Leukemia treatment side effects

Side Effects

Therapy for acute lymphoblastic leukemia (ALL) can produce side effects. For most patients, treatment side effects are temporary and go away once therapy ends. For other patients, side effects can be severe, sometimes requiring hospitalization.

Before you start treatment, talk with your doctor about potential side effects. Drugs and other therapies can prevent or manage many side effects. Many treatment side effects go away or become less noticeable over time.

Common Side Effects

The side effects you may experience depend on:

  • The intensity of chemotherapy
  • The drugs used during therapy
  • Your overall health and whether you have any chronic health conditions like diabetes or kidney disease

Side effects common to ALL and its treatment, especially chemotherapy and stem cell transplantation, include:

  • Low blood cell counts. ALL can cause a decrease in normal blood cell production. Chemotherapy is toxic to both normal blood cells and ALL cells, making the number of your healthy cells decrease.
  • Infection. Your infection risk increases during chemotherapy when your body doesn’t produce enough white cells to keep your immune system working properly.
  • Graft versus host disease. If you undergo an allogeneic stem cell transplantation, you’re at risk of developing graft versus host disease (GVHD). GVHD develops when the donor’s immune cells mistakenly attack the patient’s normal cells. GVHD can be mild, moderate or severe – even life threatening.
  • Tumor Lysis Syndrome (TLS). TLS is characterized by metabolic abnormalities caused by the sudden release of the cellular contents of dying cells into the bloodstream, which is induced by chemotherapy. If untreated, TLS can lead to heart arrhythmias, seizures, loss of muscle control, acute kidney failure and even death. Patients are constantly monitored for the development of this condition and are drugs such asallopurinol (Zyloprim®) or rasburicase (Elitek®) to prevent or lessen the effects of TLS. Patients who have very high white blood cell counts before induction therapy are at an especially high risk for developing TLS.
  • In patients who have received corticosteroids such as prednisone and dexamethasone as part of their treatment:
    • Hyperglycemia and steroid-induced diabetes. Patients should be monitored for glucose control.
    • Gastric ulcers. Use of proton-pump inhibitor drugs to reduce stomach acid is recommended during steroid therapy to reduce these risks.

The following side effects are also common. to read more about these side effects.

  • Mouth sores
  • Diarrhea
  • Hair loss
  • Rashes
  • Itchy skin
  • Nausea and vomiting
  • Headaches
  • Loss of appetite
  • Fatigue
  • Neuropathy-numbness, tingling or muscle weakness (usually in the hands or feet)

Long-Term and Late Effects of Treatment

For some patients, side effects may last or appear well after they finish treatment To read more about lingering side effects, see The Leukemia & Lymphoma Society’s free publications:

  • Long-Term and Late Effects of Treatment in Adults
  • Long-Term and Late Effects of Treatment for Childhood Leukemia and Lymphoma Facts.

For information about the drugs listed on this page, visit Drug Listings.

Related Links

  • Download or order The Leukemia & Lymphoma Society’s free booklet,
    • Acute Lymphoblastic Leukemia
    • Understanding Side Effects of Drug Therapy
  • Managing Side Effects
  • Integrative Medicine and Complementary and Alternative Therapies

Targeted therapy

Targeted therapy is a treatment that targets the leukemia’s specific genes, proteins, or the tissue environment that contributes to the growth and survival of the leukemia. This type of treatment blocks the growth and spread of leukemia cells while limiting damage to healthy cells.
Recent studies show that not all cancers have the same targets. To find the most effective treatment, your doctor may run tests to identify the genes, proteins, and other factors in your tumor. This helps doctors better match each patient with the most effective treatment whenever possible. In addition, many research studies are taking place now to find out more about specific molecular targets and new treatments directed at them. Learn more about the basics of targeted treatments.

The following targeted treatments may be used for AML, depending on the gene mutations found in the leukemia cells:

  • Enasidenib (IDHIFA) for people who have relapsed or refractory AML with an IDH2 mutation.

  • Gilteritinib (Xospata) for people who have relapsed or refractory AML with a FLT3 gene mutation.

  • Ivosidenib (Tibsovo) for people with relapsed or refractory AML that has an IDH1 gene mutation.

  • Midostaurin (Rydapt) for people who have AML with a FLT3 gene mutation. About 25% to 30% of people with AML have AML with a FLT3 gene mutation.

Talk with your doctor about possible side effects for a specific medication and how they can be managed

Acute promyelocytic leukemia (APL) treatment

The treatment of the APL subtype of AML is very different. This subtype is very sensitive to the effects of all-trans retinoic acid (ATRA). ATRA is a drug that is similar to vitamin A and is given by mouth. People with the APL subtype who receive a combination of ATRA and arsenic trioxide (Trisenox) are very likely to have a CR. Less commonly, chemotherapy containing regimens (see above) with idarubicin, daunorubicin, or cytarabine may also be used. Arsenic trioxide may be used during induction therapy alone or in combination with ATRA during post-remission therapy or if APL comes back after treatment.

Mild to severe bleeding is a common symptom of APL. Patients with this subtype often need many platelet and blood transfusions during initial treatment. Compared with other subtypes of AML where maintenance therapy is not used, some patients with APL may benefit from use of ATRA plus low dose oral chemotherapy for 1 to 2 years after the initial treatment.

Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles to destroy cancer cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist. The most common type of radiation therapy is called external-beam radiation therapy, which is radiation given from a machine outside the body. A radiation therapy regimen, or schedule, usually consists of a specific number of treatments given over a set period of time. Because AML is found throughout the blood, radiation therapy is generally used only when leukemia cells have spread to the brain or to shrink a myeloid sarcoma.

Side effects from radiation therapy may include fatigue, mild skin reactions, upset stomach, and loose bowel movements. Most side effects go away soon after treatment is finished. Learn more about the basics of radiation therapy.

Getting care for symptoms and side effects

Leukemia and its treatment often cause side effects. In addition to treatments intended to slow, stop, or eliminate the disease, an important part of care is relieving a person’s symptoms and the side effects of treatment. This approach is called palliative or supportive care, and it includes supporting the patient with his or her physical, emotional, and social needs.
Palliative care is any treatment that focuses on reducing symptoms, improving quality of life, and supporting patients and their families. Any person, regardless of age or type and stage of cancer, may receive palliative care. It works best when palliative care is started as early as needed in the cancer treatment process. People often receive treatment for the leukemia at the same time that they receive treatment to ease side effects. In fact, patients who receive both at the same time often have less severe symptoms, better quality of life, and report they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional support, and other therapies. You may also receive palliative treatments similar to those meant to eliminate the leukemia, such as chemotherapy or radiation therapy. Talk with your doctor about the goals of each treatment in the treatment plan.

Before treatment begins, talk with your health care team about the possible side effects of your specific treatment plan and palliative care options. During and after treatment, be sure to tell your doctor or another health care team member if you are experiencing a problem so it can be addressed as quickly as possible. Learn more about palliative care.

Refractory AML

If leukemia is still present after initial treatment, the disease is called refractory AML. If this happens, it is a good idea to talk with doctors who have experience in treating it. Doctors can have different opinions about the best standard treatment plan. Also, clinical trials might be an option. Learn more about getting a second opinion before starting treatment, so you are comfortable with your chosen treatment plan.

Your treatment plan may include new drugs being tested in clinical trials. An ALLO stem cell transplant should also be considered. Palliative care will also be important to help relieve symptoms and side effects.

For most patients, a diagnosis of refractory leukemia is very stressful and, at times, difficult to bear. Patients and their families are encouraged to talk about the way they are feeling with doctors, nurses, social workers, or other members of the health care team. It may also be helpful to talk with other patients, including through a support group.

Remission and the chance of recurrence

A remission is when the leukemia cannot be detected in the body, there are no symptoms, and a patient’s blood counts are normal. This may also be called having “no evidence of disease” or NED.
A remission may be temporary or permanent. This uncertainty causes many people to worry that the cancer will come back. While many remissions are permanent, it’s important to talk with your doctor about the possibility of the leukemia returning. Understanding your risk of recurrence and the treatment options may help you feel more prepared if the disease does return. Learn more about coping with the fear of recurrence.
If the leukemia does return after the original treatment, it is called recurrent or relapsed leukemia. When this occurs, a new cycle of testing will begin again to learn as much as possible about the recurrence. After this testing is done, you and your doctor will talk about your treatment options. Often the treatment plan will include the treatments described above, such as chemotherapy, stem cell transplantation, targeted therapy, and radiation therapy, but they may be used in a different combination or given at a different pace. Your doctor may suggest clinical trials that are studying new ways to treat this type of recurrent leukemia. Whichever treatment plan you choose, palliative care will be important for relieving symptoms and side effects.

The treatment for recurrent AML often depends on the length of the initial remission. If the AML comes back after a long remission, the original treatment may work again. If the remission was short, then other drugs are used, often through a clinical trial. An ALLO stem cell transplant may be the best option for patients whose leukemia has come back after initial treatment. However, many drugs and other approaches are being researched in clinical trials and these may provide other treatment options.
People with recurrent leukemia often experience emotions such as disbelief or fear. Patients are encouraged to talk with their health care team about these feelings and ask about support services to help them cope. Learn more about dealing with cancer recurrence.

If treatment doesn’t work

Recovery from leukemia is not always possible. If the leukemia cannot be cured or controlled, the disease may be called advanced or terminal.

This diagnosis is stressful, and for many people, advanced leukemia may be difficult to discuss because it is incurable. However, it is important to have open and honest conversations with your doctor and health care team to express your feelings, preferences, and concerns. The health care team is there to help, and many team members have special skills, experience, and knowledge to support patients and their families. Making sure a person is physically comfortable and free from pain is extremely important.
Patients with advanced leukemia who have no more effective treatment options available may want to consider a type of palliative care called hospice care. Hospice care is designed to provide the best possible quality of life for people who are near the end of life. You and your family are encouraged to talk with the health care team about hospice care options, which include hospice care at home, a special hospice center, or other health care locations. Nursing care and special equipment can make staying at home a workable option for many families. Learn more about advanced cancer care planning.
After the death of a loved one, many people need support to help them cope with the loss. Learn more about grief and loss.

The next section in this guide is About Clinical Trials. It offers more information about research studies that are focused on finding better ways to care for people with cancer. You may use the menu to choose a different section to read in this guide.

For Patients & Families
Complications & Side Effects

Most people find it helpful to have information about side effects so they know what to expect and how to manage them. Although the side effects of treatment can be unpleasant, it is important to know that they are usually temporary. Many of these side effects and complications can be treated with medications and careful monitoring.

Remember that all patients are unique. No two persons will have the same experience with side effects. The degree and intensity of each possible side effect also vary greatly from person to person.

