Acute myeloid leukemia (AML) goes by many names, including acute myelocytic leukemia, acute myelogenous leukemia, acute granulocytic leukemia, and acute non-lymphocytic leukemia. "Acute" means that the leukemia can progress quickly, and if not treated, would probably be fatal in a few months.
AML is a cancer that starts in the cells that are supposed to mature into different types of blood cells. AML starts in the bone marrow (the soft inner part of the bones, where new blood cells are made), but in most cases it quickly moves into the blood. It can sometimes spread to other parts of the body including the lymph nodes, liver, spleen, central nervous system (brain and spinal cord), and testes. In contrast, other types of cancer can start in these organs and then spread to the bone marrow (or elsewhere). Those cancers are not leukemia. Both children and adults can get leukemia.
At this time, there are no special tests that can find AML early. The best course of action is to report any symptoms to the doctor right away.
Signs and Symptoms of AML
General Symptoms
Patients with AML often have several symptoms that can include weight loss, fatigue, fever, and loss of appetite. Of course, AML is not the only problem that causes these symptoms. They are most often caused by something other than cancer.
Shortage of Blood Cells
Most signs and symptoms of AML result from a shortage of normal blood cells, which happens when the leukemia cells crowd out the normal blood-making cells in the bone marrow. As a result, people do not have enough normal red blood cells, white blood cells, and blood platelets. These shortages show up on blood tests, but they can also cause symptoms.
Anemia: Anemia is a result of a shortage of red blood cells. It causes shortness of breath, fatigue, and a pale color to the skin, nails, and gums.
Shortage of white blood cells: Not having enough normal white blood cells can increase the risk of infection. Although people with leukemia may have very high white blood cell counts, the cells are usually not normal and can not protect against infection. Fevers and other signs of infection are common symptoms.
Shortage of blood platelets: Not having enough blood platelets can lead to bruising, bleeding, frequent or severe nosebleeds, and bleeding from the gums.
Bone or Joint Pain
Some people have bone pain or joint pain caused by the buildup of leukemia cells in bones or joints.
Swelling in the Belly
Leukemia can also cause swelling of the liver and spleen.
Spread to the Skin
If leukemia cells spread to the skin, they can cause lumps or spots that may look like common rashes.
Spread to the Gums
Certain types of AML is prone to spread to the gums, causing them to swell, become painful, and bleed.
Spread to Other Organs
Sometimes, leukemia cells may spread to other organs. Spread to central nervous system (brain and spinal cord) can cause headaches, weakness, seizures, vomiting, trouble with balance, facial numbness, and blurred vision. Rarely, AML may spread to the eyes, testicles, kidneys, or other organs.
Enlarged lymph nodes: In rare cases, AML may spread to lymph nodes. Nodes in the neck, groin, under arms, or above the collarbone may appear swollen.
This information represents the views of the doctors and nurses serving on the American Cancer Society's Cancer Information Database Editorial Board. These views are based on their interpretation of studies published in medical journals, as well as their own professional experience.
The treatment information in this document is not official policy of the Society and is not intended as medical advice to replace the expertise and judgment of your cancer care team. It is intended to help you and your family make informed decisions, together with your doctor.
Your doctor may have reasons for suggesting a treatment plan different from these general treatment options. Don't hesitate to ask him or her questions about your treatment options.
This section starts with general comments about types of treatments used for AML. This is followed by a discussion of the typical treatment approach for AML.
As noted before, AML is not a single disease. It is really a group of diseases, and people with different subtypes vary in how they respond to treatment. Treatment options are based on the subtype as well as on the prognostic features. Several different types of treatment may be used in people with AML. The major treatment is chemotherapy. Surgery and radiation treatment may be used in special cases.
Treatment of AML is divided into 2 phases:
induction (or remission induction)
consolidation (post-remission)
Chemotherapy
Chemotherapy (often referred to as simply, "chemo") refers to the use of drugs to kill cancer cells. Usually the drugs are given into a vein or by mouth or into the fluid around the brain and spine (CSF). Except when given into the CSF, these drugs enter the bloodstream and reach all the areas of the body. Chemotherapy for AML usually involves a combination of 2 or 3 anti-cancer drugs.
