Mastocytosis

Treatment Options

This section outlines treatments that are the standard of care (the best proven treatments available) for this specific type of disease. 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

The treatment of mastocytosis depends on the symptoms of the disease, its extent, and the person's overall health. In many cases, 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.

Descriptions of the most common treatment options for mastocytosis are listed below. There is no cure for mastocytosis, although several treatments can be used to relieve symptoms and remove a mastocytoma (tumor). One important treatment for mastocytosis is to avoid anything that may cause the mast cells to release histamine, such as extreme temperatures, alcohol, emotional stress, insect bites, and certain medications.

Palliative/supportive care

Mastocytosis 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. The most common treatments to help relieve the symptoms of mastocytosis are listed below.

Medications. Antihistamines can help relieve symptoms, such as itching, flushing, and other skin reactions. Other medications can help relieve diarrhea and stomach pain. Steroids may be used to reduce the size of skin lesions. Nonsteroidal anti-inflammatory drugs (NSAIDs) should be used with caution, as these medicines may trigger the release of histamine and can cause severe reactions. Often, people being treated for mastocytosis will carry a syringe of epinephrine (a hormone made by the adrenal glands) in case of a severe allergic reaction.

Ultraviolet light. Sometimes, exposing the skin rashes to a source of ultraviolet (UV) light can help relieve symptoms.

Palliative care can help a person at any stage of illness. People often receive treatment for the disease 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.

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

Surgery. Surgery may be performed to remove lesions. Many surgeries on the skin can be performed quickly and easily. 

Chemotherapy

Chemotherapy is sometimes used when mastocytosis becomes cancerous. 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.

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. 

Targeted therapy

Targeted therapy is a treatment that targets the disease's specific genes, proteins, or the tissue environment unique to mastocytosis that contribute to its growth and survival. This type of treatment blocks the growth of mast cells while limiting damage to normal cells. Talk with your doctor about possible side effects for a specific medication and how they can be managed.

Recent studies show that not all diseases 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 mastocytosis. 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. 

Tyrosine kinase inhibitors are a type of targeted therapy. For mastocytosis, the target is the unique protein called the c-kit tyrosine kinase receptor. Treatment with tyrosine kinase inhibitors, including imatinib (Gleevec), dasatinib (Sprycel), nilotinib (Tasigna), and midostaurin, is sometimes considered for patients with a mutation in the c-kit tyrosine kinase receptor . Patients with more advanced systemic mastocytosis should be tested for this mutation.

Stem cell transplantation/bone marrow 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.

Stem cell transplantation is not used frequently in people with mastocytosis because treatment results are inconsistent and there are significant risks with this treatment approach. 

Recurrent mastocytosis-related cancer

A remission is when cancer cannot be detected in the body and there are no symptoms. This may also be called “no evidence of disease” or NED. Recurrent mastocytosis-related cancer is cancer that comes back after treatment.

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

Often the treatment plan for a recurrent mastocytosis-related cancer will include the therapies described above (such as surgery and chemotherapy) 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 cancer. Supportive care will also be important to help relieve symptoms and side effects.

People with recurrent cancer 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. 

If treatment fails

Recovery from mastocytosis-related cancer is not always possible. If treatment is not successful, the disease may be called advanced or terminal mastocytosis.

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 mastocytosis, 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 mastocytosis are described in detail within the Treatment section. Side effects depend on a variety of factors, including 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 mastocytosis. 

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 mastocytosis ends, talk with your doctor about developing a follow-up care plan. This plan may include regular physical examinations and/or medical tests to monitor your recovery for the coming months and years. Follow-up visits will include blood tests and possibly scans or other imaging studies. Normally, follow-up visits are most frequent in the first three years after treatment, but patients are encouraged to have lifelong, follow-up care.

People who received ultraviolet (UV) rays for the treatment of cutaneous mastocytosis should be aware of increased risk of skin cancer in the future. Follow-up physical examinations and skin evaluations are important in the early diagnosis and prevention of this condition.

Chemotherapy may cause several long-term side effects:

People who have received certain drugs may develop lung damage.

Heart damage in the form of a weakened heart muscle may occur in people who have received a higher dose of doxorubicin (Adriamycin) or radiation therapy to the chest.

Infertility (inability to have children) or premature (early) menopause can occur in people who have received high-dose cyclophosphamide (Cytoxan, Neosar) or other chemotherapy. 

Secondary cancers (cancers that develop as a result of the treatment for another type of cancer), including leukemia, are more common in people who received chemotherapy.

People recovering from mastocytosis are encouraged to follow established guidelines for good health, such as maintaining a healthy weight, 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 mastocytosis, 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.

Targeted therapy. As explained in Treatment, some people with mastocytosis have a mutation of the c-kit tyrosine kinase receptor, which causes mast cell growth. Early results from clinical trials show that the drug imatinib and other new tyrosine kinase inhibitors, such as dasatinib and nilotinib, may help treat mastocytosis. In addition, a new drug called midostaurin is also being studied for mastocytosis.

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