Your BMT healthcare team will work closely with you to minimise any discomfort that you may have as a result of your treatment.

What Are the Common Side Effects?

The common side effects and management of chemo/radiotherapy are:

  • Bladder Effects
  • Bleeding & Transfusion
  • Cardiac Effects
  • Diarrhea
  • Fatigue
  • Female Sexuality
  • Fertility Issues
  • Graft-Versus-Host Disease (GVHD)
  • Hair Loss
  • Liver Effects
  • Lung Effects
  • Male Sexuality
  • Nausea & Vomiting
  • Neurologic Effects
  • Relapse
  • Skin Rashes
  • Sore Mouth & Throat
  • Taste Effects

Why Does Chemotherapy Have Side Effects?

Chemotherapy and radiotherapy affect the rapidly dividing cells of the body. Besides affecting the cancer cells, chemo/radiotherapy also cause changes to cells in the hair, nails, lining of the mouth, gastrointestinal tract and blood. As a result, hair loss, nail loss, sore mouth, diarrhea and decreases in the blood cell counts may occur in patients receiving these treatments. It is not possible to prevent these effects, although many of the symptoms can be controlled or minimised.

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Coping With Leukemia Treatment Side Effects

Leukemia treatment side effects can range from minimal to severe, depending on what therapies you and your medical team choose. If you are suffering, don’t just grit your teeth and bear it. Doctors have several ways to lessen treatment side effects, and there are things you, the leukemia patient, can do to help yourself.

“The worse conversation I ever have is when a patient comes back to start their second cycle of treatment and informs me that they had weeks of suffering with nausea and vomiting — and they never called,” says Stuart Goldberg, MD, chief of the division of leukemia at the John Theurer Cancer Center at the Hackensack University Medical Center in New Jersey. “A brief visit to our clinic, intravenous fluids, and an anti-nausea shot would have halted the problem,” he says.

Chemotherapy is often a first-line leukemia treatment. Standard chemotherapy kills rapidly dividing cells, whether they are healthy or cancerous. The drugs typically affect cells in the hair, blood, and gastrointestinal tract, frequently causing side effects.

Your treatment plan will depend on several factors including: how aggressive the leukemia is (acute or chronic), the specific type of cancer, and your age. Aside from chemotherapy, treatments can include drugs that block the growth of leukemia cells, radiation, biological therapy, surgery, and sometimes a bone marrow or stem cell transplant.

The severity of treatment side effects generally depends on what drugs are used, their dosage, and the length of treatment. If chemotherapy is combined with other therapies, like radiation, some chemotherapy side effects can be worse.

Managing Leukemia Treatment Side Effects

Chemotherapy or radiation side effects vary from person to person. Your doctor can tell you what to expect. Keep in mind that most side effects are temporary. Drinking plenty of fluids, eating as well as you can to prevent weight loss, and getting plenty of rest can help ease some of these common problems:

  • Fatigue. Chemotherapy side effects can include a drop in red blood cells, which can leave you feeling worn out. If you become severely anemic, you’ll be given a transfusion of red blood cells. Fatigue can also be a radiation side effect, especially as treatment progresses.
  • Infection. Your white blood cell count probably will fall if you are getting chemo, leaving you at risk of infection. To avoid catching something, steer clear of sick people and wash your hands. Don’t eat raw fruits or vegetables and stay away from fresh flowers because of germs and mold. People who get a stem cell transplant also are at high risk of infection until their immune system fully recovers. Your doctor may put you on preventive medications or opt to treat you if an infection occurs. Call your doctor if you start running a fever.
  • Bleeding. A low blood platelet count can cause problems like nosebleeds, bleeding gums, and blood in the stool or urine. If platelets get too low, you’ll probably get a platelet transfusion to avoid more serious bleeding. Use a soft toothbrush, an electric razor instead of a blade, and don’t strain during bowel movements.
  • Diarrhea and constipation. Changes in diet and adding more fluids can help. Anti-diarrhea medicines and stool softeners may be needed; talk to your doctor.
  • Nausea and vomiting. Eating dry toast or crackers may keep queasiness at bay. But call your doctor; medication can help. “We’ve come a long way with anti-nausea medications in the last 10 years; this has been a benefit for many leukemia patients,” Dr. Goldberg says.
  • Sore mouth. Mouth sores are not only painful, they can get infected, so it’s important to keep your mouth clean. Rinse four times a day with a solution of a one-half teaspoon of baking soda in eight ounces of water. Avoid alcohol and spicy foods.
  • Hair loss. Not everyone loses their hair, but if you do, it can be a hit to your self-image. If you want to get a wig, snip some natural hair before it all falls out and take it with you to the store to get the best match. Hospitals also have information about what else you can do about hair loss.

Goldberg points out that advances in the last decade have revolutionized leukemia treatment for many people. For instance, in chronic myeloid leukemia, tyrosine kinase inhibitor drugs, like imatinib mesylate (Gleevec), have dramatically increased life expectancy in most patients with minimal side effects, he says.

He adds that new targeted therapies are being developed for acute leukemia in elderly people. These drugs aggressively attack the cancer but have fewer side effects than standard treatment.

One Woman’s Story

Elaine, a retired teacher from Ringwood, N.J., was diagnosed with acute myeloid leukemia in June 2008 and was treated by Goldberg. She was hospitalized and given intensive chemotherapy for three weeks, which put the leukemia into remission. Over the next four months, Elaine returned to the hospital and received more chemo for five days each month.

