Acute myeloid leukemia

Acute myeloid leukemia (AML) is a cancer of the myeloid line of blood cells, characterized by the rapid growth of abnormal cells that build up in the bone marrow and blood and interfere with normal blood cell production. Symptoms may include feeling tired, shortness of breath, easy bruising and bleeding, and increased risk of infection. Occasionally, spread may occur to the brain, skin, or gums. As an acute leukemia, AML progresses rapidly, and is typically fatal within weeks or months if left untreated.

Risk factors include getting older, being male, smoking, previous chemotherapy or radiation therapy, myelodysplastic syndrome, and exposure to the chemical benzene. The underlying mechanism involves replacement of normal bone marrow with leukemia cells, which results in a drop in red blood cells, platelets, and normal white blood cells. Diagnosis is generally based on bone marrow aspiration and specific blood tests. AML has several subtypes for which treatments and outcomes may vary.

The first-line treatment of AML is usually chemotherapy, with the aim of inducing remission. People may then go on to receive additional chemotherapy, radiation therapy, or a stem cell transplant. The specific genetic mutations present within the cancer cells may guide therapy, as well as determine how long that person is likely to survive.

Since 2017, 12 new agents have been approved for AML in the U.S., including venetoclax (BCL2 inhibitor), gemtuzumab ozogamicin (CD33 antibody-drug conjugate), and several inhibitors targeting FMS-like tyrosine kinase 3, isocitrate dehydrogenase, and other pathways. Additionally, therapies like CPX351 and oral formulations of azacitidine and decitabine-cedazuridine have been introduced. Ongoing research is exploring menin inhibitors and other antibody-drug conjugates.

Low-intensity treatment with azacitidine plus venetoclax has emerged as the most effective option for older or unfit AML patients, based on a network meta-analysis of 26 trials involving 4,920 participants. It showed the highest survival and remission rates, with low-dose cytarabine (LDAC) plus glasdegib and LDAC plus venetoclax also showing clinical benefit.

In 2015, AML affected about one million people, and resulted in 147,000 deaths globally. It most commonly occurs in older adults. Males are affected more often than females. The five-year survival rate is about 35% in people under 60 years old and 10% in people over 60 years old. Older people whose health is too poor for intensive chemotherapy have a typical survival of five to ten months. It accounts for roughly 1.1% of all cancer cases, and 1.9% of cancer deaths in the United States.