Induction
This first part of treatment is aimed at getting rid of all the leukemia that can be found. It usually involves treatment with 2 or 3 chemo drugs, This first phase destroys most of the leukemia cells as well as most of the normal bone marrow cells. The treatment usually takes place in the hospital and lasts about a week. In rare cases where the leukemia has spread to the brain or spinal cord, chemo may be given into the cerebrospinal fluid as well.
During this time and in the weeks right after, the patient's blood cell counts will be very low. The doctor will take measures to protect against problems. If one week of treatment fails to bring about a remission, the process is repeated once or twice more. The success rate for induction varies widely and depends on each person's own case.
Induction usually does not destroy all the leukemia cells, and a small number often persist. Without more treatment, called consolidation, the leukemia is likely to return within several months.
Consolidation (Post-Remission) Therapy
While the first phase of treatment usually destroys nearly all of the cancer cells, there may still be some "hidden" leukemic cells. The purpose of the second phase is to destroy these cells and prevent a relapse. The options for post-remission treatment are either more chemo or a stem cell transplant, described later in this article. Four years after this treatment, 40% of young patients (younger than 60 years) will not show any signs of leukemia. In older adults, this number is around 15%. Doctors look at several factors when recommending what type of post-remission treatment might be best for a patient. These factors include:
how much chemotherapy it took to bring about a remission
whether there is a stem cell donor (brother, sister, or unrelated donor) who matches the patient’s tissue type
whether it looks as if leukemia-free bone marrow stem cells can be collected from the patient
whether the prognostic factors are good or poor
the patient’s age
what the patient prefers
Side Effects of Chemotherapy
Chemo drugs work by attacking cells that are dividing quickly, which is why they work against cancer cells. But other cells in the body, such as those in the bone marrow, the lining of the mouth and intestines, and the hair follicles, also divide quickly. These cells are also likely to be affected by chemo, which can lead to side effects.
The side effects of chemo depend on the type and dose of drugs given and how long they are taken. These side effects may include:
hair loss
mouth sores
loss of appetite
nausea and vomiting
greater chance of infection (due to a shortage of white blood cells)
easy bruising or bleeding (due to a shortage of blood platelets)
tiredness (due to a shortage of red blood cells)
These side effects usually go away not long after treatment ends. There are often ways to manage these side effects during treatment. For example, there are drugs than can be taken along with the chemotherapy to prevent or reduce nausea and vomiting. Drugs known as growth factors are sometimes given to keep blood counts higher and reduce the chance of infection.
Other ways you can reduce the risk of infection are by avoiding exposure to germs as much as possible and by carefully washing your hands and not eating raw fruits and vegetables. While in treatment you should also avoid large crowds and people who are sick. It’s best to also avoid fresh flowers and plants because they may carry germs.
During and after treatment, you might also get antibiotics as added protection against infections. If your platelet counts are low, you might get platelet transfusions to protect against bleeding. Low red blood cell counts, causing shortness of breath and tiredness, can be treated with drugs or with transfusions.
Tumor lysis syndrome is a side effect caused by the rapid breakdown of leukemia cells during treatment. When these cells die, they release their contents into the bloodstream. These chemicals can affect the kidneys, heart, and nervous system. Extra fluids or certain drugs that help rid the body of these substances can help prevent this problem.
Organs that could be damaged by chemotherapy include the kidneys, liver, testes, ovaries, brain, heart, and lungs. By watching the patient carefully, the doctor may be able to prevent many of these side effects. If serious side effects happen, though, the drugs may have to be reduced or stopped. Be sure to tell your doctor about any problems you have.
Monoclonal Antibodies
Monoclonal antibodies are man-made version of immune system that are designed to attach to certain targets on the surface of AML cells. Some of these antibodies have radioactive chemicals or cell poisons attached to them so that when they are injected into the patient, they lock onto the cancer cells and kill them. One such antibody (Mylotarg) has recently been approved for use in older adults with AML who have relapsed after treatment or who might not be able to take the side effects of further chemotherapy. Studies are still going on to look at the best way to use this drug.
Bone Marrow or Peripheral Blood Stem Cell Transplant
While very high doses of chemotherapy drugs might work better to kill cancer cells, the damage to the bone marrow could be fatal.. A stem cell transplant (SCT) offers a way for doctors to use high doses of chemotherapy. Although the drugs destroy the patient's bone marrow, the transplanted stem cells can restore it.
