Gastrointestinal Stromal Tumor - GIST: Types of Treatment

Approved by the Cancer.Net Editorial Board, 02/2020

ON THIS PAGE: You will learn about the different types of treatments doctors use for people with a GIST. Use the menu to see other pages.

This section explains the types of treatments that are the standard of care for GIST. “Standard of care” means the best treatments known. When making treatment plan decisions, you 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. Clinical trials are an option to consider for treatment and care for all stages of cancer. Your doctor can help you consider all your treatment options. Learn more about clinical trials in the About Clinical Trials and Latest Research sections of this guide.

Treatment overview

In cancer care, 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. It is important for people with a GIST to have their treatment plan determined by a medical team that specializes in sarcomas, as it has been shown that this improves a person's chances of survival. Cancer care teams include a variety of other health care professionals, such as physician assistants, nurse practitioners, oncology nurses, social workers, pharmacists, counselors, dietitians, and others.

Descriptions of the common types of treatments used for GIST are listed below. Your care plan also includes treatment for symptoms and side effects, an important part of cancer care.

Treatment options and recommendations depend on several factors, including the type and stage of tumor, possible side effects, the patient’s preferences, and the patient’s overall health. The doctor will look at many factors to determine the best treatment, including the tumor’s:

  • Size

  • Number of dividing cells

  • Genetic makeup

  • Primary location

  • Whether it has already spread

  • Whether the tumor ruptured, either on its own or as a result of surgery

Take time to learn about all of your treatment options and be sure to ask questions about things that are unclear. Talk with your doctor about the goals of each treatment and what you can expect while receiving the treatment. These types of talks are called “shared decision making.” Shared decision making is when you and your doctors work together to choose treatments that fit the goals of your care. Shared decision making is particularly important for GIST because there are different treatment options. Learn more about making treatment decisions.

Surgery

Surgery is the removal of the tumor and some surrounding healthy tissue during an operation. A surgical oncologist is a doctor who specializes in treating cancer by surgery.

For people with a localized GIST, surgery is the standard treatment and should be performed whenever possible. If the GIST cannot be completely removed by surgery, such as if it has spread somewhere else, it often cannot be cured. However, surgery may be considered if there is only a limited amount of disease.

Before surgery, talk with your health care team about the possible side effects from the specific surgery you will have. Learn more about the basics of cancer surgery.

Therapies using medication

Systemic therapy is the use of medication to destroy tumor cells. This type of medication is given through the bloodstream to reach tumor cells throughout the body. Systemic therapies are generally prescribed by a medical oncologist, a doctor who specializes in treating cancer with medication.

Common ways to give systemic therapies include an intravenous (IV) tube placed into a vein using a needle or in a pill or capsule that is swallowed (orally).

Targeted therapy is the type of systemic therapy used for GIST. Different targeted therapies are discussed below in more detail. A person may receive 1 type of systemic therapy at a time or a combination of systemic therapies given at the same time. They can also be given as part of a treatment plan that includes surgery and/or radiation therapy.

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. It is also important to let your doctor know if you are taking any other prescription or over-the-counter medications or supplements. Herbs, supplements, and other drugs can interact with cancer medications. Learn more about your prescriptions by using searchable drug databases.

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 and limits damage to healthy cells.

Not all tumors have the same targets. Because some of these genetic changes in the tumor may mean the drugs described below may not work well, your doctor may run tests to identify the genes, proteins, and other factors in your tumor. This helps doctors better match each patient with the most effective treatment whenever possible. In addition, research studies continue to find out more about specific molecular targets and new treatments directed at them.

Tyrosine kinase inhibitors (TKIs) are drugs that target specific proteins called kinases that contribute to the tumor’s growth and survival. TKIs are often used to treat GIST.

The drugs described in this section are listed in the order in which they were approved by the U.S. Food and Drug Administration (FDA). The order in which they are used may depend on factors discussed above. Learn more about the basics of targeted treatments.

Talk with your doctor about the possible side effects for each medication and how they can be managed.

Imatinib (Gleevec). This was the first targeted therapy approved for GIST by the FDA. It is often the first drug used to treat GIST. Since this drug became available, the prognosis for patients with a GIST has improved a lot.

Research is being done to find out how long imatinib should be given after surgery to help delay or prevent a recurrence, and imatinib is the only drug approved for this use. If a GIST has spread to other parts of the body, imatinib is taken for the rest of the patient’s life to help control the tumor.

The usual dose of imatinib is 400 milligrams (mg) daily. For some patients, the dose may be raised to 800 mg daily, especially for those who have an exon 9 genetic mutation in the KIT gene.

