Extended treatment with decitabine appears as effective as, but safer than, standard induction chemotherapy in older patients with acute myeloid leukemia (AML), according to research presented at the EHA 2022 Hybrid Congress.

A phase 3 study showed that extended decitabine produced a similar overall survival (OS) rate as standard chemotherapy, but decitabine had a better safety profile. 

The randomized trial (ClinicalTrials.gov Identifier: NCT02172872) included 606 patients with newly diagnosed de novo or secondary, untreated AML who were 60 years of age or older. 

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The 303 patients in the decitabine arm received 10 days of decitabine (20 mg/m2) in cycle 1 and either 10 or 5 days in subsequent cycles, depending on bone marrow blast clearance at day 28. 

The 303 patients in the standard chemotherapy arm received daunorubicin (60 mg/m2) for 3 days, followed by cytarabine (200 mg/m2) for 7 days, followed by 1 to 3 additional chemotherapy cycles. 

Patients with an HLA-matched donor and at least stable disease were encouraged to undergo allogeneic HSCT after completing at least 1 treatment cycle. Patients in the decitabine arm who did not undergo HSCT could continue on decitabine treatment.

The median age was 67 years in the decitabine arm and 68 years in the standard chemotherapy arm. About one-third of patients in each arm were 70 years of age or older, roughly half had normal karyotype, and 18% in each arm had TP53 mutations. 

The rate of complete response (CR) or CR with incomplete count recovery was 48% with decitabine and 61% with standard chemotherapy. The proportion of patients who underwent HSCT on protocol was 40% in the decitabine arm and 39% in the standard chemotherapy arm.

In the overall cohort, OS was not significantly different between the treatment arms. The 4-year OS rate was 26% with decitabine and 30% with standard chemotherapy (hazard ratio, 1.04; 95% CI, 0.86-1.26; P =.68). 

The rates of progression and transplant-related mortality (TRM) were similar between the treatment arms as well. The rate of progression at 4 years was 51% in the standard chemotherapy arm and 57% in the decitabine arm. The rate of TRM was 25% and 21%, respectively.

A subgroup analysis for OS showed that patients ages 60-64 and patients with NPM1 mutations benefited more from standard chemotherapy. Patients age 70 or older and those with monosomal karyotype-positive disease benefited more from decitabine. 

When the researchers looked at HSCT recipients alone, the 4-year OS rate was 47% in the standard chemotherapy arm and 45% in the decitabine arm. The rate of progression was 22% and 24%, respectively. The rate of TRM was 33% and 31%, respectively.

During induction, there were generally more grade 3-5 adverse events in the standard chemotherapy arm than in the decitabine arm. In particular, rates of blood and lymphatic disorders, infections, and gastrointestinal disorders were all higher with standard chemotherapy. 

The incidence of fatal treatment-related adverse events after HSCT was comparable between the treatment arms (25% with decitabine and 22% with standard chemotherapy). 

The researchers also looked at health economics and found that decitabine resulted in fewer days of hospitalization (P =.007), fewer patients treated with IV antibiotics (P <.001), and fewer red blood cell (P =.024) and platelet (P <.001) transfusion needs, compared with standard chemotherapy. 

Disclosures: This research was supported by Janssen. The presenter declared affiliations with Janssen, AbbVie, Astex Pharmaceuticals, Imago BioSciences, Otsuka, Syros, and Cheplapharm. 


Lubbert M, Wijermans P, Kicinski M, et al. 10-day decitabine vs. conventional chemotherapy (‘3+7’) followed by allografting (HSCT) in AML patients ≥60 years: A randomized phase III study of the EORTC Leukemia Group, GIMEMA, CELG, and GMDS-SG. Presented at EHA 2022; June 9-12, 2022. Abstract S125.