At different times in her treatment, Elaine experienced chemotherapy side effects including fatigue, hair loss, and sore gums. She also lost the tips of her fingernails. But all in all, Elaine says she feels “blessed” she didn’t experience anything severe. “My body was able to tolerate the chemo and my treatments worked as they were supposed to; I’m very thankful.”

Elaine listened to her doctor’s orders and didn’t eat anything that could put her at risk of infection and was “very careful around knives or anything harmful.” She managed to avoid serious infection or bleeding.

Elaine says her faith — and the love and support of family and friends — helped her get through the treatment. Today, her hair has grown back — and she’s happy to say she’s in total remission.

Side effects of ALL treatment

All treatments can cause side effects. However, the type and severity will vary between individuals, depending on the type of treatment used and how an individual responds to it. In general, more intensive treatment is associated with more severe side effects. It is important to report any symptoms you are having to your doctor or nurse. In most cases they can be treated and are reversible.

When to contact your doctor or hospital

As a general rule, while you are having treatment you will need to contact your doctor or hospital immediately if you experience any of the following:

  • a temperature of 38oC or higher (even if it returns to normal) and/or an episode of uncontrolled shivering (a rigor)
  • bleeding or bruising, for example blood in your urine, faeces, or sputum; bleeding gums, or a persistent nose bleed
  • nausea or vomiting that prevents you from eating or drinking or taking your normal medications
  • severe diarrhoea, stomach cramps or constipation
  • coughing or shortness of breath
  • a new rash, reddening of the skin, itching
  • a persistent headache
  • a new pain or soreness anywhere
  • if you cut or otherwise injure yourself
  • if you notice pain, swelling, redness or pus anywhere on your body.

What are the side effects of ALL treatment?

Effects on the bone marrow

Chemotherapy affects the bone marrow’s ability to produce adequate numbers of blood cells. As a result, your blood count (the number of white cells, platelets and red cells circulating in your blood) will generally fall within a week of treatment. The length of time it takes for your bone marrow and blood counts to recover mainly depends on the type of chemotherapy you are given.

When your platelet count is very low (thrombocytopenic) you can bruise and bleed more easily. In many cases a transfusion of platelets is given to reduce the risk of bleeding until the platelet count recovers.

If your red blood cell count and haemoglobin levels drop you will probably become anaemic. When you are anaemic you feel more tired and lethargic than usual. If your haemoglobin level is very low, your doctor may prescribe a blood transfusion.

Risk of infection

The point at which your white blood cell count is at its lowest is called the nadir. This is usually expected 10-14 days after having your chemotherapy. During this time you will be at a higher risk of developing an infection. At this stage you will also be neutropenic, which means that your neutrophil count is low.

Neutrophils are important white blood cells that help us to fight infection. While your white blood cell count is low you should take sensible precautions to help prevent infection. These include avoiding crowds, avoiding close contact with people with infections who are contagious (for example colds, flu, chicken pox) and only eating food that has been properly prepared and cooked. If you do develop an infection you may experience a fever, which may or may not be accompanied by an episode of shivering where you shake uncontrollably. Infections while you are neutropenic can be quite serious and need to be treated with antibiotics as soon as possible. Sometimes your doctor may decide to use a drug like G-CSF to help the recovery of your neutrophil count. This drug works by stimulating the bone marrow to increase the production of neutrophils. G-CSF is given as an injection under the skin (subcutaneous).

Nausea and vomiting

Nausea and vomiting are often associated with chemotherapy and some forms of radiotherapy. These days however, thanks to significant improvements in anti-sickness (anti-emetic) drugs, nausea and vomiting are generally very well controlled. You will be given anti-sickness drugs before and for a few days after your chemotherapy treatment. Be sure to tell the nurses and doctors if the anti-emetics are not working for you and you still feel sick. Some people find that eating smaller meals more frequently during the day, rather than a few large meals, helps to reduce nausea and vomiting. Many find that eating cool or cold food is more palatable, for example jelly or custard. Drinking ginger ale or soda water and eating dry toast may also help if you are feeling sick.

Changes in taste and smell

Both chemotherapy and radiation therapy can cause changes to your sense of taste and smell. This is usually temporary but in some cases it lasts up to several months.


Mucositis, or inflammation of the lining of the mouth, throat or gut is a common and uncomfortable side effect of chemotherapy and some forms of radiotherapy. It usually starts about a week after the treatment has finished and goes away once your blood count recovers, usually a couple of weeks later. During this time your mouth and throat could get quite sore. Soluble paracetamol and other topical drugs (ones which can be applied to the sore area) can help. If the pain becomes more severe, stronger pain killers might be needed. It is important to keep your mouth as clean as possible while you are having treatment to help prevent infection. Avoid commercial mouthwashes, as these are often too strong or may contain alcohol, which will hurt your mouth.

Bowel changes

Chemotherapy can cause some damage to the lining of your bowel wall. This can lead to cramping, wind, abdominal swelling and diarrhoea. Be sure to tell the nurses and doctors if you experience any of these symptoms. If you are constipated or if you are feeling any discomfort or tenderness around your anus when you are trying to move your bowels, you may need a gentle laxative to help soften your bowel motions.

