Acute myeloid leukemia

Acute myeloid leukemia (AML), also known as acute myelogenous leukemia or acute nonlymphocytic leukemia (ANLL), is a cancer of the blood and bone marrow characterized by the rapid, uncontrolled proliferation of abnormal myeloid progenitor cells (myeloblasts). These immature cells accumulate in the bone marrow, interfering with the production of normal blood cells, and can spread into the bloodstream and other organs. AML primarily affects the hematopoietic (blood-forming) system but can involve the central nervous system, skin, gums, liver, spleen, and lymph nodes. The disease can arise de novo or secondary to prior chemotherapy, radiation therapy, or progression from myelodysplastic syndromes or other hematologic disorders. Key symptoms result from bone marrow failure and include fatigue, weakness, pallor, and shortness of breath due to anemia; recurrent or severe infections due to neutropenia (low white blood cell counts); and easy bruising, petechiae, and abnormal bleeding due to thrombocytopenia (low platelet counts). Patients may also experience fever, bone pain, weight loss, and hepatosplenomegaly (enlargement of the liver and spleen). Gingival hyperplasia (swollen gums) and skin infiltration (leukemia cutis) are particularly associated with certain AML subtypes, especially those with monocytic differentiation. AML is classified according to the World Health Organization system based on genetic and molecular abnormalities, which are critical for prognosis and treatment decisions. Numerous recurrent cytogenetic abnormalities have been identified, including t(8;21), inv(16), t(15;17), and mutations in genes such as FLT3, NPM1, CEBPA, IDH1, IDH2, and TP53. Treatment typically involves intensive induction chemotherapy (commonly a combination of cytarabine and an anthracycline), followed by consolidation therapy, which may include additional chemotherapy cycles or allogeneic hematopoietic stem cell transplantation depending on risk stratification. Targeted therapies have expanded the treatment landscape significantly, including FLT3 inhibitors (midostaurin, gilteritinib), IDH inhibitors (ivosidenib, enasidenib), the BCL-2 inhibitor venetoclax (often combined with hypomethylating agents for older or unfit patients), and all-trans retinoic acid (ATRA) with arsenic trioxide for the acute promyelocytic leukemia subtype. Despite advances, AML remains a serious condition with variable prognosis depending on patient age, overall health, and the specific genetic profile of the leukemia.

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Common questions about Acute myeloid leukemia