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AML Treatment and Therapy Options

What This Article Covers:

Adult acute myeloid leukemia (AML) is a cancer that causes the bone marrow to produce a large number of abnormal blood cells that then affect the functioning of the body. AML is the most common type of leukemia in adults and will usually get worse quickly if not treated because it is a fast-growing cancer. Acute Myeloid Leukemia affects the body’s blood-forming tissues including red blood cells, white blood cells and platelets.  AML causes the body to produce abnormal versions of these vital blood cells thus leaving the patient feeling tired, prone to bleeding or bruising and experiencing other symptoms of AML.  Depending on certain prognosis factors, a variety of treatment options are available for AML patients and should be discussed with your doctor after diagnosis.

A simple blood test will be used to confirm a suspected AML diagnosis, but the final diagnosis is usually made after a bone marrow test and sometimes by a spinal tap, also known as a lumbar (lower spine) puncture (LP).  While an AML diagnosis is devastating, several treatment options help to alleviate symptoms and allow patients to live a long and healthy life. The treatment of AML and other leukemia cancers relies not only on health care providers but also the patient’s friends and family to provide a support team dedicated to treating both physical and emotional symptoms that arise during treatment.  

Some of the following treatments are standard treatments for AML that are actively used, while other treatments, like Sierra Trial’s 131I apamistamab (Iomab-B), are still being studied in a clinical trial and evaluated for efficacy.  Iomab-B is an investigational drug (not yet approved by the FDA, recently completed enrollment) that targets bone marrow and blood cancer (leukemia) cells. It is used to help patients get ready for bone marrow transplant (BMT) also known as hematopoietic stem cell transplantation (HSCT). A bone marrow transplant remains an important, potentially curative option for adult patients with acute myeloid leukemia.


Chemotherapy for AML Treatment

doctor and patient

Chemotherapy is a common cancer treatment that uses anti-cancer drugs to kill cancer cells throughout the body.  Because AML is a type of cancer, chemotherapy is an often-used treatment option.  Chemotherapy drugs can be taken by mouth, intravenously through an IV, or as an injection under the skin.  The chemo drugs then enter the bloodstream and can reach cancer cells throughout the body, killing these cancer cells or stopping them from dividing.  This is sometimes referred to as systemic chemotherapy since it treats the whole body system.

In cases where the AML cancer has spread to the brain or spine, chemo is administered through the cerebrospinal fluid (CSF).   The drugs will not enter the bloodstream but rather will be a more localized treatment option, and is often referred to as regional chemotherapy.  

Chemotherapy is the main treatment for most people with acute myeloid leukemia (AML). Intense chemo might not be recommended for patients in poor health, but advanced age by itself is not a barrier to getting chemo.

The administration of the chemotherapy and type of chemotherapy drug used depends on the subtype of AML being treated and whether leukemia cells have spread to the brain and spinal cord as part of the AML disease progression.


There are two phases to Leukemia Treatment Options:

Remission Induction Therapy for AML

The goal of remission induction therapy is to put the leukemia into complete remission.  This is accomplished by killing the leukemia blasts circulating in the blood and reducing the leukemia blasts in the bone marrow to normal numbers. Induction is typically the first phase of a treatment plan, and is a short and intense phase of treatment for leukemia.  Patients receive high doses of chemotherapy that target the unhealthy leukemia cells.  Often targeted drugs (including but not limited to: cytarabine, daunorubicin, idarubicin, and hydroxyurea) are used in this initial treatment phase as well.  

Some older adults may not be able to have induction therapy with the standard drugs. 

Within four to six weeks of induction therapy your body should begin to produce healthy blood cells as a result of treatment.  Your doctor will perform tests to diagnose if any leukemia cells are left in your blood.  If no signs of leukemia are present in the bone marrow, blood counts are normal, and the patient is no longer showing signs and symptoms of acute myeloid leukemia, then the patient is “in remission,” and the next phase of treatment for AML can begin.  

Post-Remission Therapy for AML

The goal of post remission therapy is to kill any remaining leukemia cells.  These leukemia cells may no longer be active, but present a danger of relapse if they begin to regrow after treatment.  This phase is sometimes called post-remission consolidation.  During treatment, chemotherapy is given in cycles, with short recovery periods between each cycle.  This gives the body a chance to recover.  For some patients a bone marrow transplant / stem cell transplant is recommended for this second phase of AML treatment.  

Side Effects of Chemotherapy Treatments

Chemotherapy kills quickly dividing cells in the body, of which, cancer is one.  Unfortunately, other cells in the body divide quickly including immune cells, cells that line the mouth and intestines and your hair follicles.  Chemotherapy can also damage some of these healthy cells and this is the source of many of the side effects patients experience with chemotherapy treatments.  

