Jad Othman , Ho Pui Jeff Lam , Sarah Leong , Faisal Basheer , Islam Abdallah , Kathryn Fleming , Priyanka Mehta , Heba Yassin , John Laurie , Michael Austin , Paolo Gallipoli , Tom Taylor , Mike Dennis , Johnathon Elliot , Georgina Clarke , Raymond Dang , Jennifer Vidler , Pramila Krishnamurthy , Anne-Louise Latif , Pallavi Kalkur , Richard Dillon
{"title":"Real-world outcomes of newly diagnosed AML treated with venetoclax and azacitidine or low-dose cytarabine in the UK NHS","authors":"Jad Othman , Ho Pui Jeff Lam , Sarah Leong , Faisal Basheer , Islam Abdallah , Kathryn Fleming , Priyanka Mehta , Heba Yassin , John Laurie , Michael Austin , Paolo Gallipoli , Tom Taylor , Mike Dennis , Johnathon Elliot , Georgina Clarke , Raymond Dang , Jennifer Vidler , Pramila Krishnamurthy , Anne-Louise Latif , Pallavi Kalkur , Richard Dillon","doi":"10.1016/j.bneo.2024.100017","DOIUrl":null,"url":null,"abstract":"<div><h3>Abstract</h3><p>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 <em>NPM1</em>, <em>RUNX1</em>, <em>STAG2</em>, and <em>IDH2</em> were associated with improved survival, whereas age, secondary and therapy-related AML, +8, <em>MECOM</em> rearrangements, complex karyotype, <em>ASXL1</em>, and <em>KIT</em> 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 <em>NPM1</em> mutated AML, outcomes were similar for those treated with venetoclax-azacitidine and venetoclax-LDAC.</p></div>","PeriodicalId":100189,"journal":{"name":"Blood Neoplasia","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2024-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2950328024000177/pdfft?md5=1de290f83adace13a5f6e05842b39ad6&pid=1-s2.0-S2950328024000177-main.pdf","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Blood Neoplasia","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2950328024000177","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
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.
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