Mastocytosis: Treatment Options

Approved by the Cancer.Net Editorial Board, 05/2015

ON THIS PAGE: You will learn about the different ways doctors use to treat people with mastocytosis. To see other pages, use the menu on the side of your screen.

This section outlines treatments that are the standard of care (the best known treatments available) for the various types of mastocytosis. 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 approach to treatment to evaluate whether it is safe, effective, and possibly better than the standard treatment. Clinical trials may test such approaches as a new drug, a new combination of standard treatments, or new doses of current therapies. Your doctor can help you review all treatment options. For more information, visit the About Clinical Trials and Latest Research sections.

Treatment overview

The treatment of mastocytosis depends on the type, 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. Medical teams also include a variety of other health care professionals, including physician assistants, nurses, social workers, pharmacists, counselors, dietitians, and others.

There is no cure for mastocytosis, although several treatments can be used to relieve symptoms and remove a mastocytoma (see the Overview section). Descriptions of the most common treatment options for mastocytosis are listed below. Take time to learn about all of your treatment options and be sure to ask questions about things that are unclear. Also, talk about the goals of each treatment with your doctor and what you can expect while receiving the treatment. Learn more about making treatment decisions.

Managing symptoms

An important part of treating mastocytosis is controlling a person’s symptoms. One important way to do this is to avoid anything that may cause mast cells to release histamine. This may include extreme temperatures, alcohol, emotional stress, insect bites, and certain medications. For example, nonsteroidal anti-inflammatory drugs (NSAIDs) should be used with caution, as these drugs may trigger the release of histamine and can cause severe reactions or an attack.

Doctors may also recommend the following treatments to help relieve mastocytosis symptoms.

  • Antihistamines. Antihistamines help relieve itching, flushing, and other skin reactions.

  • Steroids. Steroids may be used to reduce the size of skin lesions.

  • Epinephrine. People with mastocytosis often carry a syringe of epinephrine, a hormone made by the adrenal glands, in case of a severe allergic reaction.

  • Other medications. Other medications can help relieve diarrhea and stomach pain.

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


Surgery may be performed to remove lesions, such as a mastocytoma. Many surgeries on the skin can be performed quickly and easily. Learn more about the basics of surgery.

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 healthy cells.

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. 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.

Tyrosine kinase inhibitors are a type of targeted therapy. For mastocytosis, the target is the unique protein called the c-kit tyrosine kinase receptor (see the Risk Factors section). Treatment with tyrosine kinase inhibitors, including dasatinib (Sprycel), midostaurin (PKC412, still being tested in clinical trials), and less commonly imatinib (Gleevec) and nilotinib (Tasigna), may be considered for patients with mast cells that have a mutation in the c-kit tyrosine kinase receptor. See the Latest Research section for more information. Patients with more advanced systemic mastocytosis should be tested for this genetic mutation. 

Talk with your doctor about possible side effects for a specific targeted therapy and how they can be managed. Learn more about the basics of targeted treatments.

Stem cell transplantation/bone marrow transplantation

A stem cell transplant is a medical procedure in which unhealthy bone marrow is replaced by highly specialized cells, called hematopoietic stem cells, that develop into healthy bone marrow. Hematopoietic stem cells are blood-forming cells 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 stem cells in the blood that are typically being transplanted, not the actual bone marrow tissue.

There are two types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO). ALLO uses donated stem cells, while AUTO uses the patient’s own stem cells. In both types, the goal is to destroy all unhealthy cells in the marrow, blood, and other parts of the body using high doses of chemotherapy and/or radiation therapy and then allow replacement blood stem cells to create healthy bone marrow.

Stem cell transplantation is not used frequently as a treatment for mastocytosis because treatment results are inconsistent and there are significant risks associated with this treatment approach. Learn more about the basics of bone marrow and stem cell transplantation.

Chemotherapy for mastocytosis-related cancer

Chemotherapy is sometimes recommended if mastocytosis becomes cancerous. Chemotherapy is the use of drugs to destroy cancer cells, usually by stopping the cancer cells’ ability to grow and divide. 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.

Systemic chemotherapy gets into the bloodstream to reach cancer cells throughout the body. Common ways to give chemotherapy include an intravenous (IV) tube placed into a vein using a needle or in a pill or capsule that is swallowed (orally).

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, hair loss, loss of appetite, and diarrhea. These side effects usually go away once treatment is finished.

Learn more about the basics of chemotherapy and preparing for treatment. 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. Learn more about your prescriptions by using searchable drug databases.

Getting care for side effects

Mastocytosis treatment often causes side effects. In addition to treatment to manage the condition, an important part of treatment is relieving these 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 is any treatment that focuses on reducing symptoms (see section above) and treatment side effects, improving quality of life, and supporting patients and their families. Any person, regardless of age or type of disease, may receive palliative care. It works best when palliative care is started as early as needed in the treatment process.

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.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional support, and other therapies. Talk with your doctor about the goals of each treatment in your 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. 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 can be addressed as quickly as possible. Learn more about palliative care.

The next section in this guide is About Clinical Trials, and it offers more information about research studies that are focused on finding better ways to care for people with mastocytosis. Or, use the menu on the side of your screen to choose another section to continue reading this guide.