Side effects of treatment can include:

Chemo drugs also affect the normal cells in bone marrow that divide quickly.  This can lower the count of white blood cells, red blood cells and platelets.

Side effects from low blood cell counts include:

While the majority of these side effects go away once treatment stops, chemotherapy may also affect the patient’s fertility, or ability to have a child in the future. Patients concerned about this are encouraged to talk with their doctor before treatment begins.


Bone Marrow Transplant or Stem Cell Transplant

Chemotherapy to treat acute myeloid leukemia is sometimes limited by the severe side effects the treatment causes.  Higher doses of the chemotherapy drugs might kill more cancer cells, however, they can’t be used because of the severe damage they would cause to the bone marrow and the problems associated with the low blood count the high dose treatment would cause. 

A bone marrow transplant is often used in conjunction with chemotherapy to help restore the patient’s health and allow higher doses of chemotherapy to be used to fight the cancer cells.  Chemotherapy often affects both the healthy and diseased cells of the bone marrow, and as such, an infusion of healthy cells is sometimes needed.  These healthy stem cells will then grow into new, healthy blood cells. These blood-forming stem cells for the stem cell transplant can come either from blood or from bone marrow. Sometimes stem cells from a baby’s umbilical cord blood are used as a treatment option. 

There are two common classifications of bone marrow transplants:

Allogeneic Bone Marrow Transplant

Allogeneic bone marrow transplants use stem cells from a donor.  The donor can be related or unrelated but must share a similar genetic makeup with the patient. 

Autologous Bone Marrow Transplant

In an autologous stem cell transplant the patient is able to donate stem cells from their own body.  Stem cells are taken from the patient, frozen, treated and then implanted back in the patient.  This AML treatment option is used if there is not a viable donor with a similar genetic makeup.

For most patients with AML, especially those at higher risk of having the leukemia return after treatment, using an allogeneic stem cell transplant is preferred over an autologous stem cell transplant. Leukemia is a disease that originates in the blood and marrow.  When the patient diagnosed with AML receives their own cells back after treatment, they may receive some leukemia cells back as well.

Donor cells are also sometimes preferred because of the graft-versus-leukemia effect. When the donor immune cells are infused into the body, the immune cells may recognize any remaining leukemia cells as being foreign to them and attack them. This effect doesn’t happen with autologous stem cell transplants. 

Unfortunately, there is also a similar negative effect known as graft-versus-host disease that occurs when the patient’s immune system begins to reject the new stem cells and attacks them.  Both concerns should be discussed with your healthcare provider.

Complications from bone marrow transplantation include:


Radiation Therapy for Treatment of AML (Acute Myeloid Leukemia)

Radiation therapy is not a mainstream treatment for AML cancers, however, radiation therapy can be used to kill cancer cells and is occasionally used as an  acute myeloid leukemia treatment option.  The most common use of external-beam radiation therapy is when the leukemia cancer has spread to the brain, spinal fluid or other localized area as AML progresses.

In radiation therapy high-energy x-rays or other types of radiation are directed toward the area of the body that has the cancer to keep the cancer cells from growing or to kill the cancer cells.  Total-body irradiation therapy is sometimes used on the whole body to prepare the body for a stem cell transplant if the leukemia has recurred.

Radiation therapy can also be used to reduce pain in an area of bone that has been overtaken by leukemia if chemotherapy treatments do not eradicate the leukemia cells in patients.

Complications from radiation therapy treatment include:


Targeted Drug Therapy for an Acute Myeloid Leukemia Diagnosis

Targeted drug therapy is a promising new AML treatment that target’s the specific genes, proteins or tissue environment of leukemia cells.  This type of treatment can hone in on leukemia cells, blocking their growth and spread, but limits the damage to healthy cells in the body.  These drugs can be used both as a stand alone leukemia treatment or in conjunction with chemotherapy.  Several drugs are currently being used to treat acute myeloid leukemia and acute myelogenous leukemia.

FLT3 Inhibitors

Some AML patients exhibit a mutation of the FLT3 gene that helps the body make the FLT3 protein.  By blocking FLT3 (and other proteins on cancer cells), the cancer’s growth can be slowed.  A blood test for AML reveals whether you have this mutation and thus, would respond to an FLT3 Inhibitor.

IDH Inhibitors

Some AML patients exhibit a mutation of the IDH1 or IDH2 genes.  These genes are associated with the IDH1 and IDH2 proteins that help cells to mature.  An IDH Inhibitor can suppress these proteins which enables the leukemia cells to mature into more normal blood and bone marrow cells thus reducing the symptoms of AML for patients with this mutation.

