Treatment for Mastocytosis
This section tells you the treatments that are the standard of care for mastocytosis. “Standard of care” means the best treatments known. When making treatment plan decisions, patients are encouraged to consider clinical trials as an option. A clinical trial is a research study that tests a new approach to treatment. Doctors want to learn whether the new treatment is safe, effective, and possibly better than the standard treatment. Clinical trials can test a new drug, a new combination of standard treatments, or new doses of standard drugs or other treatments. Your doctor can help you consider all your treatment options.
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. Cancer care teams include a variety of other health care professionals, such as physician assistants, nurses, social workers, pharmacists, counsellors, dietitians, and others.
There is no cure for mastocytosis, although several treatments can be used to relieve symptoms and remove a mastocytoma. 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. Talk with your health care team about the goals of each treatment and what you can expect while receiving the treatment.
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 because 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 they need to treat a severe allergic reaction.
- Other medications. Other medications can help relieve diarrhoea 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. Before surgery, talk with your health care team about the possible side effects from the specific surgery you will have.
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. Treatment with tyrosine kinase inhibitors, including dasatinib (Sprycel), midostaurin (PKC412, recently approved by the FDA for the treatment of systemic mastocytosis), 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. Patients with more advanced systemic mastocytosis should be tested for this genetic mutation.
Talk with your health care team about possible side effects for a specific targeted therapy and how they can be managed.
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.
Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the results of any previous treatment and the patient’s age and general health.
There are 2 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.
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 ending 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 haematologist, 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, or schedule, usually consists of a specific number of cycles given over a set period of time. A patient may receive 1 drug at a time or combinations of different drugs given 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 diarrhoea. These side effects usually go away after 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.
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 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. Talk with your health care team 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. 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.
TAMS wish to acknowledge the American Society of Clinical Oncology and Cancer.Net as the original authors of the above information. View original source on Cancer.Net.