46.5% of patients achieved a complete response in the ASCERTAIN V clinical trial, which assessed an all-oral combination therapy for older patients with acute myeloid leukemia.
The U.S. Food and Drug Administration (FDA) approved the regimen on May 13 for newly diagnosed adults aged 75 years or older and for adults unable to undergo intensive chemotherapy.
Trial Details
The international phase 1/phase 2 trial enrolled 189 newly diagnosed patients across the United States, Canada, and Spain. The research was led by investigators from Weill Cornell Medicine, NewYork-Presbyterian, Yale University, and MD Anderson Cancer Center.
Treatment Regimen
The oral regimen consists of venetoclax and decitabine-cedazuridine. Decitabine-cedazuridine is an oral formulation of the injectable hypomethylating agent decitabine, combined with cedazuridine to prevent its breakdown. Patients take decitabine-cedazuridine for the first five days of each cycle.
Results
According to the trial data:
- 46.5% of patients achieved a complete response.
- 63% achieved either a complete response or a complete response with incomplete hematologic recovery.
- Median overall survival was 15.5 months.
Researchers stated this survival outcome is comparable to existing intravenous therapies.
Safety and Monitoring
The safety profile was consistent with standard AML therapies. Common serious side effects included anemia, neutropenia, and febrile neutropenia. Researchers recommend monitoring leukemia cells and pausing venetoclax to allow blood cell recovery.
Publication
Results were published on June 3 in the New England Journal of Medicine.
Future Directions
Lead author Dr. Gail J. Roboz stated that the oral regimen is expected to become a standard of care for older or frail patients. She noted that most patients must continue treatment to maintain remission but can have good quality of life. Future research aims to identify when treatment can be stopped and to explore triplet therapies.
Funding
The study was funded by Taiho Oncology, Inc., the manufacturer of INQOVI (decitabine-cedazuridine).