Real-world outcomes of newly diagnosed AML treated with venetoclax and azacitidine or low-dose cytarabine in the UK NHS
Othman, Jad Lam, Ho Pui Jeff Leong, Sarah Basheer, Faisal Abdallah, Islam Fleming, Kathryn Mehta, Priyanka Yassin, Heba Laurie, John Austin, Michael
Othman, Jad
Lam, Ho Pui Jeff
Leong, Sarah
Basheer, Faisal
Abdallah, Islam
Fleming, Kathryn
Mehta, Priyanka
Yassin, Heba
Laurie, John
Austin, Michael
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Affiliation
King's College London; Guy's and St Thomas' NHS Foundation Trust; University of Sydney; Sandwell and West Birmingham NHS Trust; et al.
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Publication date
2024-05-23
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Abstract
Venetoclax with azacitidine is the standard of care for patients with acute myeloid leukemia (AML) who are unfit for intensive chemotherapy; however, uncertainties remain regarding the treatment schedule, accurate prognostication, and outcomes for patients treated outside clinical trials. The option of venetoclax with low-dose cytarabine (LDAC) is also available; however, it is not clear for which patients it may be a useful alternative. Here, we report a large real-world cohort of 654 patients treated in 53 UK hospitals with either venetoclax and azacitidine (n = 587) or LDAC (n = 67). The median age was 73 years, and 59% had de novo AML. Most patients received 100 mg of venetoclax with an azole antifungal. In cycle 1, patients spent a median of 14 days in the hospital, and 85% required red cell transfusion, 59% platelet transfusion, and 63% required IV antibiotics. Supportive care requirements significantly reduced after the first cycle. Patients receiving venetoclax-azacitidine had a complete remission (CR)/CR with incomplete hematological recovery rate of 67%, day 30 and day 60 mortality of 5% and 8%, respectively, and median overall survival of 13.6 months. Mutations in NPM1, RUNX1, STAG2, and IDH2 were associated with improved survival, whereas age, secondary and therapy-related AML, +8, MECOM rearrangements, complex karyotype, ASXL1, and KIT mutations were associated with poorer survival. Prognostic systems derived specifically for patients treated with venetoclax-azacitidine performed better than the European LeukemiaNet and Medical Research Council classifications; however, improved risk classifications are still required. In the 149 patients with NPM1 mutated AML, outcomes were similar for those treated with venetoclax-azacitidine and venetoclax-LDAC.
Citation
Othman J, Lam HPJ, Leong S, Basheer F, Abdallah I, Fleming K, Mehta P, Yassin H, Laurie J, Austin M, Gallipoli P, Taylor T, Dennis M, Elliot J, Clarke G, Dang R, Vidler J, Krishnamurthy P, Latif AL, Kalkur P, Shahidianakbar M, Campbell V, Mannari D, Sutherland E, Wickramaratne T, Collins A, Zhao R, Mak H, Belsham E, Banerjee S, Bashir J, Pillai S, Whitmill R, Galli S, Amer M, Murthy V, Murray D, Wandroo F, Hogan F, Crolla F, Fowler N, Khan A, O'Nions J, Dillon R. Real-world outcomes of newly diagnosed AML treated with venetoclax and azacitidine or low-dose cytarabine in the UK NHS. Blood Neoplasia. 2024 May 23;1(3):100017. doi: 10.1016/j.bneo.2024.100017
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Article