The Evolution of Care for Acute Myeloid Leukemia and the Challenges of Defining Value

Abstract

A dult acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It is the second most common type of acute leukemia in adults. AML is also sometimes referred to as acute myelogenous leukemia, acute myeloblastic leukemia, acute granulocytic leukemia, and acute nonlymphocytic leukemia. In AML, there is a block in differentiation and uncontrolled proliferation of myeloid precursors, and the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). 1 The myeloblasts in AML are abnormal and do not become healthy white blood cells. Leukemia cells, or leukemic stem cells (LSCs), can build up in the bone marrow and blood, so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or hemorrhaging may occur. The leukemia cells can spread outside the blood to other parts of the body. AML is the most common type of acute leukemia in adults, and its incidence increases with age. 2 This year, an estimated 21,380 people of all ages (11,960 men and 9420 women) in the United States will be diagnosed with AML. 3 It is primarily a disease of older adults; the disease rarely occurs before age 45, and the median age of onset is around 67 years. With post-remission therapy, 5-year survival rates of <5% to 20% and >40% may be achieved for patients older and younger than 60 years, respectively. 3

Toward Integrated Genomic Diagnosis in Routine Diagnostic Pathology by the World Health Organization Classification of Acute Myeloid Leukemia

“…All of the above advances now increasingly necessitate comprehensive, integrated diagnostics, including genomics for AML diagnosis, prognosis, and therapy. Considerations to implement such testing for AML include (1) first, correct diagnostic classification of AML with combined testing modalities and multidisciplinary teams as described above, with the pathologist best able to integrate all findings, (2) cost of testing, which is likely to be much less than treatment costs [56], and (3) turnaround time, which can take much longer by NGS than single-gene PCR assays, especially for FLT3 and IDH1/IDH2 mutations. Apart from institution-specific assays [57], commercial assays to allow clinical NGS results in possibly two days from sample collection will soon be available for AML [58].…”