Precision medicine treatment in acute myeloid leukemia using prospective genomic profiling: Feasibility and preliminary efficacy of the Beat AML Master Trial Research Letter
| Authors: | Burd, A.; Levine, R. L.; Ruppert, A. S.; Mims, A. S.; Borate, U.; Stein, E. M.; Patel, P.; Baer, M. R.; Stock, W.; Deininger, M.; Blum, W.; Schiller, G.; Olin, R.; Litzow, M.; Foran, J.; Lin, T. L.; Ball, B.; Boyiadzis, M.; Traer, E.; Odenike, O.; Arellano, M.; Walker, A.; Duong, V. H.; Kovacsovics, T.; Collins, R.; Shoben, A. B.; Heerema, N. A.; Foster, M. C.; Vergilio, J. A.; Brennan, T.; Vietz, C.; Severson, E.; Miller, M.; Rosenberg, L.; Marcus, S.; Yocum, A.; Chen, T.; Stefanos, M.; Druker, B.; Byrd, J. C. |
| Title: | Precision medicine treatment in acute myeloid leukemia using prospective genomic profiling: Feasibility and preliminary efficacy of the Beat AML Master Trial |
| Abstract: | Acute myeloid leukemia (AML) is the most common diagnosed leukemia. In older adults, AML confers an adverse outcome1,2. AML originates from a dominant mutation, then acquires collaborative transformative mutations leading to myeloid transformation and clinical/biological heterogeneity. Currently, AML treatment is initiated rapidly, precluding the ability to consider the mutational profile of a patient’s leukemia for treatment decisions. Untreated patients with AML ≥ 60 years were prospectively enrolled on the ongoing Beat AML trial (ClinicalTrials.gov NCT03013998), which aims to provide cytogenetic and mutational data within 7 days (d) from sample receipt and before treatment selection, followed by treatment assignment to a sub-study based on the dominant clone. A total of 487 patients with suspected AML were enrolled; 395 were eligible. Median age was 72 years (range 60–92 years; 38% ≥75 years); 374 patients (94.7%) had genetic and cytogenetic analysis completed within 7 d and were centrally assigned to a Beat AML sub-study; 224 (56.7%) were enrolled on a Beat AML sub-study. The remaining 171 patients elected standard of care (SOC) (103), investigational therapy (28) or palliative care (40); 9 died before treatment assignment. Demographic, laboratory and molecular characteristics were not significantly different between patients on the Beat AML sub-studies and those receiving SOC (induction with cytarabine + daunorubicin (7 + 3 or equivalent) or hypomethylation agent). Thirty-day mortality was less frequent and overall survival was significantly longer for patients enrolled on the Beat AML sub-studies versus those who elected SOC. A precision medicine therapy strategy in AML is feasible within 7 d, allowing patients and physicians to rapidly incorporate genomic data into treatment decisions without increasing early death or adversely impacting overall survival. © 2020, The Author(s), under exclusive licence to Springer Nature America, Inc. |
| Journal Title: | Nature Medicine |
| Volume: | 26 |
| Issue: | 12 |
| ISSN: | 1078-8956 |
| Publisher: | Nature Publishing Group |
| Date Published: | 2020-12-01 |
| Start Page: | 1852 |
| End Page: | 1858 |
| Language: | English |
| DOI: | 10.1038/s41591-020-1089-8 |
| PUBMED: | 33106665 |
| PROVIDER: | scopus |
| PMCID: | PMC8530434 |
| DOI/URL: | |
| Notes: | Article -- Export Date: 4 January 2021 -- Source: Scopus |
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