Acute myeloid leukaemia (AML) predominantly occurs in older adults over 65 years, who exhibit a lower tolerance to conventional induction chemotherapy (IC) compared with younger patients.
Despite treatment with IC, older patients with AML have poor long-term survival without haematopoietic stem cell transplantation (HSCT).
For the past decade, DNA-hypomethylating agents such as decitabine have been considered a safer alternative for patients who are unfit for IC.
Prolonged (10-day) decitabine treatment has shown promising efficacy in previous studies with older patients with AML, and may be a more suitable treatment before HSCT than IC in fit patients.
The open-label randomized phase III study of the EORTC Leukaemia group, GIMEMA, CELG, and GMDSSG (NCT02172872) compared the efficacy and safety of 10-day decitabine with conventional 3+7 IC as a treatment before HSCT in older (≥60 years) patients with newly diagnosed AML.
Patients with at least stable disease and an HLA-matched donor were encouraged to undergo HSCT after ≥1 treatment cycle.
The rate of HSCT in decitabine-treated patients was similar to IC-treated patients (40% vs 39%). Although IC achieved higher complete remission rates compared with decitabine (61% vs 48%), the overall survival was comparable, with a median overall survival of 15 months in the decitabine group and 18 months in the IC group. At 4 years, 26% of patients from the decitabine arm and 30% of patients from the IC arm were alive.
A notable difference between the treatment arms was the incidence of grade 3–5 adverse events before HSCT.
Decitabine treatment showed lower incidences of febrile neutropenia, platelet reduction, oral mucositis, and diarrhoea. In addition, the 30-day mortality rate was 3.6% for decitabine compared with 6.4% for IC.
In conclusion, decitabine treatment for older patients with AML exhibited a superior safety profile compared with IC while maintaining similar overall survival and rates of HSCT.