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ASH 2018: Rapid Genetic Screening Shows Feasibility of Precision Medicine for AML

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Key Points

  • Eligible patients were profiled using local cytogenetics and a next-generation sequencing assay, with all molecular data required for treatment assignment obtained within 7 days. 
  • Researchers assigned treatment within 7 days in 106 of the 109 patients (97.2%) in the feasibility phase and 200 of 210 (95.2%) of the overall cohort.

A new study demonstrated it is feasible for health-care providers to determine which molecular subtype of acute myeloid leukemia (AML) a patient has before beginning treatment and to use this information to pick an approach that best matches the individual. The results, presented by Burd et al at the 2018 American Society for Hematology (ASH) Annual Meeting & Exposition (Abstract 559), showed that using patient-specific information to guide treatment decisions is possible, even for patients with blood cancers that must be treated urgently.

Because AML is a rapidly progressing cancer, treatment is typically started on the day of diagnosis, and physicians have been traditionally reluctant to wait the 2 to 3 weeks for genomic analysis. This leaves doctors with little time to learn which AML subtype the patient has; so in current practice, all patients are given the same treatment regimen. 

However, in this study, researchers demonstrated the ability to determine the AML subtype based on genetic analysis of blood samples within 7 days. The findings suggest rapid genetic screening could soon be an integral part of AML diagnostics.

Beat AML Study

The results represent the first findings from the Beat AML study, a multiarm, multisite collaborative trial designed to test precision medicine approaches for improving the generally poor prognosis among patients with AML. Many experimental new therapies target specific AML subtypes, so the ability to determine a patient’s AML subtype is crucial to realizing the full benefits of these therapies, said lead study author Amy Burd, PhD, Vice President for Research Strategy at The Leukemia & Lymphoma Society (LLS).

Eligible patients were profiled using local cytogenetics and a next-generation sequencing (NGS) assay, with all molecular data required for treatment assignment obtained within 7 days. Enrollment took place from 2016 to 2018, and at data cutoff, 268 patients had enrolled—43% were female, with a median age of 72 years.

Key Findings

Of the 268 patients, 210 had AML with treatment assignment. All patients had cytogenetic results available by 7 days. Researchers assigned treatment within 7 days in 106 of the 109 patients (97.2%) in the feasibility phase and 200 of 210 patients (95.2%) of the overall cohort, meeting feasibility requirements. 

The study is designed to incorporate new experimental therapies as they are developed. Having started in 2016 with 3 treatment arms, the study has now grown to include 11 arms testing therapies developed by 7 different pharmaceutical companies. Initial results from some of these studies suggest patients benefit from therapies specifically chosen based on their individual disease subtype, such as TP53 mutation–positive, NPM1-positive FLT3-ITD–negative, and even marker-negative disease.

“I think the future of treatment for AML will include point-of-care screening to determine what type of AML the patient has and then make the treatment decision based on that information,” said Dr. Burd. “Being able to do genetic screening rapidly and efficiently is critical to making a decision for that patient within 7 days. This study demonstrates that the precision medicine approach is feasible and effective.”

Beat AML, LLC, a division of LLS, a nonprofit organization dedicated to fighting blood cancers, is the sponsor of the trial and holds the investigational new drug approval from the U.S. Food and Drug Administration. ASH is an exclusive partner with LLS in educating physicians about the Beat AML initiative.

Disclosure: See the study authors’ full disclosures at ash.confex.com.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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