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Cancer Medicine :: Leukemia - Eosinophilic

Leukemia - Eosinophilic

Treatment Options

This section outlines treatments that are the standard of care (the best proven treatments available) for this specific type of leukemia. When making treatment plan decisions, patients are also encouraged to consider clinical trials as an option. A clinical trial is a research study to test a new treatment to evaluate whether it is safe, effective, and possibly better than standard treatment. Your doctor can help you review all treatment options. 

Treatment overview

For leukemia, different types of doctors often work together to create a patient's overall treatment plan that combines different types of treatments. This is called a multidisciplinary team.

According to the National Cancer Institute, the best treatment for eosinophilic leukemia remains unclear. As explained in the Overview, eosinophilic leukemia is classified as a myeloproliferative disorder. Descriptions of the most common treatment options for a myeloproliferative disorder are listed below. Treatment options and recommendations depend on several factors, including the type of leukemia, possible side effects, and the patient's preferences and overall health. Usually, myeloproliferative disorders cannot be cured, but there is often effective treatment to control the symptoms over the long term. 


Chemotherapy is the use of drugs to kill cancer cells, usually by stopping the cancer cells' ability to grow and divide. Systemic chemotherapy is delivered through the bloodstream to reach cancer cells throughout the body. Chemotherapy is given by a medical oncologist, a doctor who specializes in treating cancer with medication, or a hematologist, a doctor who specializes in treating blood disorders. A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a set period of time. A patient may receive one drug at a time or combinations of different drugs at the same time.

Chemotherapy used to treat eosinophilic leukemia includes hydroxyurea (Droxia, Hydrea), cyclophosphamide (Cytoxan, Neosar), and vincristine (Vincasar, Oncovin).

The side effects of chemotherapy depend on the individual and the dose used, but they can include fatigue, risk of infection, nausea and vomiting, loss of appetite, and diarrhea. These side effects usually go away once treatment is finished.

The medications used to treat cancer are continually being evaluated. Talking with your doctor is often the best way to learn about the medications prescribed for you, their purpose, and their potential side effects or interactions with other medications. 


Surgery to remove the spleen (called a splenectomy), which also makes white blood cells, may be recommended for some patients. A surgical oncologist is a doctor who specializes in treating cancer using surgery.  


Immunotherapy (also called biologic therapy), is designed to boost the body's natural defenses to fight the cancer. It uses materials made either by the body or in a laboratory to bolster, target, or restore immune system function. Recombinant interferon alpha (Alferon, Roferon-A, Intron A) is a biologic therapy that is occasionally used to treat eosinophilic leukemia. 

Targeted therapy

Targeted therapy is a treatment that targets the cancer's specific genes, proteins, or the tissue environment that contributes to cancer growth and survival. This type of treatment blocks the growth and spread of cancer cells while limiting damage to normal 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 involved in your leukemia. As a result, doctors can 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. 

In many patients with hypereosinophilic syndrome, the leukemia cells have a specific genetic change that makes an abnormal protein (called the FIP1-like-1/platelet-derived growth factor alpha) that stimulates the cells to grow.

Imatinib (Gleevec) is a drug that stops enzymes called tyrosine kinases from working and can kill abnormal eosinophils by blocking the effect of the FIP1-like-1-platelet-derived growth factor alpha protein. Imatinib is more likely to work for patients who have this mutation; however, if a patient does not have this mutation, there is still a possibility that the drug will work.

Talk with your doctor about possible side effects for a specific medication and how they can be managed. The main side effects of imatinib include swelling around the eyes, leg swelling, muscle cramping, headache, fatigue, rash, and musculoskeletal (joint) pain. If this treatment works, it is often continued throughout a person's lifetime.

Stem cell transplantation

A stem cell transplant is a medical procedure in which diseased bone marrow is replaced by highly specialized cells, called hematopoietic stem cells. Hematopoietic stem cells are found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a stem cell transplant, rather than bone marrow transplant, because it is the blood stem cells that are typically being transplanted, not the actual bone marrow tissue.

Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the type of cancer, results of any previous treatment, and the patient's age and general health.

Transplantation is a higher-risk treatment and is not used often for people with eosinophilic leukemia because it is not always effective for this disease and because many patients with this disease are older and the risks of the procedure are higher.

There are two types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO).

In both types, the goal of transplantation is to destroy cancer cells in the marrow, blood, and other parts of the body and allow replacement blood stem cells to create healthy bone marrow. In most stem cell transplants, the patient is treated with high doses of chemotherapy and/or radiation therapy to destroy as many cancer cells as possible.

Palliative/supportive care

Leukemia and its treatment often cause side effects. In addition to treatment to slow, stop, or eliminate the disease, an important part of care is relieving a person's symptoms and side effects. 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 can help a person at any stage of illness. People often receive treatment for the leukemia and treatment to ease side effects at the same time. In fact, patients who receive both often have less severe symptoms, better quality of life, and report they are more satisfied with treatment. Palliative care for leukemia may include the following treatments:

Steroids. Steroid medications are used to control some symptoms of eosinophilic leukemia.

Radiation therapy. Radiation therapy is the use of high-energy x-rays or other particles to kill 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. When radiation therapy is given using implants, it is called internal radiation therapy or brachytherapy. A radiation therapy regimen (schedule) usually consists of a specific number of treatments given over a set period of time. Side effects from radiation therapy include fatigue, mild skin reactions, upset stomach, and loose bowel movements. Most side effects go away soon after treatment is finished.  