Stem cells for a transplant come from either from the blood or from the bone marrow Bone marrow transplants were more common in the past, but they have has largely been replaced by peripheral blood stem cell transplant (PBSCT). The stem cells can come from either the patient or from a matched donor. There is a good reason to use stem cells from someone else for the transplant. These cells seem to help fight any remaining leukemia cells through an immune reaction. This is called a "graft-versus-leukemia" reaction.
The Transplant Process
The treatment works like this: stem cells for are collected from the bloodstream in a process called apheresis. The cells are frozen and stored. Patients are then given very high doses of chemotherapy to kill the cancer cells. They also receive total body radiation to kill any remaining cancer cells. After treatment, the stored stem cells are given to the patient as a blood transfusion. The stem cells settle into the patient's bone marrow over the next several days and start to grow and make new blood cells.
People who receive a donor's stem cells are given drugs to prevent rejection as well as other medicines as needed to prevent infections. Usually within a couple of weeks after the stem cells are given, they start making new white blood cells. Then they begin making platelets, and finally, red blood cells.
Patients having SCT have to be kept away from germs as much as possible until their white blood cell count is at a safe level. They are kept in the hospital until the white cell count reaches a certain number, usually around 1,000. After they go home, they will be seen in the outpatient clinic almost every day for several weeks.
Some Things to Keep in Mind
Stem cell transplantation is a complex treatment. If the doctors think that a person with leukemia might be helped by this treatment, it is important that it be done at a hospital where the staff has experience with the procedure. Some transplant programs may not have experience in certain transplants, especially those from unrelated donors.
Stem cell transplantation is very expensive (more than $100,000) and involves a long hospital stay. Because some insurance companies see it as an experimental treatment, they might not pay for it. It is important to find out what your insurer will cover and what you might have to pay before deciding on a transplant.
Side Effects of Stem Cell Transplant
Side effects from stem cell transplantation can be divided into early and long-term effects. The early side effects are basically the same as those caused by any other type of high-dose chemotherapy. One of the most common and serious short-term effects is the greater risk of infection. Antibiotics are often given to try to prevent these infections. Other side effects, like low red blood cell and platelet counts, may mean the patient will need transfusions.
Long-term Side Effects
Some side effects can last for a long time, and may not happen until years after the transplant. These long-term side effects can include the following:
graft-versus-host disease (GVHD), which occurs only in a donor transplant (see below)
radiation damage to the lungs, causing shortness of breath
damage to the ovaries causing infertility and the loss of menstrual periods
damage to the thyroid gland that causes problems with changing food into energy
cataracts (damage to the eye that can affect vision)
bone damage (if damage is severe, the patient will need to have part of the bone and joint replaced.)
Graft-versus-host disease is the main problem of a donor stem cell transplant. It happens when the immune system of the patient is taken over by that of the donor. The donor immune system then starts to attack the patient’s other tissues and organs.
Symptoms can include severe skin rashes with itching and severe diarrhea. The liver and lungs may also be damaged. The patient may also become tired and have aching muscles. If severe enough, the disease can be fatal. Drugs that weaken the immune system may be given to try to control it. The plus side of graft-versus-host disease is that the donor bone marrow usually kills any remaining leukemia cells. This is called the "graft-versus-leukemia" effect.
"Mini Transplant"
Most patients over the age of 55 can't have a regular transplant that uses high doses of chemo. Some may be able to have what is called a "mini-transplant" (non-myeloablative transplant or reduced-intensity transplant), where they get lower doses of chemo and radiation that do not destroy the all cells in their bone marrow. They then are given the donor stem cells. These cells enter the body and form a new immune system, which sees the leukemia cells as foreign and attacks them (a "graft-versus-leukemia" effect). This approach is still considered experimental, and studies are under way to find out how useful it may be against AML. To learn more about stem cell transplants, see the American Cancer Society document, Bone Marrow & Peripheral Blood Stem Cell Transplants.
Treatment of M3 Leukemia
Patients with M3 leukemia can develop serious blood clotting or bleeding problems. This can usually be prevented or treated by giving them a blood thinner. Other treatments might include transfusions of platelets or other blood products.
