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Impact of NGS on Treatment Selection in AML

Next-generation sequencing (NGS) has the potential to directly impact treatment decisions and outcomes in patients with acute myeloid leukemia (AML), according to a study presented at the American Society of Clinical Oncology (ASCO) Annual Meeting (May 29-31, 2020).

In recent years, therapy options such as targeted agents have increased for patients with AML. The advent of NGS and such agents calls into question how broader use of testing will impact treatment decisions and patient outcomes.

Rita Elias Assi, MD, The University of Texas MD Anderson Cancer Center, and colleagues designed an analysis to evaluate the impact of NGS on treatment selection in AML. Researchers included 1470 patients with AML from October 2012 to June 2016 who had available NGS-based detection of somatic mutations. Seventeen genes (ALK, CSF1R, FGFR1/2/3, FLT3, IDH1/2, JAK2, KDR, KRAS/NRAS, NPM1, PDGFRA, PTPN11, RET, and TP53) were considered potentially actionable because of available standard or investigational targeted agents.

Dr Assi and colleagues found that among the 1271 treated patients, 77% (n = 982) had a median of two actionable mutations: TP53 (n = 241), IDH2 (n = 240), IDH1 (n = 238), FLT3 (n = 200), NPM1 (n = 195), NRAS (n = 175), JAK2 (n = 103), and KRAS (n = 82). Importantly, 41% (n = 271 of 518) of patients started new therapy after NGS results were available. NGS results guided targeted therapy in 36% of patients, among whom 53% enrolled in clinical trials and 6% received off-label agents.

Considering actionable mutations only, patients with relapsed or refractory AML were more likely to receive targeted therapy compared with newly diagnosed patients (51% vs 23%, respectively; P < .0001).

Furthermore, researchers noted that patients who received targeted therapy had a higher response rate compared with patients who did not, regardless of having relapsed or refractory disease (31% vs 21%, respectively; P = .001) or newly diagnosed disease (72% vs 60%, respectively; P = .04).

In their concluding remarks, authors of the study wrote that the results of ongoing clinical trials involving NGS and targeted therapies may change the way patients with AML are treated.—Zachary Bessette