Hair loss

Hair loss is unfortunately a very common side effect of chemotherapy and some forms of radiotherapy. It is, however, usually only temporary. The hair can start to fall out within a couple of weeks of treatment and tends to grow back three to six months later. Avoiding using heat or chemicals and only using a soft hairbrush and a mild baby shampoo can help reduce the itchiness and scalp tenderness which can occur while you are losing your hair. You need to avoid direct sunlight on your exposed head (wear a hat) because chemotherapy and radiotherapy makes your skin even more vulnerable to the damaging effects of the sun (like sunburn and skin cancers). Remember that without your hair your head can get quite cold so a beanie might be useful. Hair can also be lost from your eyebrows, eyelashes, arms and legs.


Most people experience some degree of tiredness in the days and weeks following chemotherapy and radiotherapy. Getting plenty of rest and doing a little light exercise each day may help to make you feel better during this time. It is important to listen to your body and rest when you are tired.


Some types of chemotherapy and radiotherapy may cause a temporary or permanent reduction in your fertility. If possible, before you commence treatment you should discuss any questions or concerns you might have regarding your future fertility with your doctor. In women, some types of chemotherapy and radiotherapy can cause varying degrees of damage to the normal functioning of the ovaries.

In men, sperm production can be impaired for a while but the production of new sperm may become normal again in the future. There are some options for preserving your fertility, if necessary, while you are having treatment for leukaemia.

Early menopause

Some cancer treatments can affect the normal functioning of the ovaries. This can sometimes lead to infertility and an earlier than expected onset of menopause, even at a young age. The onset of menopause in these circumstances can be sudden and, understandably, very distressing. Hormone changes can lead to many of the classic symptoms of menopause. Some women experience decreased sexual drive, anxiety and even depressive symptoms during this time. It is important that you discuss any changes to your periods with your doctor or nurse.

Body image, sexuality and sexual activity

Hair loss, skin changes and fatigue can all interfere with how you feel about yourself as a man or a woman and as a ‘sexual being’. During treatment you may experience a decrease in libido and it may take some time for things to return to ‘normal’. It’s perfectly reasonable and safe to have sex while you are on treatment or shortly afterwards, but there are some precautions you need to take. It is usually recommended that you or your partner do not become pregnant as some of the treatments given might harm the developing baby. As such, you need to ensure that you or your partner use a suitable form of contraception. Condoms are also necessary in the seven days following a treatment session, to ensure that your partner is not exposed to any residual drugs. Partners are sometimes afraid that sex might in some way harm the patient. This is not likely as long as the partner is free from any infections and the sex is relatively gentle.


Parotitis is an inflammation of the saliva-producing glands in the mouth, which can occur if these glands are within the treatment field used. These include the parotid or submandibular glands, which are situated at the top of the jaw line, in front of the ears. Parotitis causes dryness of the mouth and jaw pain, which usually settles down within a few days, once the inflammation subsides.

More information about ALL

” Treatment
” ALL diagnosis
” Living with ALL
” Booklets and factsheets

Last updated on February 11th, 2019

Developed by the Leukaemia Foundation in consultation with people living with a blood cancer, Leukaemia Foundation support staff, haematology nursing staff and/or Australian clinical haematologists. This content is provided for information purposes only and we urge you to always seek advice from a registered health care professional for diagnosis, treatment and answers to your medical questions, including the suitability of a particular therapy, service, product or treatment in your circumstances. The Leukaemia Foundation shall not bear any liability for any person relying on the materials contained on this website.

What are leukemia treatment options?

There are a number of different medical approaches to the treatment of leukemia. Treatment will typically depend upon the type of leukemia, the patient’s age and health status, as well as whether or not the leukemia cells have spread to the cerebrospinal fluid. The genetic changes or specific characteristics of the leukemia cells as determined in the laboratory can also determine the type of treatment that may be most appropriate.

Watchful waiting may be an option for some people with a chronic leukemia who do not have symptoms. This involves close monitoring of the disease so that treatment can begin when symptoms develop. Watchful waiting allows the patient to avoid or postpone the side effects of treatment. The risk of waiting is that it may eliminate the possibility of controlling the leukemia before it worsens.

Treatments for leukemia include chemotherapy (major treatment modality for leukemia), radiation therapy, biological therapy, targeted therapy, and stem cell transplant. Combinations of these treatments may be used. Surgical removal of the spleen can be a part of treatment if the spleen is enlarged.

Acute leukemia needs to be treated when it is diagnosed, with the goal of inducing a remission (absence of leukemia cells in the body). After remission is achieved, therapy may be given to prevent a relapse of the leukemia. This is called consolidation or maintenance therapy. Acute leukemias can often be cured with treatment.

Chronic leukemias are unlikely to be cured with treatment, but treatments are often able to control the cancer and manage symptoms. Some people with chronic leukemia may be candidates for stem cell transplantation, which does offer a chance for cure.

Many patients opt to receive a second opinion before beginning treatment for leukemia. In most cases, there is time to receive a second opinion and consider treatment options without making the treatment less effective. However, in rare cases of very aggressive leukemias, treatment must begin immediately. One should discuss with a doctor the possibility of obtaining a second opinion and any potential delays in treatment. Most doctors welcome the possibility of a second opinion and should not be offended by a patient’s wish to obtain one.


Chemotherapy is the administration of drugs that kill rapidly dividing cells such as leukemia or other cancer cells. Chemotherapy may be taken orally in pill or tablet form, or it may be delivered via a catheter or intravenous line directly into the bloodstream. Combination chemotherapy is usually given, which involves a combination of more than one drug. The drugs are given in cycles with rest periods in between.

