Alternative option to intensive chemotherapy for patients with relapsed/refractory acute myeloid leukemia (AML)

Patients with RR-AML have poor survival and limited treatment options. HMAs are a type of epigenetic therapy that changes the methylation patterns of DNA rather than killing cells the traditional way like chemotherapy does. The two commonly used HMAs in the Unite States are azacitidine and decitabibe, although they are not yet FDA approved for this indication. In general, HMAs are better tolerated with fewer side effects compared to traditional chemotherapy. Patients are also able to receive their therapy in the outpatient setting.

Led by Maximilian Stahl, MD (first author) and Amer Zeidan, MD (senior author), study authors collected data from 11 cancer centers in the United States and Europe from 2006-2016 and analyzed the records of 656 patients treated with HMAs for RR-AML. The authors found that 16% of patients achieved complete response (CR) or complete response with incomplete count recovery (CRi). Those patients who achieved CR/CRi had a median survival of 19 months. The median survival for all patients in the study was only 6.5 months. Further analysis of the clinical and molecular predictors of response and survival is ongoing.

"These results suggest that outpatient therapy with HMAs is a reasonable option for patients with RR-AML who don't have clinical trial options available to them," said Maximilian Stahl, MD.