Acute myeloid leukemia (AML) is a fast-growing cancer that begins in the blood-forming cells of the bone marrow. Because AML can progress quickly, treatment often needs to begin promptly after diagnosis. The treatment plan depends on several factors,
including the AML subtype, genetic and molecular characteristics of the leukemia, age, overall health, previous treatment, and the person's preferences.
For patients and families in the United States, understanding the available AML treatment options can make conversations with a hematologist or oncologist easier. Treatment may involve chemotherapy, targeted therapy, stem cell transplantation, supportive care, or clinical trials.
What Is AML Treatment?
AML treatment is designed to reduce or eliminate leukemia cells and restore healthy blood-cell production. Treatment is commonly organized into different phases, although the exact approach varies between patients.
For many adults, treatment includes remission induction followed by consolidation. Maintenance treatment may also be used in certain situations. Acute promyelocytic leukemia (APL), a specific AML subtype, is treated differently from most other forms of AML.
How Doctors Choose an AML Treatment
There is no single treatment plan that applies to every AML patient. Doctors evaluate the disease and the patient's overall health before recommending therapy.
Important factors may include:
- AML subtype
- Genetic and chromosome changes
- Age
- Overall health
- Previous cancer treatment
- Risk of relapse
- Ability to tolerate intensive treatment
- Patient preferences
Genetic and molecular testing can be particularly important because certain AML mutations may make targeted treatments appropriate.
Chemotherapy for AML
Chemotherapy remains a major component of treatment for most types of AML. It uses medicines that destroy or control rapidly growing leukemia cells.
Remission Induction
The first phase is usually called remission induction. Its goal is to reduce leukemia cells in the blood and bone marrow and achieve remission.
For some adults who can tolerate intensive treatment, a combination involving cytarabine and an anthracycline may be used. This approach is commonly known as a "7+3" regimen. Depending on the leukemia's characteristics, a targeted medicine may also be incorporated.
Intensive treatment can significantly reduce normal blood-cell counts, which can increase the risk of infections and bleeding. Patients may require close monitoring, transfusions, and medicines to prevent or treat complications.
Consolidation Treatment
If remission is achieved, additional treatment may be recommended to reduce the risk of leukemia returning.
Consolidation may involve additional chemotherapy or a stem cell transplant, depending on the patient's relapse risk and other factors.
Targeted Therapy for AML
Targeted therapies work on specific proteins or genetic changes found in some leukemia cells. Testing can help doctors determine whether a patient has characteristics that may make a targeted medicine appropriate.
For example, FLT3 inhibitors may be used for AML with certain FLT3 mutations. IDH inhibitors may be considered when AML cells contain IDH1 or IDH2 mutations. Other targeted medicines may be used when specific proteins or molecular changes are identified.
Targeted therapy may be used alone or together with chemotherapy, depending on the patient's diagnosis and treatment plan.
Less-Intensive Treatment
Not every patient can tolerate intensive chemotherapy. Older adults or people with other significant health conditions may receive less-intensive approaches.
Options can include medicines such as azacitidine, decitabine, low-dose cytarabine, or combinations involving targeted drugs such as venetoclax. The appropriate approach depends on overall health and the characteristics of the leukemia.
Age alone does not determine whether someone can receive intensive treatment. Doctors consider overall health, functional status, and other medical factors.
Stem Cell Transplant
A stem cell transplant, also called a bone marrow transplant, may be considered for selected AML patients.
The procedure involves intensive treatment followed by infusion of blood-forming stem cells. An allogeneic transplant uses stem cells from a donor, while an autologous transplant uses the patient's own stem cells.
Transplantation can involve significant risks, so doctors carefully evaluate factors such as relapse risk, overall health, donor availability, and the potential benefits and complications.
Treatment for APL
Acute promyelocytic leukemia (APL) is a distinct subtype of AML and generally requires a different treatment approach. Medicines such as all-trans-retinoic acid (ATRA) and arsenic trioxide are important components of treatment for many people with APL.
Because APL can be associated with serious bleeding and clotting complications, prompt medical management is particularly important.
AML Treatment Costs in the United States
The cost of AML treatment can vary substantially from one patient to another. Expenses may include hospitalization, chemotherapy, targeted medicines, laboratory testing, imaging, transfusions, specialist consultations, and stem cell transplantation.
Insurance coverage can also significantly affect out-of-pocket costs.
Patients and families may want to ask their healthcare team and insurance provider about:
- Deductibles
- Coinsurance
- Out-of-pocket maximums
- Prescription coverage
- Hospital costs
- Financial assistance programs
- Clinical trial-related expenses
Cost should be considered alongside medical suitability rather than used as the only factor when evaluating treatment.
Clinical Trials
Clinical trials are an important part of AML research. Studies may investigate new medicines, combinations, targeted treatments, and approaches for patients whose AML has returned or has not responded to previous therapy.
Eligibility depends on factors such as AML subtype, genetic characteristics, previous treatments, age, and overall health. Patients can ask their oncology team whether a clinical trial may be appropriate.
If AML Returns or Does Not Respond
Some AML cases do not achieve remission after initial treatment, while others return after a period of remission. This is known as refractory or recurrent AML.
Depending on the circumstances, doctors may consider additional chemotherapy, targeted therapy, stem cell transplantation, or clinical trials. Treatment decisions depend on the leukemia's characteristics and the patient's ability to tolerate further treatment.
Final Thoughts
AML treatment can involve chemotherapy, targeted therapy, stem cell transplantation, supportive care, and clinical trials. The appropriate combination depends on the AML subtype, genetic findings, overall health, previous treatment, and individual treatment goals.
For patients in the United States, comparing treatment approaches and understanding potential costs can help with discussions about care. However, AML treatment decisions should always be made with a qualified hematologist or oncologist who can evaluate the individual's diagnosis and medical circumstances.
This article is for general educational purposes only and should not replace personalized medical advice from a qualified healthcare professional.