Sometimes, chemotherapy drugs for leukemia are delivered directly to the cerebrospinal fluid (known as intrathecal chemotherapy). Intrathecal chemotherapy is given in addition to other types of chemotherapy and can be used to treat leukemia in the brain or spinal cord or, in some cases, to prevent spread of leukemia to the brain and spinal cord. An Ommaya reservoir is a special catheter placed under the scalp for the delivery of chemotherapy medications. This is used for children and some adult patients as a way to avoid injections into the cerebrospinal fluid.

Side effects of chemotherapy depend on the particular drugs taken and the dosage or regimen. Some side effects from chemotherapy drugs include hair loss, nausea, vomiting, mouth sores, loss of appetite, tiredness, easy bruising or bleeding, and an increased chance of infection due to the destruction of white blood cells. There are medications available to help manage the side effects of chemotherapy.

Some adult men and women who receive chemotherapy sustain damage to the ovaries or testes, resulting in infertility. Most children who receive chemotherapy for leukemia will have normal fertility as adults, but depending on the drugs and dosages used, some may have infertility as adults.

Biological therapy

Biological therapy is any treatment that uses living organisms, substances that come from living organisms, or synthetic versions of these substances to treat cancer. These treatments help the immune system recognize abnormal cells and then attack them. Biological therapies for various types of cancer can include antibodies, tumor vaccines, or cytokines (substances that are produced within the body to control the immune system). Monoclonal antibodies are antibodies that react against a specific target that are used in the treatment of many kinds of cancer. An example of a monoclonal antibody used in the treatment of leukemia is alemtuzumab, which targets the CD52 antigen, a protein found on B-cell chronic lymphocytic leukemia (CLL) cells. Interferons are cell signaling chemicals that have been used in the treatment of leukemia.

Side effects of biological therapies tend to be less severe than those of chemotherapy and can include rash or swelling at the injection site for IV infusions of the therapeutic agents. Other side effects can include headache, muscle aches, fever, or tiredness.

Targeted therapies are drugs that interfere with one specific property or function of a cancer cell, rather than acting to kill all rapidly growing cells indiscriminately. This means there is less damage to normal cells with targeted therapy than with chemotherapy. Targeted therapies may cause the target cell to cease growing rather than to die, and they interfere with specific molecules that promote growth or spread of cancers. Targeted cancer therapies are also referred to as molecularly targeted drugs, molecularly targeted therapies, or precision medicines.

Monoclonal antibodies (described above in the section on biologic therapy) are also considered to be targeted therapies since they specifically interfere and interact with a specific target protein on the surface of cancer cells. Imatinib (Gleevec) and dasatinib (Sprycel) are examples of targeted therapies that are used to treat CML, some cases of ALL, and some other cancers. These drugs target the cancer-promoting protein that is formed by the BCR-ABL gene translocation.

Targeted therapies are given in pill form or by injection. Side effects can include swelling, bloating, and sudden weight gain. Other side effects can include nausea, vomiting, diarrhea, muscle cramps, or rash.

Radiation therapy uses high energy radiation to target cancer cells. Radiation therapy may be used in the treatment of leukemia that has spread to the brain, or it may be used to target the spleen or other areas where leukemia cells have accumulated.

Radiation therapy also causes side effects, but they are not likely to be permanent. Side effects depend on the location of the body that is irradiated. For example, radiation to the abdomen can cause nausea, vomiting, and diarrhea. With any radiation therapy, the skin in the area being treated may become red, dry, and tender. Generalized tiredness is also common while undergoing radiation therapy.

Stem cell transplant

In stem cell transplantation, high doses of chemotherapy and/or radiation are given to destroy leukemia cells along with normal bone marrow. Then, transplant stem cells are delivered by an intravenous infusion. The stem cells travel to the bone marrow and begin producing new blood cells. Stem cells may come from the patient or from a donor.

Autologous stem cell transplantation refers to the situation in which the patient’s own stem cells are removed and treated to destroy leukemia cells. They are then returned to the body after the bone marrow and leukemia cells have been destroyed.

An allogeneic stem cells transplant refers to stem cells transplanted from a donor. These may be from a relative or an unrelated donor. A syngeneic stem cell transplant uses stem cells taken from a healthy identical twin of the patient.

Stem cells may be removed (harvested) in different ways. Typically, they are taken from the blood. They can also be harvested from the bone marrow or from umbilical cord blood.

Stem cell transplantation is done in a hospital, and it is necessary to remain in the hospital for several weeks. Risks of the procedure include infections and bleeding due to the depletion of normal blood cells. A risk of stem cell transplant with donor cells is known as graft-versus-host disease (GVHD). In GVHD, the donor white blood cells react against the patient’s normal tissues. GVHD can be mild or very severe, and often affects the liver, skin, or digestive tract. GVHD can occur at any time after the transplant, even years later. Steroids or medications that suppress the immune response may be used to treat this complication.

Chimeric antigen receptor (CAR) T-cell treatment

Chimeric antigen receptor (CAR) T-cell treatment is a new form of treatment in which a patient’s own normal T lymphocytes are re-engineered in a laboratory to attack the leukemia cells and are then reintroduced into the patient’s bloodstream. This treatment has been used for people with B-cell lymphomas that have relapsed or are refractory to treatment. It is also an approved treatment option for certain cases of leukemia. The U.S. FDA approved tisagenlecleucel (Kymriah) in 2018 for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.

