- Burd, Amy;
- Levine, Ross L;
- Ruppert, Amy S;
- Mims, Alice S;
- Borate, Uma;
- Stein, Eytan M;
- Patel, Prapti;
- Baer, Maria R;
- Stock, Wendy;
- Deininger, Michael;
- Blum, William;
- Schiller, Gary;
- Olin, Rebecca;
- Litzow, Mark;
- Foran, James;
- Lin, Tara L;
- Ball, Brian;
- Boyiadzis, Michael;
- Traer, Elie;
- Odenike, Olatoyosi;
- Arellano, Martha;
- Walker, Alison;
- Duong, Vu H;
- Kovacsovics, Tibor;
- Collins, Robert;
- Shoben, Abigail B;
- Heerema, Nyla A;
- Foster, Matthew C;
- Vergilio, Jo-Anne;
- Brennan, Tim;
- Vietz, Christine;
- Severson, Eric;
- Miller, Molly;
- Rosenberg, Leonard;
- Marcus, Sonja;
- Yocum, Ashley;
- Chen, Timothy;
- Stefanos, Mona;
- Druker, Brian;
- Byrd, John C
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.