Treatment for acute myeloid leukemia (AML) typically begins with intensive chemotherapy designed to wipe out leukemia cells in the bone marrow, followed by additional therapy to keep the disease from returning. The specific approach depends on several factors: your age, overall health, and the genetic makeup of your leukemia cells. Most people with AML go through two main phases of treatment, and some will need a stem cell transplant.
Induction: The First Phase
The immediate goal of treatment is to achieve remission, meaning leukemia cells are no longer detectable in the bone marrow. This first phase is called induction therapy. For younger, otherwise healthy patients, the standard approach is known as the “7+3” regimen: seven continuous days of one chemotherapy drug (cytarabine) paired with a second drug (an anthracycline) given during the first three days. This combination is delivered intravenously and requires a hospital stay.
Expect to be in the hospital for roughly four to six weeks during induction. The chemotherapy itself lasts about a week, but it destroys both leukemia cells and healthy blood cells, leaving your immune system severely weakened. You’ll stay in the hospital while your blood counts recover, receiving transfusions and medications to prevent infections during that vulnerable window. Antibacterial, antifungal, and antiviral drugs are routinely given to patients whose white blood cell counts drop very low for more than a week.
If your leukemia cells carry certain genetic changes, targeted drugs may be added to chemotherapy. For example, patients with an FLT3 gene mutation often receive an oral drug that blocks the protein produced by that mutation. Similarly, if the leukemia cells display a specific surface marker called CD33, a targeted drug that delivers chemotherapy directly to those cells can be combined with the standard regimen. These additions improve the odds of achieving remission.
People with heart problems that make anthracycline drugs unsafe are treated with alternative chemotherapy combinations instead.
Consolidation: Preventing Relapse
Reaching remission is a critical milestone, but it’s not the end of treatment. Without further therapy, AML almost always comes back. Consolidation therapy aims to destroy any remaining leukemia cells that survived induction.
The most common consolidation approach is high-dose cytarabine, the same drug used during induction but at much higher doses. A typical schedule involves infusions over two to three hours, given twice daily for three consecutive days. This cycle repeats every 28 days, for up to four cycles total. Each cycle again causes a period of low blood counts, though hospital stays during consolidation tend to be shorter than during induction.
For patients at higher risk of relapse based on their leukemia’s genetic profile, doctors often recommend a stem cell transplant (also called a bone marrow transplant) as consolidation instead. This involves receiving very high doses of chemotherapy or radiation to completely destroy the bone marrow, then replacing it with healthy donor stem cells. It’s more intensive and carries greater risks, but for certain genetic subtypes of AML, it offers the best chance of long-term cure.
Treatment for Older Adults
Intensive chemotherapy like the 7+3 regimen can be too harsh for older adults or people with significant health problems. For these patients, a lower-intensity combination has become standard: a pill that blocks a protein leukemia cells rely on to survive, paired with an injectable drug that reprograms how genes are expressed in cancer cells. This combination is given in monthly cycles, typically starting with about 25 days of the oral medication per cycle.
This lower-intensity approach has shown strong results. In certain AML subtypes, nearly all newly diagnosed patients achieve remission after the first cycle. The treatment is largely outpatient, which means far less time in the hospital compared to intensive chemotherapy. Side effects still include low blood counts and infection risk, but they’re generally more manageable.
A Special Subtype: Acute Promyelocytic Leukemia
One subtype of AML, called acute promyelocytic leukemia (APL), responds to a fundamentally different treatment. Instead of relying on standard chemotherapy, APL is treated with two agents: a vitamin A derivative that forces immature leukemia cells to mature into normal blood cells, and arsenic trioxide, which destroys APL cells through a separate mechanism.
For patients with lower white blood cell counts at diagnosis, this two-drug combination alone is used for both induction and consolidation, with no conventional chemotherapy needed. Patients with higher white blood cell counts (above 10,000 per microliter) typically receive one of these agents along with a single chemotherapy drug or a targeted therapy during induction, then continue with the non-chemotherapy combination for consolidation. APL has gone from being one of the most dangerous forms of AML to one of the most curable, largely because of this approach.
When AML Comes Back or Doesn’t Respond
If AML returns after remission or doesn’t respond to initial treatment, the situation is more challenging. There is no single preferred regimen for relapsed or refractory AML. Treatment typically involves a different combination of chemotherapy drugs than what was used initially, with the goal of achieving remission a second time and then proceeding to a stem cell transplant. A transplant using donor cells remains the only reliable path to cure in this setting.
Two new drugs approved in late 2025 have expanded options for a specific group of relapsed patients. Both are menin inhibitors, a new class of targeted therapy for patients whose leukemia carries an NPM1 mutation. These oral medications work by disrupting a protein interaction that drives leukemia cell growth in this genetic subtype. They’re approved for adults (and in one case, children as young as one year old) who have already tried other treatments without success.
Clinical trials are strongly encouraged for patients with relapsed or refractory AML, since standard salvage chemotherapy produces one-year survival rates that remain poor. Trials offer access to newer drug classes and combination strategies that may improve outcomes beyond what existing regimens can offer.
What Genetic Testing Means for Your Treatment
One of the first things that happens after an AML diagnosis is genetic testing of the leukemia cells. This isn’t about your inherited DNA. It’s about mutations and chromosomal changes that occurred specifically in the cancer cells. These results directly shape which drugs you’ll receive, whether a transplant is recommended, and how aggressively doctors pursue consolidation.
Certain mutations, like changes in the FLT3 or NPM1 genes, have specific drugs designed to target them. The chromosomal rearrangement that defines APL triggers an entirely different treatment protocol. Other genetic patterns help classify your leukemia as favorable, intermediate, or adverse risk, which determines whether consolidation chemotherapy alone is sufficient or whether a stem cell transplant is the better option. Genetic testing results sometimes take a week or more to finalize, but they are essential for tailoring treatment to your specific disease.
Supportive Care During Treatment
AML treatment temporarily shuts down normal bone marrow function, which means your body can’t produce enough red blood cells, platelets, or infection-fighting white blood cells on its own. Transfusions of red blood cells and platelets are routine throughout treatment. You’ll likely receive preventive antibiotics and antifungal medications during periods when your white blood cell count is critically low, typically defined as fewer than 100 infection-fighting cells per microliter for more than a week.
Patients who have previously been exposed to herpes simplex virus receive antiviral medication during induction to prevent reactivation. Those at risk for hepatitis B reactivation also receive preventive treatment. Nausea, fatigue, mouth sores, and appetite loss are common side effects during both induction and consolidation. Hospital teams include nutritionists, social workers, and palliative care specialists who help manage symptoms and quality of life throughout the process.