Learn The BasicsThere is currently no cure for systemic mastocytosis (SM). Therefore, the treatment options that are available focus on managing disease symptoms.
The type of treatment will depend on the type of SM and the severity of the symptoms. Treatment usually involves controlling triggers, regular medical care and routine monitoring.
Controlling triggers
The symptoms of SM may become worse when certain triggers activate mast cells. These are called non-immunoglobulin (Ig)E mechanisms and may include alcohol, certain foods, certain medications such as opioids and nonsteroidal anti-inflammatory drugs (NSAIDs), sudden changes in temperature, exercise, insect venom and stress. These triggers and their severity vary for every patient.
Identifying and avoiding these triggers can help keep the symptoms of the disease under control.
Treatment for milder subtypes
There are several medications that can help manage the symptoms of SM.
These include antacids or H2 blockers to treat stomach problems, antihistamines for itching, flushing and other skin reactions, bisphosphonates to treat osteoporosis, corticosteroids to help reduce inflammation, monoclonal antibodies such as anti-immunoglobulin E antibodies and pain medication. These treatment options are mainly used in patients with milder forms of the disease, such as indolent systemic mastocytosis (ISM) and smoldering systemic mastocytosis (SSM).
Mast cell stabilizers like cromolyn sodium and ketotifen together with agents that inhibit leukotriene may also be beneficial to treat the milder forms of the disease.
Treatment for more severe subtypes
For the treatment of more severe forms of the disease such as aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL), more aggressive treatments may be used.
These may include therapies that target mast cells or tyrosine kinase inhibitors that target the KIT gene, which is mutated in most SM cases, thereby reducing both the number of mast cells in the body and the severity of disease symptoms.
Other options for the treatment of advanced SM include cladribine, a purine analog showing activity against neoplastic mast cells and interferon alpha. This is considered the first-line cytoreductive therapy in symptomatic SM.
In case of advanced disease, chemotherapy may also be considered. These drugs will target and destroy the mast cells.
When other treatment options are not effective, a stem cell transplant may be considered to treat patients with SM. The treatment is usually preceded by chemotherapy to destroy the blood cells followed by the introduction of stem cells to form new healthy blood cells.
Treatment for anaphylaxis
To treat sudden attacks of anaphylaxis, epinephrine may be needed, and patients should carry an EpiPen at all times. They may also want to consider wearing a medical alert bracelet showing they have SM in case of an allergic reaction.
Regular monitoring
A large part of SM treatment and care is regular monitoring. The status of the condition can be monitored with regular urine and blood tests that a patient may collect at home or in a medical facility when their symptoms flare up.
Patients’ bone density should also be monitored regularly so that complications such as osteoporosis can be detected as quickly as possible.
Clinical trials
Since there are no curative treatments for SM, some patients may have the opportunity to participate in clinical trials testing potential new therapies.