Imatinib does have some common side effects that are often successfully managed. The dose can be adjusted to reduce side effects, or other medications can be added to the treatment plan to manage side effects. The most common side effects of imatinib are fluid accumulationrashnauseadiarrheafatigue, and minor muscle aches. Serious but relatively rare side effects include bleeding and inflammation of the liver. Many of these side effects can be managed with the help of the health care team. Also, some side effects get better over time. People who have serious side effects may take a lower dose of imatinib and still benefit from it. If you want to stop treatment because of the side effects, talk with your health care team first to learn whether this is the best option.

Sunitinib (Sutent). Sunitinib is a TKI that works in many different ways to slow tumor growth. Sunitinib also blocks both KIT and the process of making new blood vessels, which is called angiogenesis. Sunitinib is used to treat a GIST that continues to grow even after treatment with imatinib. Sunitinib may also be used when the side effects of imatinib are too serious.

The side effects of sunitinib include fatigue, diarrhea, nausea and vomiting, heartburn, changes in taste, high blood pressure, low blood counts, and changing skin color.

Regorafenib (Stivarga). Regorafenib is a TKI that works in many different ways to slow tumor growth, including blocking the KIT molecule and blocking blood vessel growth into tumors. It was approved in 2013 for people with later-stage GIST that cannot be surgically removed and when both imatinib and sunitinib did not work or caused severe side effects.

The side effects of regorafenib include fatigue, nausea, hand-foot syndrome, high blood pressure, diarrhea, and inflammation of the liver.

Larotrectinib (Vitrakvi). A small percentage of sarcomas, less than 1%, have a mutation in the neurotrophic receptor tyrosine kinase (NTRK) gene. Larotrectinib (Vitrakvi) is an NTRK inhibitor that is now approved for any cancer that has a specific mutation in the NTRK gene. These NTRK mutations are not found in the most common types of GIST, including those with KITPDGFRSDH, and RAF mutations.

The most common side effects include fatigue, nausea, dizziness, vomiting, increased liver enzymes, cough, constipation, and diarrhea.

Avapritinib (Ayvakit). Avapritinib is a TKI that also targets tumors with a specific mutation in the platelet-derived growth factor receptor alpha (PDFGRAexon 18 gene. It is approved by the FDA to treat metastatic GISTs that have a mutation in PDGFRA exon 18 that cannot be removed with surgery.

The common side effects of avapritinib were swelling, nausea, fatigue, problems with attention, memory, or thinking, vomiting, loss of appetite, diarrhea, hair color changes, increased production of tears from the eyes, abdominal pain, constipation, rash, and dizziness.

Ripretinib (Qinlock). Ripretinib is a TKI approved to treat an advanced GIST after 3 or more previous treatments with TKIs, including imatinib, have not stopped the tumor from growing and spreading.

The common side effects of ripretinib include hair loss, fatigue, nausea, abdominal pain, constipation, muscle pain, diarrhea, loss of appetite, hand-foot syndrome, and vomiting. There is also a risk of serious side effects, including skin cancer, high blood pressure, and heart problems.

Chemotherapy

Chemotherapy is the use of drugs to destroy cancer cells, usually by keeping the cancer cells from growing, dividing, and making more cells. However, standard chemotherapy is not effective for treating GIST and should not be used.

Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles to destroy cancer cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist. The most common type of radiation treatment is called external-beam radiation therapy, which is radiation given from a machine outside the body. When radiation treatment is given using implants, it is called internal radiation therapy or brachytherapy.

A radiation therapy regimen, or schedule, usually consists of a specific number of treatments given over a set period of time.

Radiation therapy is not often used for to treat people with a GIST. However, it may be used as a palliative treatment to relieve pain, such as bone pain, or to stop bleeding. Radiation therapy may damage healthy cells as well as tumor cells. Side effects from radiation therapy include tiredness, mild skin reactions, upset stomach, and loose bowel movements.

Most side effects go away soon after treatment is finished. Learn more about the basics of radiation therapy.

Physical, emotional, and social effects of cancer

A tumor and its treatment cause physical symptoms and side effects, as well as emotional, social, and financial effects. Managing all of these effects is called palliative care or supportive care. It is an important part of your care that is included along with treatments intended to slow, stop, or eliminate the cancer.

Palliative care focuses on improving how you feel during treatment by managing symptoms and supporting patients and their families with other, non-medical needs. Any person, regardless of age or type and stage of cancer, may receive this type of care. And it often works best when it is started right after a cancer diagnosis. People who receive palliative care along with treatment for the cancer often have less severe symptoms, better quality of life, and report that they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional and spiritual support, and other therapies. You may also receive palliative treatments similar to those meant to get rid of the tumor, such as drug therapy, surgery, or radiation therapy.

Before treatment begins, talk with your doctor about the goals of each treatment in the treatment plan. You should also talk about the possible side effects of the specific treatment plan and palliative care options.

During treatment, your health care team may ask you to answer questions about your symptoms and side effects and to describe each problem. Be sure to tell the health care team if you are experiencing a problem. This helps the health care team treat any symptoms and side effects as quickly as possible. It can also help prevent more serious problems in the future.