The treatment of M3 leukemia (APL) differs from usual AML treatment because a non-chemo drug known as ATRA (related to vitamin A) is also used. For best results, ATRA is combined with other chemo drugs. Treatment with ATRA and chemo brings about a remission in about 8 to 9 out of 10 patients.
Consolidation treatment is usually 2 or more courses of chemo, usually along with ATRA. This is often followed by maintenance therapy with ATRA for at least a year. Some doctors may also add low doses of chemo. After remission, further treatment consists of 2 or more courses of chemotherapy followed by maintenance with ATRA for a least one year. About 70% to 90% of patients with APL are cured with this approach.
Side effects from ATRA can include retinoic acid syndrome. This very serious side effect can cause breathing problems from fluid buildup, low blood pressure, and kidney damage.
Surgery
Surgery is not generally used to treat leukemia because leukemia is a disease of blood and bone marrow and it is not possible to bring about a cure with surgery. But surgery may be used to help deliver treatment. A plastic tube can be placed into a large vein. The tube, called a venous access device, allows chemotherapy drugs or other medicines to be given and blood samples to be removed. This lowers the number of needle sticks needed during treatment. The patient must learn how to take care of the device to prevent it from getting infected.
Radiation Therapy
Radiation therapy uses high-energy x-rays to kill cancer cells, but plays a limited role in the treating leukemia. There are a few times in which radiation may be used to help treat leukemia:
It is sometimes used to treat leukemia that has spread to the brain and spinal fluid or to the testicles.
Radiation to the whole body is often an important part of treatment before a stem cell transplant.
It is used (rarely) to help shrink a tumor if it is pressing on the windpipe and causing breathing problems. But chemo is often used instead, as it may work more quickly.
Radiation can also be used to reduce pain in a bone to which cancer has spread, if chemo hasn't helped.
What if the Leukemia Doesn’t Respond or Comes Back After Treatment?
If AML doesn't go away with the first treatment, newer or stronger doses of chemo drugs may be tried. A stem cell transplant may be tried in younger patients if a matched stem cell donor can be found. Clinical trials of new treatment approaches may also be an option.
If the disease comes back after treatment it will most often be in the bone marrow and blood. Rarely the brain or spinal fluid will be the first place it is seen. This would be treated with chemo given directly into the spinal fluid. If the leukemia went away and has come back, another remission might be possible, although most doctors think it would be only temporary. They might suggest a stem cell transplant in this case.
For fairly small percentage of patients with APL who don't respond to the first treatment or who relapse, a drug called arsenic trioxide (Trisenox) often works well in bringing about a second remission. The most serious possible side effect of this drug is a change in heart rhythm. A stem cell transplant may be another option if a donor can be found.
If the leukemia keeps coming back or doesn’t go away, chemotherapy will finally not be very helpful. If a stem cell transplant is not an option, taking part in a clinical trial might be a good idea.
Some people want to keep on having treatment to fight the leukemia as long as they can. It is a good idea, though, to think about the odds of more treatment doing any good before making the decision to continue. Some people are tempted to try more chemo, for example, even when their doctors say that the odds of benefit are less than 1%. In these cases, it is important to think about and understand the reasons for choosing this plan.
Palliative Treatment
If a clinical trial is not an option, then it may be time to focus on relieving symptoms rather than curing the cancer. This is known as palliative treatment. The doctor may suggest more mild chemotherapy to slow the growth of the leukemia to reduce symptoms.
If there is pain, then it’s important to treat it with pain killing medicines. Sometimes medicines or blood transfusions are needed to correct low blood counts and tiredness. Nausea and loss of appetite may be helped by high-calorie food supplements and medicines. Antibiotics may be needed to treat infection.
At some point, the patient might want to think about hospice care. Most of the time, this can be given at home. Hospice focuses on the patient's comfort by taking care of any symptoms or other problems. It means that the goal of care is living life as fully as possible and feeling as well as one can at this difficult stage.
While the hope for a cure may not be as bright, there is still hope for good times with family and friends -- times that can bring happiness and meaning. In a way, pausing at this time in your cancer treatment offers a chance to refocus on the most important things in your life. This is the time to do some things you've always wanted to do and to stop doing the things you no longer want to do.
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