CAR-T therapy is also available in clinical trials. Cytokine-release syndrome (CRS) is a potentially serious side effect frequently associated with CAR T-cell therapy. Cytokines are chemical messengers produced when the CAR T-cells multiply in the body and kill cancer cells. CRS may cause a range of symptoms from mild flu-like symptoms to more serious symptoms including fast heart rate, low blood pressure, and heart problems. Other side effects can include nerve damage, suppressed immune function, and a condition known as tumor lysis syndrome that results when cancer cells are rapidly destroyed.

Because CAR T-cell therapy is so new, the patients who have had this treatment have not been followed over the long term. Studies are under way to determine whether CAR-T treatment may be useful in other types of leukemia.

Supportive treatments

Because many of the treatments for leukemia deplete normal blood cells, increasing the risk for bleeding and infection, supportive treatments may be needed to help prevent these complications of treatment. Supportive treatments may also be needed to help minimize and manage unpleasant side effects of medical or radiation therapy.

Types of supportive and preventive treatments that can be used for patients undergoing treatment for leukemia include the following:

  • Vaccines against the flu or pneumonia
  • Blood or platelet transfusions
  • Anti-nausea medications
  • Antibiotics or antiviral medications to treat or prevent infections
  • White blood cell growth factors to stimulate white blood cell production (such as granulocyte-colony stimulating factor , made up of filgrastim and pegfilgrastim and granulocyte macrophage-colony stimulating growth factor , made up of sargramostim )
  • Red cell growth factors to stimulate red blood cell production (darbepoetin alfa or epoetin alfa )
  • Intravenous injections of immunoglobulins to help fight infection

Chemotherapy for Acute Myeloid Leukemia (AML)

Chemotherapy (chemo) is the use of anti-cancer drugs that are injected into a vein, under the skin, or into the cerebrospinal fluid (CSF), or drugs that are taken by mouth to destroy or control cancer cells. Except when given into the CSF, these drugs enter the bloodstream and reach all areas of the body, making this treatment useful for cancers such as leukemia that spread throughout the body.

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

How is chemo given?

Treatment of AML is usually divided into phases:

  • Induction is the first phase of treatment. It is short and intensive, typically lasting about a week. The goal is to clear the blood of leukemia cells (blasts) and to reduce the number of blasts in the bone marrow to normal.
  • Consolidation is chemo given after the patient has recovered from induction. It is meant to kill the small number of leukemia cells that are still around but can’t be seen (because there are so few of them). For consolidation, chemo is given in cycles, with each period of treatment followed by a rest period to allow the body time to recover.

A third phase called maintenance (or post-consolidation) involves giving a low dose of chemo for months or years after consolidation is finished. This is often used to treat acute promyelocytic leukemia (APL), but it is rarely used for other types of AML.

Most chemo drugs used to treat AML are typically given into a vein in the arm (IV). If there are signs that the leukemia has reached the brain or spinal cord (which is not common with AML), chemo might also be given into the CSF (known as intrathecal chemo). This can be done with a small catheter that is put in through a small hole in the skull (such as an Ommaya reservoir), or during a lumbar puncture (spinal tap).

The chemo regimens used to treat AML are intensive and can cause serious side effects so treatment is typically given in the hospital.

Which chemo drugs are used to treat AML?

The chemo drugs used most often to treat AML are a combination of:

  • Cytarabine (cytosine arabinoside or ara-C)
  • An anthracycline drug, such as daunorubicin (daunomycin) or idarubicin

Other chemo drugs that may be used to treat AML include:

For more on how chemo is used to treat AML, see Typical Treatment of Most Types of Acute Myeloid Leukemia (AML), Except APL.

Possible side effects

Chemo drugs can affect some normal cells in the body, 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. Side effects can include:

  • Hair loss
  • Mouth sores
  • Loss of appetite
  • Nausea and vomiting
  • Diarrhea or constipation

Chemo drugs also affect the normal cells in bone marrow, which can lower blood cell counts. This can lead to:

  • Increased risk of infections (from having too few normal white blood cells)
  • Easy bruising or bleeding (from having too few blood platelets)
  • Fatigue and shortness of breath (from having too few red blood cells)

Most side effects from chemo go away once treatment is finished. Low blood cell counts can last weeks, but then should return to normal. There are often ways to lessen these side effects. For example, drugs can be given to help prevent or reduce nausea and vomiting. Be sure to ask about medicines to help reduce side effects, and let your doctor or nurse know when you do have side effects so they can be managed effectively.

Low white blood cell counts: Some of the most serious side effects of chemo are caused by low white blood cell counts.

If your white blood cell counts are very low during treatment, you can help lower your risk of infection by carefully avoiding exposure to germs. During this time, your doctor or nurse may tell you to:

  • Wash your hands often.
  • Avoid fresh, uncooked fruits and vegetables and other foods that might carry germs.
  • Avoid fresh flowers and plants because they may carry mold.
  • Make sure other people wash their hands before they come in contact with you.
  • Avoid large crowds and people who are sick.

You may get antibiotics before you have signs of infection or at the earliest sign that an infection may be developing (such as a fever). You may also get drugs that help prevent viral and fungal infections.

Drugs known as growth factors, such as filgrastim (Neupogen), pegfilgrastim (Neulasta), and sargramostim (Leukine), are sometimes given to increase the white blood cell counts after chemo, to help lower the chance of infection. However, it’s not clear if they have an effect on treatment success.

Low platelet counts: If your platelet counts are low, you may be given drugs or platelet transfusions to help prevent bleeding.

Low red blood cell counts: Shortness of breath and extreme fatigue caused by low red blood cell counts (anemia) may be treated with drugs or with red blood cell transfusions.

