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Acute myeloblastic leukemia (AML) in childhood is a malignant disease of the hematopoietic system, characterized by the production of abnormal, immature myeloid blood cells—myeloblasts—by the bone marrow. These blasts can spread through the bloodstream to various organs and the central nervous system (CNS). In children, AML most frequently occurs during the first two years of life or in adolescence.
The 5-year survival rate for patients with AML is lower than in acute lymphoblastic leukemia (ALL), ranging between 55–65%. In infants under one year of age, this indicator is even lower. Nevertheless, survival rates vary depending on the AML subtype and the presence of unfavorable genetic mutations.
Hereditary and acquired syndromes associated with an increased risk of AML include: Down syndrome, Fanconi anemia, Bloom syndrome, Neurofibromatosis type 1 (Noonan syndrome), congenital severe neutropenia, Diamond–Blackfan anemia, congenital amegakaryocytic thrombocytopenia, and others.
Clinical manifestations of AML include anemia (the most common syndrome, presenting with pallor, fatigue, tachycardia, and headaches), bleeding and hemorrhages, fever (frequent or unexplained), recurrent or prolonged infections, bone and joint pain, weight loss and decreased appetite, gingival hypertrophy, chloroma, and CNS involvement.
Diagnosis begins with a thorough medical history, a physical examination, a complete blood count, and a peripheral blood smear morphology. The disease is confirmed by the presence of ≥20% blast cells in peripheral blood and/or bone marrow. CNS involvement may be diagnosed or ruled out by the detection of blast cells in cerebrospinal fluid or clinically, when signs of neurological impairment are present.
Treatment approaches include chemotherapy, immunotherapy, targeted therapy, and allogeneic stem cell transplantation (SCT).
