Abstract
We recently published a clinically-meaningful improvement in median overall survival (OS) for patients with acute myeloid leukaemia (AML), >30% bone marrow (BM) blasts and white blood cell (WBC) count ≤15 G/L, treated with front-line azacitidine versus conventional care regimens within a phase 3 clinical trial (AZA-AML-001; NCT01074047; registered: February 2010). As results obtained in clinical trials are facing increased pressure to be confirmed by real-world data, we aimed to test whether data obtained in the AZA-AML-001 trial accurately represent observations made in routine clinical practice by analysing additional AML patients treated with azacitidine front-line within the Austrian Azacitidine Registry (AAR; NCT01595295; registered: May 2012) and directly comparing patient-level data of both cohorts. We assessed the efficacy of front-line azacitidine in a total of 407 patients with newly-diagnosed AML. Firstly, we compared data from AML patients with WBC ≤ 15 G/L and >30% BM blasts included within the AZA-AML-001 trial treated with azacitidine (“AML-001” cohort; n = 214) with AAR patients meeting the same inclusion criteria (“AAR (001-like)” cohort; n = 95). The current analysis thus represents a new sub-analysis of the AML-001 trial, which is directly compared with a new sub-analysis of the AAR. Baseline characteristics, azacitidine application, response rates and OS were comparable between all patient cohorts within the trial or registry setting. Median OS was 9.9 versus 10.8 months (p = 0.616) for “AML-001” versus “AAR (001-like)” cohorts, respectively. Secondly, we pooled data from both cohorts (n = 309) and assessed the outcome. Median OS of the pooled cohorts was 10.3 (95% confidence interval: 8.7, 12.6) months, and the one-year survival rate was 45.8%. Thirdly, we compared data from AAR patients meeting AZA-AML-001 trial inclusion criteria (n = 95) versus all AAR patients with World Health Organization (WHO)-defined AML (“AAR (WHO-AML)” cohort; n = 193). Within the registry population, median OS for AAR patients meeting trial inclusion criteria versus all WHO-AML patients was 10.8 versus 11.8 months (p = 0.599), respectively. We thus tested and confirmed the efficacy of azacitidine as a front-line agent in patients with AML, >30% BM blasts and WBC ≤ 15 G/L in a routine clinical practice setting. We further show that the efficacy of azacitidine does not appear to be limited to AML patients who meet stringent clinical trial inclusion criteria, but instead appears efficacious as front-line treatment in all patients with WHO-AML.
Highlights
Acute myeloid leukaemia (AML) is predominantly a disease of the elderly, with a median age at diagnosis of roughly 70 years [1]
We further selected for the presence of >30% bone marrow (BM) blasts and ≤15 G/L white blood cell (WBC) and directly compared baseline and treatment characteristics, as well as treatment outcomes and overall survival (OS) of patients included within the AZA-acute myeloid leukaemia (AML)-001 trial (n = 214; “AML-001” subset) versus patients included within the Azacitidine Registry (AAR) (n = 95; “AAR (001-like)” subset), using patient-level data from both cohorts (Figure 1)
We tested and confirmed the efficacy of azacitidine as a front-line agent in patients with AML treated within the Arbeitsgemeinschaft Medikamentöse Tumortherapie (AGMT)-AAR
Summary
Acute myeloid leukaemia (AML) is predominantly a disease of the elderly, with a median age at diagnosis of roughly 70 years [1]. In 2008, azacitidine was approved by the European Medicines Agency (EMA) for the treatment of AML patients with 20%–30% bone marrow (BM) blasts, older than 64 years and who are ineligible for HSCT. For AML patients with more than 30% BM blasts, azacitidine remained an off-label indication until 30 October 2015 and was not reimbursed in most countries. These patients were either treated with other options or not treated at all, which is substantiated by a large population-based study published in 2012, showing that only 38% of 5480 AML patients older than 65 years received leukaemia therapy, whereas 62% received BSC [6].
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
