ON THIS PAGE: You will learn about the different types of treatments doctors use for people with a desmoid tumor. Use the menu to see other pages.
This section explains the types of treatments, also known as therapies, that are the standard of care for desmoid tumors. “Standard of care” means the best treatments known. Information in this section is based on medical standards of care for a desmoid tumor in the United States. Treatment options can vary from one place to another.
When making treatment plan decisions, you are encouraged to discuss with your doctor whether clinical trials offer additional options to consider. A clinical trial is a research study that tests a new approach to treatment. Doctors learn through clinical trials whether a 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 for all stages of tumors. 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.
How a desmoid tumor is treated
For a desmoid tumor, different types of doctors who specialize in tumors, called oncologists, often work together to create a patient’s overall treatment plan that combines different types of treatments. This is called a multidisciplinary team. Tumor care teams include other health care professionals, such as physician assistants, nurse practitioners, oncology nurses, social workers, pharmacists, counselors, dietitians, physical therapists, occupational therapists, and others. Because desmoid tumors are rare, it is important for your treatment plan to be developed by a team that specializes in sarcomas. Learn more about the clinicians who provide cancer care.
Treatment options and recommendations depend on several factors, including the:
Growth rate of the desmoid tumor
Whether the desmoid is causing symptoms
Possible side effects of treatment
Patient’s age, preferences, and overall health
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 conversations 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 important for desmoid tumors because there are different treatment options. Learn more about making treatment decisions.
The common types of treatments used for a desmoid tumor are described below. Your care plan may also include treatment for symptoms and side effects, an important part of tumor care.
Active surveillance may be an option for people with desmoid tumors that are not causing any symptoms or entangling with vital organs. This approach is sometimes called “watchful waiting.” While some desmoid tumors can grow quickly, others grow slowly, stay the same size, or shrink without active treatment. During active surveillance, the patient is monitored closely with periodic examinations and tests (see Diagnosis) to watch for changes in the tumor, along with appropriate treatments to manage symptoms. Active treatment may begin if the tumor progresses, causes symptoms, or is getting close to vital structures (see Symptoms and Signs).
Surgery is the removal of the tumor and some surrounding healthy tissue during an operation. Surgery used to be the standard treatment for desmoid tumors. Today, many doctors are considering more conservative, non-surgical, treatment options first. That is because a desmoid tumor frequently comes back at or near the original tumor site after surgery. Recurrence rates have been reported to be as high as 70% by some researchers. In addition, desmoid tumors that recur after surgery are often more aggressive. However, surgery may sometimes still be a successful treatment option for some patients.
Before surgery, it is important to have a biopsy and appropriate imaging to confirm the diagnosis of a desmoid tumor (see Diagnosis). Surgical oncologists and orthopedic oncologists are doctors who specialize in treating desmoid tumors and other sarcomas using surgery.
The surgeon's goal is to remove the entire tumor and enough normal tissue surrounding it to obtain a clean margin around the tumor. A clean margin is when there are no tumor cells visible at the borders of the surgical specimen. This is currently the best method available to ensure that there are no tumor cells left in the area from which the tumor was removed. But because desmoid tumors can intertwine with nearby tissue and structures, it may be difficult or even impossible for the surgeon to identify where the tumor starts and stops. Radiation therapy (see below) may be used after surgery to destroy any remaining tumor cells, if the patient did not receive radiation therapy before surgery. This type of treatment after surgery is called “adjuvant therapy.”
Before surgery, talk with your health care team about the risk of the tumor returning. Certain types of desmoid tumors are more likely to recur after surgery than others. You should also discuss possible side effects from the specific surgery you will have, including the recovery period. Learn more about the basics of cancer surgery.
Radiation therapy is the use of high-energy x-rays or other particles to destroy tumor cells. A doctor who specializes in giving radiation therapy to treat a tumor is called a radiation oncologist.
Many sarcoma experts do not recommend radiation therapy for desmoid tumors. This is because, in rare cases, radiation therapy can cause cancerous tumors or new and more aggressive sarcomas. In the unlikely event that this happens, a cancer may develop 7 to 20 years after radiation therapy. It is important to talk with your health care team about this risk and other potential side effects of radiation therapy before you have this treatment (see “Physical, emotional, social, and financial effects of a tumor”). Since desmoid tumors are rare, if radiation therapy is being considered it is very important to talk with a radiation oncologist who has experience treating them.
Learn more about radiation therapy.
Cryosurgery, also called cryotherapy or cryoablation, is a localized treatment that uses extremely cold substances to freeze and kill tumor cells. It may be used if surgery is not an option. The therapy appears to be more effective in controlling smaller extra-abdominal tumors than larger extra-abdominal tumors that have invaded vital structures. Typically, several cycles of cryosurgery are done. One recent study found that cryosurgery stopped tumor progression in almost 90% of patients. In other patients, the tumor was completely destroyed.
