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Rebecca Olin, MD, MS


Using Targeted Therapy in Older Patients With AML

By: Lauren Harrison, MS
Posted: Sunday, March 1, 2020

Use of a novel precision medicine approach to select treatment based on cytogenetic and molecular alterations for older patients with acute myeloid leukemia (AML) produced improved overall survival compared with standard-of-care treatment. These results were presented at the 2019 American Society of Hematology (ASH) Annual Meeting & Exposition by Amy Burd, PhD, of the Leukemia and Lymphoma Society in White Plains, New York (Abstract 175) and published in the journal Blood.

“Our data support the feasibility of a rapid precision medicine approach in older patients with previously untreated AML,” the investigators concluded.

This trial enrolled 374 patients with treatment-naive AML who were older than age 60. Patients were profiled by local cytogenetics analysis and next-generation sequencing. The data were used for treatment assignment, which was conducted using a predetermined algorithm that considered somatic cytogenetic and molecular alterations in the dominant clone as well as available targeted therapies for AML and the likelihood of cure with intensive therapy. The most common cytogenetic/genomic groups were TP54-mutated (19%) and marker negative (18%) molecular groups.

A total of 103 patients received standard therapy, which was defined as induction therapy or hypomethylation agent. An additional 28 patients received an alternative investigational agent, and 38 opted for palliative care. The remaining 224 patients elected to be part of the BEAT AML substudy and received targeted treatment. Demographic, clinical, performance, and molecular characteristics did not differ among patients who elected for standard versus targeted therapies.

The overall survival was significantly longer for patients who received targeted therapy compared with standard therapy, with median overall survival of 12.8 months and 3.9 months, respectively. The 30-day death rate was 3.7% for patients in the targeted-therapy group, compared with a 20.4% death rate in the standard-therapy group, 0% in the investigational group, and 72.6% in the palliative-care group.

Disclosure: For full disclosures of the study authors, visit

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