Receiving a diagnosis of Acute Myeloid Leukemia (AML) often happens suddenly, turning normal life upside down in a matter of days. Symptoms like extreme fatigue, unexplained bruising, or persistent infections can make daily routines difficult and frightening. Because AML is a fast-growing cancer of the blood and bone marrow, it requires prompt attention to restore healthy blood cell production. While the diagnosis is serious, advancements in medical science have expanded the toolkit for managing this condition.

Treatment is essential to halt the rapid production of abnormal leukemia cells that crowd out healthy blood cells. Without intervention, the body cannot fight infections, carry oxygen, or control bleeding. The specific treatment plan varies significantly from person to person. Doctors tailor the approach based on the patient’s age, overall fitness, and the specific genetic mutations found within the leukemia cells (National Cancer Institute, 2023).

Overview of treatment options for Acute Myeloid Leukemia

The primary goal of AML treatment is to achieve remission, meaning no evidence of leukemia is left in the bone marrow. Treatment typically follows two distinct phases: induction and consolidation. Induction therapy aims to clear the blood of visible leukemia cells and return the bone marrow to normal working order. Once remission is achieved, consolidation therapy (or post-remission therapy) is used to destroy any remaining invisible cells to prevent the cancer from returning.

While stem cell transplants are a critical procedure for many eligible patients, medication, specifically chemotherapy and targeted therapy, forms the foundation of treatment. In some cases, lower-intensity medication regimens are used for patients who may not tolerate harsh chemotherapy due to age or other health conditions.

Medications used for Acute Myeloid Leukemia

Chemotherapy remains the standard first-line treatment for most cases of AML. The most common induction regimen involves a combination of two types of drugs. This usually includes a continuous infusion of cytarabine for seven days, paired with a short course of an anthracycline drug, such as daunorubicin or idarubicin. Clinical experience suggests that this combination clears visible leukemia cells in a significant percentage of patients after the first cycle.

In recent years, targeted therapies have become standard for patients with specific genetic mutations. For example, if the leukemia cells carry an FLT3 mutation, doctors may add drugs like midostaurin or gilteritinib to the chemotherapy regimen. For mutations in the IDH1 or IDH2 genes, medications like ivosidenib or enasidenib may be used.

Another important class includes BCL-2 inhibitors, such as venetoclax. This medication is often combined with low-dose chemotherapy or hypomethylating agents (like azacitidine) for older adults or those who cannot undergo intensive chemotherapy. For a specific subtype called Acute Promyelocytic Leukemia (APL), treatment relies on differentiation agents like all-trans retinoic acid (ATRA) and arsenic trioxide, which are highly effective (Leukemia & Lymphoma Society, 2023).

How these medications work

Chemotherapy drugs target rapidly dividing cells’ DNA, damaging them. Leukemia cells are sensitive because they divide fast, but this also harms healthy fast-growing cells like those in hair follicles and the mouth lining.

Targeted therapies work differently, “locking on” to specific cancer cell proteins. For example, FLT3 inhibitors block an enzyme promoting leukemia cell growth. BCL-2 inhibitors block a survival protein, causing cells to self-destruct. Differentiation agents, used in APL, prompt immature leukemia cells to mature and die naturally, rather than killing them directly (American Cancer Society, 2024).

Side effects and safety considerations

AML treatments have significant side effects. Chemotherapy suppresses bone marrow, causing low blood counts, which results in a high risk of infection (neutropenia), fatigue (anemia), and easy bleeding (thrombocytopenia). Patients often need blood transfusions and preventative antibiotics. Other common effects are nausea, hair loss, and mouth sores.

Targeted therapies have unique risks. FLT3 inhibitors can cause GI issues, and differentiation agents may lead to differentiation syndrome (fluid buildup and fever). Anthracyclines require heart monitoring due to potential cardiac effects. Since the immune system is severely compromised, fever is a medical emergency, requiring immediate care due to the risk of rapid infection progression.

Since everyone’s experience with the condition and its treatments can vary, working closely with a qualified healthcare provider helps ensure safe and effective care.

References

1. American Cancer Society. https://www.cancer.org
2. Leukemia & Lymphoma Society. https://www.lls.org
3. National Cancer Institute. https://www.cancer.gov
4. Mayo Clinic. https://www.mayoclinic.org