Learn more about the importance of tracking side effects in another part of this guide. Learn more about palliative care in a separate section of this website.

Treatment by stage of GIST

Different treatments may be recommended for each stage of a GIST. Your doctor will recommend a specific treatment plan for you based on the tumor's stage and other factors. Detailed descriptions of each type of treatment are provided earlier in this page. Clinical trials may also be a treatment option for each stage.

Localized GIST

A localized GIST is a tumor that has stayed in the part of the body where it began. Smaller localized GISTs may be removed completely with surgery. For some, this may be the only treatment that is needed.

If the tumor is large or has spread to nearby organs, it may first be treated with imatinib (see "Targeted therapy," above). The purpose of this approach is to shrink the tumor so it is easier to remove with surgery. This is called "neoadjuvant therapy." The surgeon will eventually try to remove the entire tumor to reduce the risk that the tumor will come back or block the GI tract.

To remove the entire tumor, the surgeon may have to remove parts of nearby organs, depending on the tumor’s location. The removal of lymph nodes is not generally needed because GISTs do not often spread to the lymph nodes. Lymph nodes are tiny, bean-shaped organs that help fight infection. A tumor that cannot be removed using surgery is called "unresectable." The doctor will recommend targeted therapy for an unresectable localized GIST.

GIST has the ability to come back after being surgically removed and can spread to other organs. Some GISTs have a higher risk of returning or spreading than others. After surgery for a localized GIST, people who may have a high risk of recurrence often receive imatinib for at least 3 years to help prevent or delay it from coming back. This type of treatment after surgery is called "adjuvant therapy."

Metastatic GIST

If a GIST has spread to another part in the body from where it started, doctors call it metastatic. If this happens, it is a good idea to talk with doctors who have experience in treating it. Doctors can have different opinions about the best standard treatment plan. Clinical trials might also be an option. Learn more about getting a second opinion before starting treatment, so you are comfortable with your chosen treatment plan. In many instances, people with metastatic GIST are able to live normal lives for many years, being treated only with pills and regular hospital visits. Learn more about living with chronic cancer.

Depending on previous treatment, your treatment plan may include surgery, a higher dose of a TKI, or treatment through a clinical trial. Your doctor may continue to recommend imatinib, regorafenib, or sunitinib, even if they are not working perfectly. They may also recommend going back to 1 of these drugs (usually imatinib) after the others have been tried and/or after they have stopped working. In general, some treatment with targeted therapy for GIST is better than no treatment at all. Palliative care will also be important to help relieve symptoms and side effects.

For most people, a diagnosis of metastatic GIST is very stressful and difficult. You and your family are encouraged to talk about how you feel 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.

Remission and the chance of recurrence

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

A remission may be temporary or permanent. This uncertainty causes many people to worry that the tumor will come back. While many remissions are permanent, it is important to talk with your doctor about the possibility of the tumor returning. Understanding your risk of recurrence and the treatment options may help you feel more prepared if the tumor does return. Learn more about coping with the fear of recurrence.

If the tumor returns after the original treatment, it is called a recurrent tumor. It may come back in the same place (called a local recurrence), nearby (regional recurrence), or in another place (distant recurrence).

When this occurs, a new cycle of testing will begin again to learn as much as possible about the recurrence. After this testing is done, you and your doctor will talk about the treatment options. Often the treatment plan will include the treatments described above, such as surgery and targeted therapy. However, they may be used in a different combination or given at a different pace. Your doctor may suggest clinical trials that are studying new ways to treat this type of recurrent tumor. Whichever treatment plan you choose, palliative care will be important for relieving symptoms and side effects.

People with a recurrent or metastatic GIST often experience emotions such as disbelief or fear. You are encouraged to talk with the health care team about these feelings and ask about support services to help you cope. Learn more about dealing with a recurrence.

If treatment does not work

Recovery from a GIST is not always possible. If the growth of the tumor cannot be controlled, you may not ever recover from the disease. This condition is called terminal.

This diagnosis is stressful, and for many people, advanced cancer is difficult to discuss. However, it is important to have open and honest conversations with health care team to express your feelings, preferences, and concerns. The health care team has special skills, experience, and knowledge to support patients and their families and is there to help. Making sure a person is physically comfortable, free from pain, and emotionally supported is extremely important.

People who are expected to live less than 6 months may want to consider hospice care. Hospice care is designed to provide the best possible quality of life for people who are near the end of life. You and your family are encouraged to talk with the health care team about hospice care options, which include hospice care at home, a special hospice center, or other health care locations. Nursing care and special equipment can make staying at home a workable option for many families. Learn more about advanced care planning.

After the death of a loved one, many people need support to help them cope with the loss. Learn more about grief and loss.

The next section in this guide is About Clinical Trials. It offers more information about research studies that are focused on finding better ways to care for people with a GIST. Use the menu to choose a different section to read in this guide.