Monoclonal antibodies

Monoclonal antibodies are lab created immune proteins that link to a specific target on cancer cells or other cells that facilitate the growth of cancer.  The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading.  These drugs can be used as a stand alone treatment or in conjunction with chemotherapy and are often used as a first line defense against leukemia for AML patients who are not healthy enough for a chemotherapy treatment.

Gemtuzumab ozogamicin is a type of monoclonal antibody that is used to treat patients with a new AML diagnosis or who have relapsed from previous treatments. It binds to a protein called CD33.  The antibody then helps direct the chemo drug to the leukemia cells, allowing for a much more targeted treatment.

See Drugs Approved for Acute Myeloid Leukemia for more treatment options and drug information.


Surgery for Acute Myeloid Leukemia Cancer

Because leukemia cells are spread widely throughout the bone marrow and blood, surgery is not usually a viable treatment option for AML patients. On rare occasions, an isolated tumor of leukemia cells may be treated with surgery.

A minor surgery to place a central venous catheter into a vein on the patient’s chest is a typical part of a chemotherapy program.  This catheter is typically left in place over a number of months and allows doctors to give intravenous chemotherapy drugs to the patient and to easily take blood samples for ongoing diagnostic tests.  An important part of chemotherapy treatment includes learning how to care for this CVC to prevent infection.

Maintenance and Follow Up Testing

The goal of all AML treatment options is complete remission of the cancer and a return to normal bone marrow and blood cell counts.  Most doctors look for a complete molecular revision that is tested by using sensitive lab tests to look for any signs of leukemia. Ongoing testing that checks for any signs of AML are also common even after a patient achieves remission.  Even after completion of post-remission consolidation treatments, there is a risk that AML can still return. Sometimes lower strength medications are given for several years to reduce the chance of the disease returning, called recurrence. 


Clinical Trials for AML

AML treatment clinical trials represent a unique opportunity for AML patients not eligible for other treatment options.  Clinical trials are closely monitored to help protect the rights of patients and safety of the people who participate while evaluating the effectiveness of new cancer treatment options.  Patients can help move cancer research forward and change how cancer is treated in the future.  Many of today’s standard treatments have come out of previous clinical trials and the information gained from the trials will expand the knowledge of how patient health is affected by AML and treatment options.Currently, Sierra Trial is evaluating the use of 131I apamistamab (Iomab-B) as a targeted therapy for AML, consisting of a monoclonal antibody that is connected to radioactive material (making it a radiolabeled antibody). 131I apamistamab (Iomab-B) delivers radiation preferentially to your bone marrow and spleen, where most leukemia cells are, when compared to other organs such as liver, lungs, kidneys and mucous membranes that can be harmed by too much radiation.

Key Eligibility Criteria


Criteria for Determining AML Treatment Options

Deciding on a treatment option for your AML diagnosis is an important decision that should be made in conjunction with your doctor and health care team.  The treatment options can depend heavily on the subtype of AML being treated as well as important individual health factors.  The following are typical treatment options depending on the stage of your AML journey.

Newly Diagnosed / Previously Untreated AML

A new AML diagnosis is the most typical type of untreated AML.  No therapies have been used to treat the disease or relieve the signs and symptoms of AML.  Patients typically have the following signs:

Treatment options can thus include combination chemotherapy, FLT3 Inhibitors, monoclonal antibodies, intrathecal (administered to the spine) chemotherapy, targeted therapy, low-dose chemotherapy or a combination of these.

AML in Remission

The remission stage is achieved after or during treatment when blood cell counts return to normal, a patient is symptom free, and their bone marrow contains fewer than 5% leukemia blasts.1 Remission can be affected by a person’s age, the type of AML and whether certain genes or chromosomes are mutated or present in the leukemia cells.

Patients typically have the following signs:

Treatment options can thus include combination chemotherapy, maintenance therapy with FLT3 Inhibitors or chemotherapy, high-dose chemotherapy, stem cell transplants, radiation therapy or a combination of these.

Refractory or Recurrent AML

Some patients with newly diagnosed AML will not go into remission even after chemotherapy or other treatments. This is called refractory cancer. Recurrent AML, on the other hand, is cancer that has come back after remission. The AML may come back in the blood or bone marrow and is typically found in the follow up testing phase of treatment.

Treatment options are typically more aggressive and may include chemotherapy, targeted therapy, stem cell transplants, monoclonal antibodies or a combination of these.

For all of these treatments, patient education is of premium importance and your doctor should review how each type of treatment could affect your overall health and well-being before proceeding with a treatment option.  Always ask for more information on side effects, clinical research and after treatment care that you think will affect your health from your doctor so that you understand the basics of any treatment plan.  A healthy living program can be a valuable asset to any treatment plan as well and always factors into overall patient health.

1. https://www.medicalnewstoday.com/