Radiofrequency thermal ablation (RFA) uses heat to destroy tumor cells. Like cryosurgery (see above), it is a minimally invasive treatment option that may be useful for treating desmoid tumors that cannot be removed with surgery.
Therapies using medication
The treatment plan may include medications to destroy tumor cells. Medication may be given through the bloodstream to reach tumor cells throughout the body. When a drug is given this way, it is called systemic therapy. Medication may also be given locally, which is when the medication is applied directly to the tumor or kept in a single part of the body.
This treatment is generally prescribed by a medical oncologist, a doctor who specializes in treating tumors with medication.
Medications are often given through an intravenous (IV) tube placed into a vein using a needle or as a pill or capsule that is swallowed (orally). If you are given oral medications to take at home, be sure to ask your health care team about how to safely store and handle them.
The types of medications used for desmoid tumors include:
Nonsteroidal anti-inflammatory drugs
Each of these types of therapies is discussed below in more detail. A person may receive 1 type of medication at a time or a combination of medications given at the same time. They can also be given as part of a treatment plan that includes surgery and/or radiation therapy. Some of these may be used as first-line treatment, if surgery is not recommended.
The medications used to treat tumors 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 medications used to treat tumors, causing unwanted side effects or reduced effectiveness. Learn more about your prescriptions by using searchable drug databases.
Nonsteroidal anti-inflammatory drugs (NSAIDs) can sometimes slowly shrink a desmoid tumor, as well as relieve associated pain and swelling. However, data from recent clinical trials have shown that it is less clear whether NSAIDs may actively shrink tumors. These drugs may include celecoxib (Celebrex), ibuprofen (multiple brand names), indomethacin (Tivorbex), naproxen (multiple brand names), or sulindac (available as a generic drug). NSAIDs may be used in combination with other treatments, such as hormone therapy.
Research shows that the hormone estrogen appears to contribute to desmoid tumor growth. Hormone therapy may be used to control tumor growth. Tamoxifen (available as a generic drug) is the most commonly used hormone therapy for desmoid tumors. Tamoxifen blocks a cell’s ability to use estrogen, which may stop or slow the growth of tumor cells that depend on it. However, recent clinical trial data has shown that patients receiving a placebo also had spontaneous regression of desmoid tumors, which makes it less clear that tamoxifen is able to actively shrink tumors. The side effects of tamoxifen may include hot flashes, vaginal discharge, sexual side effects, mood changes, and a higher risk of developing uterine cancer, blood clots, and stroke. Hormone therapy may be used in combination with NSAIDs or other treatments. Patients diagnosed with a desmoid tumor should stop estrogen replacement therapy if they receive hormone therapy for a desmoid tumor. Learn more about the basics of hormone therapy.
Targeted therapy is a treatment that targets the tumor’s specific genes, proteins, or the tissue environment that contributes to its growth and survival. This type of treatment blocks the growth and spread of tumor cells and limits damage to healthy cells.
For progressing desmoid tumors that cannot be treated with surgery, a medication called nirogacestat (Ogsiveo) may be recommended. This is the first drug approved by the U.S. Food and Drug Administration (FDA) specifically for treating desmoid tumors. Nirogacestat works by blocking gamma secretase. Gamma secretase activates a protein called Notch, and researchers suspect that the Notch protein helps desmoid tumors grow. The most common side effects of nirogacestat include diarrhea, rash, nausea, fatigue, mouth sores, headache, abdominal pain, cough, hair loss, infections of the respiratory tract, and shortness of breath. Women who took this medication also experienced fertility problems linked to the function of the ovaries. For the majority of these women, the fertility problems went away after treatment was stopped.
Tyrosine kinase inhibitors (TKIs) are drugs that target and block specific proteins called kinases that contribute to the tumor’s growth and survival. Some TKIs may be useful in treating a progressing or recurrent desmoid tumor by shrinking it or halting its growth.
A global phase III clinical trial studied using a TKI called sorafenib (Nexavar) to treat desmoid tumors. Sorafenib is currently used to treat other cancers, including liver, thyroid, and kidney cancers. In this clinical trial, patients were randomly assigned to 2 groups, either given sorafenib or a placebo tablet. Of those who took sorafenib in the clinical trial, 33% of patients had their desmoid shrink and 87% had their disease stopped for more than 1 year. It is important to note that approximately 20% of patients who received placebo also had their desmoids shrink. In this study, 18% of patients who took sorafenib had to stop taking the drug because of side effects. Common side effects of sorafenib include high blood pressure, fatigue, diarrhea, decrease in appetite, nausea, vomiting, rash, dry or itchy skin, and mouth ulcers. This drug is taken as a pill by mouth.
