Amy Burd, PhD, Vice President of Research Strategy at the Leukemia and Lymphoma Society (LLS), discusses her group’s multiarm, multisite collaborative trial which aims to test targeted therapy approaches for improving the generally poor prognosis among patients with AML.

Acute myeloid leukemia (AML) is a cancer that affects the blood and bone marrow. Conditions are generally called “acute” when they develop quickly and have an aggressive course. The signs and symptoms of AML vary but may include easy bruising; bone pain or tenderness; fatigue; fever; frequent nosebleeds; bleeding from the gums; shortness of breath; and/or weightloss. AML is one of the most common types of leukemia among adults and is rarely diagnosed in people under age 40. There are many potential causes of AML such as certain blood disorders, inherited syndromes, environmental exposures, and drug exposures; however, most people who develop AML have no identifiable risk factor. Treatment may include a combination of chemotherapy, radiation therapy, bone marrow transplant and/or other drug therapy.

Dr. Burd discusses the feasible for doctors to determine which molecular subtype of AML a patient has before beginning treatment and to use this information to pick an approach that best matches the individual. The results of this study confirm that using patient-specific information to guide treatment decisions, an approach known as precision medicine, is possible even for patients with blood cancers that must be treated urgently.

Because acute myeloid leukemia (AML) is a rapidly progressing cancer it is considered an oncologic emergency. That is why treatment is typically started on the day of diagnosis. Physicians have been reluctant to wait the two to three weeks that it typically takes for genomic analysis. Delaying induction chemotherapy until molecular testing results return, may benefit some patients but can harm others. 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.

Treatment with immediate initiation of therapy is thought to be crucial to minimizing disease-related morbidity and mortality. And, in many cases this approach has been beneficial. In an ideal, younger, patient with a core binding factor cytogenetic abnormality, who is given standard anthracycline- and cytarabine-based induction chemotherapy, complete remission (CR) rates approach 85%, with long-term disease-free survival (DFS) rates of 60% or greater. [3]

But in most, less ideal, patients, for example older patients or who have other cytogenetic abnormalities or secondary AML, outcome after standard therapy is much worse, with CR rates less than 50%, treatment-related mortality rates that approach 25%, and minimal long-term DFS.