A retrospective analysis of older patients with acute myeloid leukemia (AML) treated with the hypomethylating agent decitabine at 8 Italian hematological centers from 2015 to 2017 supports the efficacy and safety of the treatment in this patient population, a study published in Leukemia Research has shown. 

Despite the fact that AML most commonly occurs in older patients, decisions related to the first-line treatment of these patients, as well as the treatment of patients with relapsed/refractory disease, are often complicated. The high frequency of organ dysfunction, poor performance status, unfavorable cytogenetics, and multidrug-resistant disease are some of the reasons underlying the poor outcomes and limited availability of treatment options for older patients with AML. Intensive chemotherapy, the standard-of-care for younger, healthier patients, is often not considered for these patients. 

Results from an open-label, phase III randomized clinical trial comparing decitabine with physician choice of supportive care or low dose cytarabine as first-line treatment of newly diagnosed older patients with AML have previously shown improved response rates for decitabine compared with the latter options, with similar safety profiles for these approaches. Nevertheless, questions remain regarding the risks and benefits of decitabine use for older patients with AML in a real-world clinical setting, for patients with both newly diagnosed and relapsed/refractory disease. 

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One hundred and four patients with a median age of 72.5 years were included in this retrospective analysis. Baseline disease-related features confirmed the presence of unfavorable prognostic factors in this group, such as secondary AML (43% of patients), bone marrow blast cells >30% (64%), and grade 3 or higher cytopenia (76%), although only 16% of patients had an Eastern Cooperative Oncology Group (ECOG) performance status greater than 2. Decitabine was administered as first-line and salvage therapy in 72% and 29% of patients, respectively. 

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The overall response rate (ORR) for older patients receiving first-line decitabine (42%) was significantly better compared with the ORR for those receiving this drug in the salvage setting (14%; P= .009). In addition, median overall survival in the complete cohort was significantly longer for patients achieving a response (22.6 vs 5.7 months; P< .0001). Although decitabine was generally well tolerated clinically, grade 3 or higher hematologic adverse events occurred in more than 90% of patients, although these were observed to diminish after 4 cycles of treatment. Common nonhematologic adverse events included infections, such as pneumonia and fever of unknown origin. 

“These data confirm the efficacy and the acceptable safety profile of decitabine in the real life management of AML in elderly patients unsuitable for intensive chemotherapy, with a significant better performance in first-line therapy,” the authors wrote in summarizing these results. 


Filì C, Candoni A, Zannier ME, et al. Efficacy and toxicity of decitabine in patients with acute myeloid leukemia (AML): A multicenter real-world experience. Leuk Res.2019;76:33-38.