Decisions about when a patient can leave the hospital are often influenced by his or her blood counts. Some people find it helpful to keep track of their counts. If you are interested in this, ask your doctor or nurse about your blood cell counts and what these numbers mean.

Side effects of specific drugs: Certain drugs have some specific possible side effects. For example:

  • High doses of cytarabine can cause dryness in the eyes and effects on certain parts of the brain, which can lead to problems with coordination or balance. The drug dose may need to be reduced or stopped altogether if these side effects appear.
  • Anthracyclines (such as daunorubicin or idarubicin) can damage the heart, so they might not be used in someone who already has heart problems.

Other organs that could be damaged by chemo drugs include the kidneys, liver, testicles, ovaries, and lungs. Doctors and nurses carefully monitor treatment to limit the risk of these side effects as much as possible.

If serious side effects occur, the chemo may have to be reduced or stopped, at least for a short time. Careful monitoring and adjustment of drug doses are important because some side effects can last a long time.

Tumor lysis syndrome: This side effect of chemo can occur in patients who have large numbers of leukemia cells in the body, mainly during the induction phase of treatment. When chemo kills these cells, they break open and release their contents into the bloodstream. This can overwhelm the kidneys, which aren’t able to get rid of all of these substances at once. Excess amounts of certain minerals can also affect the heart and nervous system. This can be prevented by giving extra fluids during treatment and by giving certain drugs, such as bicarbonate, allopurinol, and rasburicase, which help the body get rid of these substances.


Chemotherapy for leukemia

Chemotherapy for leukemia often consists of giving several drugs together in a set regimen. Because each medication destroys tumor cells in different ways, a combination of drugs may make the cells more vulnerable to treatment.

For patients with leukemia, chemotherapy is typically given orally, usually in pill form or intravenously (directly into the vein). In some cases, chemotherapy drugs may be delivered intrathecally, directly into the cerebrospinal fluid, which surrounds the brain and spinal cord. This procedure is designed to target cancer cells that have spread there. It is delivered either through a lumbar puncture (also called a spinal tap), or through a special device placed under the scalp.

For many of our patients, we place a port (central venous access catheter) to deliver chemotherapy, give intravenous fluids and obtain blood samples. This helps to reduce the discomfort of multiple needle pricks.

Throughout your leukemia chemotherapy treatment, your care team will perform regular blood tests and other diagnostic tests to check for leukemia cells and make modifications to your treatment as needed.

A common chemotherapy treatment for acute leukemias begins with induction chemotherapy, followed by intensification, or consolidation, chemotherapy. In induction chemotherapy, a combination of drugs is used to destroy as many leukemia cells as possible and bring blood counts to normal. Then, intensification chemotherapy is intended to destroy remaining leukemia cells that cannot be seen in the blood or bone marrow. Some patients may also receive maintenance chemotherapy. This less intensive course of chemotherapy is used to reduce the risk of the disease recurring after treatment has finished.

While chemotherapy destroys rapidly dividing cancer cells, it may also affect normal fast-growing cells, such as those in the hair, mouth, GI tract and bone marrow. Chemotherapy for leukemia may also temporarily interfere with the ability of the bone marrow to produce adequate numbers of blood cells. Depending on the drugs used and your individual response, you may experience side effects of chemotherapy. Your care team may recommend a combination of approaches to prevent or manage chemotherapy-related side effects throughout leukemia chemotherapy treatment.

Next topic: How is leukemia treated with radiation therapy?


Acute myeloid leukaemia

You may be able to go home between treatment rounds.

You’ll have regular blood transfusions because your blood will not contain enough healthy blood cells.

You’ll also be vulnerable to infection, so it’s important that you’re in a clean and stable environment where your health can be carefully monitored and any infection you have can be treated quickly.

You may also be prescribed antibiotics to help prevent infections.

For intensive treatment, the chemotherapy medications will be injected into a thin tube that’s inserted either into a blood vessel near your heart or into your arm.

Side effects of intensive chemotherapy for AML are common.

They can include:

  • feeling or being sick
  • bruising or bleeding easily
  • diarrhoea
  • loss of appetite
  • sore mouth and mouth ulcers (mucositis)
  • tiredness
  • skin rashes
  • hair loss
  • infertility – this may be temporary or permanent (see complications of AML for more information)

Most side effects should resolve once treatment has finished. Tell a member of your care team if side effects become particularly troublesome, as there are medicines that can help you cope better with certain side effects.

Find out more about the side effects of chemotherapy

Non-intensive chemotherapy

If your doctors do not think you’re fit enough to withstand the effects of intensive chemotherapy, they may recommend non-intensive treatment.

This involves using an alternative type of chemotherapy to the standard intensive therapy.

The medications used during non-intensive chemotherapy may be given through a drip into a vein, by mouth or by injection under the skin, and can often be given on an outpatient basis.

Other drugs

If you have the type of AML known as acute promyelocytic leukaemia, you’ll usually be given other drugs as well as having chemotherapy.

The 2 drugs most commonly used are:

  • all tans retinoic acid (ATRA) – usually given during and after induction chemotherapy, it changes immature white blood cells into healthy mature cells, and can reduce symptoms quickly
  • arsenic trioxide – usually given if the AML has come back, it speeds up the death of leukaemia cells and changes the immature blood cells into healthy mature cells

Side effects of ATRA can include headaches, nausea, bone pain, and dry mouth, skin and eyes.

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