Another commonly used TKI in desmoid tumor treatment is imatinib (Gleevec). This drug is also taken as a pill by mouth. It is approved by the FDA for a type of soft-tissue sarcoma called gastrointestinal stromal tumor (GIST), as well as other conditions. The most common side effects of imatinib are fluid accumulation, rash, nausea, diarrhea, fatigue, and minor muscle aches. Serious but relatively rare side effects include bleeding and inflammation of the liver. These side effects, and side effects of other TKIs, can often be successfully managed by your doctor.
In addition, pazopanib (Votrient) is a multitargeted TKI that is sometimes used to treat desmoid tumors. In a recent phase II clinical trial, the tumors of patients with advanced desmoid tumors who received pazopanib as a pill by mouth showed more shrinkage compared to a combined chemotherapy treatment of methotrexate and vinblastine (see “Chemotherapy”). The drug received FDA approval to treat advanced soft-tissue sarcomas in 2012. The most common side effects of pazopanib are diarrhea, fatigue, loss of coloration in the hair and skin, and high blood pressure.
Learn more about the basics of targeted treatments.
Chemotherapy is medication that kills tumor cells and keeps them from growing, dividing, and making more cells. Tumor cells grow and divide faster than normal cells. However, normal cells also grow and divide, so the side effects of chemotherapy are due to the treatment’s effects on normal cells that are growing and dividing.
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 a combination of different drugs given at the same time. Chemotherapy for a desmoid tumor can usually be given as an outpatient treatment, which means you don’t need to stay overnight at the hospital.
Different drugs are used to treat desmoid tumors. Some types of chemotherapy that might be used alone or in combination for desmoid tumor include:
Liposomal doxorubicin (Doxil)
Doxorubicin (available as a generic drug), used alone or combined with dacarbazine
Dacarbazine (available as a generic drug), only used in combination with doxorubicin
Vinorelbine (available as a generic drug)
Chemotherapy may be used to shrink an aggressive, organ-threatening desmoid tumor or one that is causing symptoms. The decision to use chemotherapy should be made after discussion with an experienced sarcoma expert.
The side effects of chemotherapy depend on the individual drug 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 after treatment is finished. In rare cases, there are long-term problems that affect the heart or kidneys or cause cancer.
Learn more about the basics of chemotherapy.
A desmoid 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 and supportive care. It is an important part of your care that is included along with treatments intended to slow, stop, or eliminate the tumor.
Palliative and supportive 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 tumor may receive this type of care. And it often works best when it is started right after a tumor diagnosis. People who receive palliative and supportive care along with treatment for the tumor often have less severe symptoms, better quality of life, and report that they are more satisfied with treatment.
Supportive treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional and spiritual support, and other therapies. You may also receive palliative treatments, such as chemotherapy, surgery, or radiation therapy, to improve symptoms.
Before treatment begins, talk with your doctor about the goals of each treatment in the recommended treatment plan. You should also talk about the possible side effects of the specific treatment plan and palliative and supportive care options. Many patients also benefit from talking with a social worker and participating in support groups. Ask your doctor about these resources, too.
Cancer care is often expensive, and navigating health insurance can be difficult. Ask your doctor or another member of your health care team about talking with a financial navigator or counselor who may be able to help with your financial concerns.
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.
A remission is when tumor cells cannot be detected in the body and there are no symptoms. This may also be called having “no evidence of disease” or NED. Sometimes desmoid tumors will shrink on their own and will not require further treatment. If this happens, it is called spontaneous regression.
Often, desmoid tumors will shrink and/or become inactive but a mass is still present. This mass is like a scar and does not require treatment. However, some patients may continue to have pain or other symptoms due to the location of the scar.
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 or becomes active again after the original treatment, it is called a recurrent tumor. Desmoid tumors do not metastasize to other parts of the body, but they can recur locally. This means they come back in the same place they started.
If a recurrence happens, a new cycle of testing will begin to learn as much as possible about it. 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, chemotherapy, targeted therapy, and radiation therapy, but 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 a recurrent desmoid tumor. Whichever treatment plan you choose, palliative and supportive care will be important for relieving symptoms and side effects.
People with a recurrent desmoid tumor sometimes experience emotions such as disbelief or fear. You are encouraged to talk with your health care team about these feelings and ask about support services to help you cope. Learn more about dealing with cancer recurrence.
Recovery from a desmoid tumor is not always possible. If the tumor cannot be removed, it can often still be controlled for a very long time. It is important to understand that patients can live with a tumor in their body as long as it does not affect the function of a major organ. Therefore, the goal of treatment is to control the tumor and preserve organ function.
If the tumor can no longer be controlled, it is called end stage or terminal. This is very rare in patients with a desmoid tumor. But if it does happen, this diagnosis is stressful and can be difficult to discuss. However, it is important to have open and honest conversations with your 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.
Planning for your future care and putting your wishes in writing is important, especially at this stage of disease. Then, your health care team and loved ones will know what you want, even if you are unable to make these decisions. Learn more about putting your health care wishes in writing.
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 tumor. Use the menu to choose a different section to read in this guide.