Oral Azacitidine as Maintenance Therapy for Acute Myeloid Leukemia
Posted: Friday, January 8, 2021
Acute myeloid leukemia (AML) often affects older people, and although many of these patients achieve a complete remission with standard induction chemotherapy, a majority of them will relapse. Gail J. Roboz, MD, of NewYork-Presbyterian Hospital, and international colleagues conducted the QUAZAR AML-001 trial to evaluate oral azacitidine (CC-486) as maintenance therapy for individuals in remission from AML. Published in The New England Journal of Medicine, the findings revealed a 30% improvement in survival with this hypomethylating agent compared with placebo.
“After intensive chemotherapy, the risk of AML relapse is high. Many older patients are not eligible to receive a stem cell transplant, and so a less toxic option to reduce disease recurrence is desirable...,” stated coauthor Andrew H. Wei, MB, BS, PhD, of Monash University’s Australian Centre for Blood Diseases, in a press release. “Based on the results of the QUAZAR study, it is very exciting to think that, by taking a tablet that is relatively well tolerated, we can help reduce relapse risk and improve survival.”
This phase III, double-blind trial enrolled a total of 472 patients 55 years of age or older who were in remission and were not candidates to receive hematopoietic stem cell transplantation. Patients were randomly assigned to receive either 300 mg of CC-486 or a placebo once a day for 14 days of each 28-day cycle.
The median age of participants was 68 years old. The median overall survival and relapse-free survival were longer with CC-486 (24.7 months and 10.2 months) than with the placebo (14.8 months and 4.8 months).
Grade 1 or 2 gastrointestinal events were the most common adverse events. Grade 3 or 4 neutropenia and thrombocytopenia affected 41% and 22% of patients receiving CC-486 and 24% and 21% receiving the placebo, respectively. Overall, the investigators reported that the treatment retained health-related quality of life.
Disclosure: For full disclosures of study authors, visit nejm.org.