Real-world data confirming the efficacy and safety of decitabine in acute myeloid leukaemia – results from a retrospective Belgian registry study

Abstract

Objectives: Currently, there is no standard treatment for patients with acute myeloid leukaemia (AML) ineligible for standard induction chemotherapy (IC). This study aimed to report real-world evidence data on the efficacy and safety of decitabine in this patient group. Methods: This study was a Belgian, retrospective, non-interventional, multicentre registry of patients ≥ 65 years, with newly-diagnosed de novo or secondary AML ineligible for IC. Patients were treated according to routine clinical practice. Overall survival (OS), progressionfree survival (PFS) and transfusion independence for ≥8 consecutive weeks were evaluated. Results: Forty-five patients were enrolled, including 67% (n = 30) with secondary AML. Median OS and PFS were 7.3 months (95% CI: 2.2-11.1) and 4.1 months (95% CI: 2.1-7.6) respectively. A subpopulation analysis showed that patients treated with ≥4 cycles (n = 21) had significantly better outcomes compared to patients receiving <4 cycles (median OS 17.5 vs 1.6 months; median PFS 17.5 vs. 1.4 months). Twenty-five percent and 58% of patients that were respectively RBC or platelet transfusion-dependent at baseline became transfusion independent during treatment. Conclusion: This real-world data confirms that decitabine can lead to transfusion independence and longer OS in AML patients, particularly after administering ≥4 cycles, as indicated in the summary of product characteristics.

The Clinical Value of Decitabine Monotherapy in Patients with Acute Myeloid Leukemia

“…When interpreting the safety data, it is important to note that exposure to medication was 83% longer in the decitabine arm (median 4.4 months) than the TC arm (median 2.4 months with cytarabine), in both the initial (2009) and mature (2010) analyses. Data from the two DACO trials and real-world studies show that the toxicity associated with decitabine is relatively mild and AEs are manageable [23][24][25][26], with no additional AEs report in realworld studies compared with those reported in the DACO-016 and DACO-017 trials [15,16].…”

“…Patients who received at least four cycles (n = 21) seemed to have significantly better outcomes than patients receiving fewer than four cycles of decitabine (median OS 17.5 vs. 1.6 months, and median PFS 17.5 vs. 1.4 months, respectively). Overall, 25% of the patients who were RBC infusion dependent and 58% of those who were platelet transfusion dependent at baseline became transfusion independent [22,23].…”

The Role of Venetoclax in Relapsed/Refractory Acute Myeloid Leukemia: Past, Present, and Future Directions

“…Driven by the promising efficacy data on heavily pretreated patients and the relatively favorable toxicity profile, the clinical development of VEN in AML rapidly shifted towards the frontline setting for elderly/unfit patients, where therapeutic standards [20][21][22] (HMAs and low-dose cytarabine (LDAC)) had been providing unsatisfactory outcomes in clinical practice [23][24][25][26][27]. Moreover, the possibility of increasing treatment efficacy without excessive toxicity with an orally available drug was valuable.…”

In Pursuit of Genetic Prognostic Factors and Treatment Approaches in Secondary Acute Myeloid Leukemia—A Narrative Review of Current Knowledge

“…87% of patients in each treatment arm experienced grade 3–4 adverse effects, of which the most frequent was thrombocytopenia in patients treated with conventional chemotherapy and febrile neutropenia in patients treated with azacytidine. Meers et al [ 85 ] reported the data on the efficacy and safety of decitabine in AML. The study proved that the treatment with decitabine leads to better overall survival and progression-free survival [ 85 ].…”

“…Meers et al [ 85 ] reported the data on the efficacy and safety of decitabine in AML. The study proved that the treatment with decitabine leads to better overall survival and progression-free survival [ 85 ].…”