medicine, the findings led to the FDA approving oral azacitidine (Onureg) in September 2020 as an AML maintenance therapy.

"At last, we have an effective treatment that can be given in the post-remission setting to help keep AML patients in remission and improve their survival," said senior author Gail Roboz, MD, professor of medicine in the Division of Hematology and Medical Oncology; director of the Clinical and Translational Leukemia Program at Weill Cornell Medicine; and a hematologist/oncologist at New York-Presbyterian/Weill Cornell Medical Center, in a press release. "We are especially gratified that the drug is very well-tolerated, so that quality of life is not compromised."

Currently, AML is the most common form of acute leukemia in adults and is a life-threatening hematological cancer. According to the National Cancer Institute, approximately 20,000 people are diagnosed with AML in the United States per year, with 11,000 dying at the same rate.

At the time of diagnosis, patients are usually either middle-aged or older adults. The 5-year OS rate is approximately 30%, with a 10% survival rate for patients older than 65 years of age. There are also frequent relapses among patients with AML, even following complete remission with initial chemotherapy.

The randomized phase 3 clinical trial, known as the QUAZAR AML-001 trial, consisted of 472 patients from 148 medical centers. Researchers investigated whether relapse could be delayed using oral azacitidine as a maintenance therapy.

The results demonstrated that patients on a regimen of 300 mg of oral azacitidine for 2 weeks each month had statistically and clinically significant improvements in relapse-free survival (RFS) and OS. Additionally, the median RFS was 10.2 months and OS was 24.7 months with oral azacitidine, whereas the median RFS was 4.8 and OS was 14.8 months for patients taking the placebo.

At the 2-year follow up, 50.6% of patients taking azacitidine had survived, compared with 37.1% of patients on the placebo.

Azacitidine works principally as a hypomethylating drug, which means that it removes methyl groups from DNA in cells. Through the regulation of gene activity, methyl groups silence nearby genes, which is thought to restore the tumor suppressor activity of genes in order to hinder the proliferation of the cancerous cell.

The adverse effects (AEs) were also found to be moderate and manageable. The AEs of oral azacitidine that were found to be moderately more common in the treatment group included vomiting, low white blood cell counts, and infections.

However, these AEs were found to be manageable and did not result in the discontinuation of treatment. In terms of quality of life, patients in both the treatment and placebo groups scored similarly, showing that oral azacytidine was tolerable.

Notably, some patients taking oral azacytidine as a maintenance therapy have been able to survive for many years, according to Roboz.

"One of my patients on the trial was so critically ill when she was initially diagnosed that she was told to get her affairs in order, but her AML was put into remission, and she has been receiving oral azacitidine maintenance therapy as part of the QUAZAR trial since 2013," Roboz said in the press release.


New maintenance treatment for acute myeloid leukemia prolongs the lives of patients. New York, NY: Weill Cornell Medicine; January 22, 2021. Accessed January 29, 2021.

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