Recurrent eosinophilic leukemia

A remission is when leukemia cannot be detected in the body and there are no symptoms. This may also be called “no evidence of disease” or NED. 

A remission can be temporary or permanent. This uncertainty leads to many survivors feeling worried or anxious that the leukemia will come back. While many remissions are permanent, it's important to talk with your doctor about the possibility of the leukemia returning. Understanding the risk of recurrence and the treatment options may help you feel more prepared if the disease does return. 

If the leukemia does return after the original treatment, it is called recurrent leukemia. When this occurs, a cycle of testing will begin again to learn as much as possible about the recurrence. After testing is done, you and your doctor will talk about your treatment options. Often the treatment plan will include the therapies described above (such as chemotherapy, immunotherapy, targeted therapy, and stem cell transplantation) but may be used in a different combination or given at a different pace. Your doctor may also suggest clinical trials that are studying new ways to treat this type of recurrent leukemia.

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. 

Refractory leukemia

If the leukemia continues to worsen despite treatment, it is called refractory leukemia.

Patients with this diagnosis are encouraged to talk with doctors who are experienced in treating this type of leukemia, because there can be different opinions about the best treatment plan.

Your doctor may recommend a treatment plan that includes a combination of the treatments discussed above (chemotherapy, targeted therapy, immunotherapy, and bone marrow/stem cell transplantation). Supportive care will also be important to help relieve symptoms and side effects.

For many patients, a diagnosis of refractory leukemia can be 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.

If treatment fails

Recovery from leukemia is not always possible. If treatment is not successful, the disease may be called advanced or terminal leukemia.

This diagnosis is stressful, and this is difficult to discuss for many people. 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.

Palliative care given toward the end of a person's life is called hospice care. You and your family are encouraged to think about where you would be most comfortable: at home, in the hospital, or in a hospice environment. Nursing care and special equipment can make staying at home a workable alternative for many families. 

Coping with Side Effects

Fear of treatment side effects is common after a diagnosis of leukemia, but it may help to know that preventing and controlling side effects is a major focus of your health care team. This is called palliative or supportive care, and it is an important part of the overall treatment plan, regardless of the stage of disease.

Common side effects from each treatment option for leukemia are described in detail within the Treatment section.  Side effects depend on a variety of factors, including the type of leukemia, the length and dosage of treatment(s), and your overall health.

Before treatment begins, talk with your doctor about possible side effects of each type of treatment you will be receiving. Ask which side effects are most likely to happen, when they are likely to occur, and what can be done to prevent or relieve them. And, ask about the level of caregiving you may need during treatment and recovery, as family members and friends often play an important role in the care of a person with leukemia. 

In addition to physical side effects, there may be psychosocial (emotional and social) effects as well. Patients and their families are encouraged to share their feelings with a member of their health care team who can help with coping strategies. 

During and after treatment, be sure to tell the health care team about the side effects you experience, even if you feel they are not serious. Sometimes, side effects can last beyond the treatment period, called a long-term side effect. A side effect that occurs months or years after treatment is called a late effect. Treatment of both types of effects is an important part of survivorship care. 

After Treatment

After treatment for eosinophilic leukemia ends, talk with your doctor about developing a follow-up care plan. This plan may include regular physical examinations, blood tests, bone marrow biopsies, and possibly scans or other imaging studies to monitor your recovery for the coming months and years. People experiencing a long-term remission are encouraged to follow cancer screening recommendations for the general population.

Some leukemia treatments may cause the following long-term side effects. It's important to talk with your health care team about the best ways to watch for these side effects and what will be done if they do occur.

Numbness, tingling, and pain in the hands and feet from vincristine.

A weakened heart muscle from higher total doses of doxorubicin (Adriamycin) or radiation therapy to the chest.

Infertility (inability to have a child) or premature menopause from high-dose cyclophosphamide or other chemotherapy. 

A secondary leukemia or another type of secondary cancer is more common after chemotherapy and radiation therapy.

People recovering from eosinophilic leukemia are encouraged to follow established guidelines for good health, such as maintaining a healthy weight, not smoking, eating a balanced diet, and having recommended cancer screening tests. Talk with your doctor to develop a plan that is best for your needs. Moderate physical activity can help rebuild your strength and energy level. Your doctor can help you create an appropriate exercise plan based upon your needs, physical abilities, and fitness level. 

Latest Research

Doctors are working to learn more about eosinophilic leukemia, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Always talk with your doctor about the diagnostic and treatment options best for you.

Genetic research. Because this type of disorder is usually associated with a genetic mutation, researchers are searching for specific genes or mutations that can indicate leukemia.

Stem cell/bone marrow transplantation. New approaches for stem cell/bone marrow transplantation are being tested to increase the use of this treatment option.

New treatments. Better combinations of chemotherapy and other treatment combinations are also being studied, as is the drug mepolizumab. Early studies have shown that mepolizumab lowers the number of eosinophils for people with allergies and HES, and it has helped control some signs and symptoms of HES.

Supportive care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current eosinophilic leukemia treatments in order to improve patients' comfort and quality of life.

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