Types of Treatment for Desmoid Tumor

Executive Summary

The different types of standard treatments for desmoid tumors are explained in detail. Standard treatments are used along with the integration of effective treatment planning. Clinical trials are used as an innovative treatment approach that combines different therapies and drugs for treating desmoid tumors. The doctor may recommend a colonoscopy to observe colon polyps or communicate with a genetic counsellor to learn whether the tumor is associated with FAP syndrome. The family members would also be recommended to carry out certain genetic tests. Active surveillance is an option for desmoid tumor patients that do not cause any side effects or mess up with the internal organs. Other common treatment for desmoid tumor involves surgery, radiation therapy, cryoablation (Radiofrequency thermal ablation (RFA)), therapies using medications, hormone therapy (non-steroid anti-inflammatory drugs), targeted therapy, and chemotherapy.

Palliative care is being integrated to improve the patients’ quality of life. The patients continue to have regular tests, scans, and physical examinations even after successful treatment to look for recurrence in the later phase after the treatment ends. The clinical trials tests are conducted to check the viability of various treatment options for dealing with recurrent desmoid tumors.

Treatment of Desmoid Tumors

Desmoid tumor is a rare disease condition. These tumors generally occur from cells called fibroblasts. These tumors can occur in any part of the body, but they are mostly seen in the abdomen, upper arms or legs. Doctors use different procedures and techniques to treat patients with desmoid tumors. Doctors use the standard care (best possible treatment option for desmoid tumor) available to treat such tumors. While deciding the treatment strategy for desmoid tumor, patients are encouraged to consider clinical trials as an option. A clinical trial refers to a detailed research study that focuses on testing new approaches to treat better and cure disease. Sometimes the treatment strategies approved in a clinical trial must be much better than the already existing treatment to treat a particular disease condition. Patients are urged to seek the help and guidance of their doctors to arrive at the apt clinical trial or in considering all possible treatment options to cure the condition.  

For treating a condition like desmoid tumors, doctors from different areas of expertise work together to design a suitable treatment plan for desmoid tumor. A multidisciplinary team will consist of different doctors to carry out the treatment. The team will consist of various healthcare professionals, including oncologists, physician assistants, oncology nurses, nurse practitioners, pharmacists, dieticians, counsellors, and social workers. In addition to treatment for desmoid tumor, the healthcare team will also focus on providing care and support to the family members associated with the patient. Since desmoid tumors are rare, your treatment plan must be designed by a team that specializes in dealing with sarcomas.

If you are diagnosed with desmoid tumors upon diagnosis, learn whether any of your family members had a history of having these tumors, multiple colon polyps or colorectal cancer in the past. Around 10% to 20% of people with FAP, a genetic syndrome, develops a desmoid tumor. While planning treatment for desmoid tumor, the doctor may recommend a colonoscopy to observe colon polyps or talk with a genetic counsellor to learn whether the tumor is associated with FAP syndrome. If the disease condition is associated with FAP, there is a high chance that other family members may also develop desmoid tumors; hence the family members would also be recommended to carry out certain genetic tests.

Described below is a list of available treatment options to treat desmoid tumors ^​1​. Treating and curing the symptoms and side effects is important for tumor treatment and care.

The healthcare team will consider the following aspects before deciding the apt treatment strategy for a patient’s desmoid tumor condition.

• The growth rate of the tumour
• Whether the tumour growth is causing any signs or symptoms
• Probable side effects of treatment
• Patient’s overall health condition, age and preferences.

Patients should take time to learn about all the available treatment options for desmoid tumors and be sure to ask questions and clear doubts about things that are unclear and appears complex. Ask your doctor about the aim of each treatment for desmoid tumor and what outcome you can expect while receiving the treatment. This is what is called “shared decision making.” Shared decision making helps the patients to have informed choices regarding treatment. This will help the healthcare team choose and plan a treatment strategy that best fits your aims and expectations.

Active surveillance

Active surveillance may be an option for desmoid tumor patients that do not cause any side effects or mess up with the internal organs. The approach is sometimes called “watchful waiting.” Some desmoid tumors tend to increase, whereas others grow at a languid pace, stay the same size or shrink even without active treatment. The patient is closely monitored with periodic tests and examinations during active surveillance to look for any changes or variations in the tumor. Active treatment may start if the tumor progresses to grow, cause signs and symptoms, or invade the body’s vital organs.

Surgery for desmoid tumour

A Surgery is a procedure where the tumor and some surrounding healthy tissues are removed via an operation. A surgical procedure is considered a standard treatment procedure for treating desmoid tumors. But today, healthcare professionals are primarily considering better, conservative, non-surgical treatment options. This is because desmoid tumors are seen to recur back in the body, even after successful surgical removal. Studies show that the recurrence rate of desmoid tumors is around 70%. In addition, desmoid tumors that recur back in the body after surgery are seen to be more aggressive. But in some cases, surgery proves to be a successful treatment option.

Before a surgical procedure, doctors may recommend a biopsy and other imaging tests to confirm the tumor diagnosis. Orthopaedic oncologists and surgical oncologists are the specialists who surgically treat desmoid tumors and other sarcomas ^​2​.

During a surgical procedure, the surgeon will remove the entire tumor and a margin, which refers to the healthy tissue surrounding the tumor, to obtain a clear margin. A clear margin indicates no visible tumor cells at the border of the surgical specimen. This method ensures that no tumor cell is left behind that can cause the tumor to recur in the body again. But since desmoid tumors can intertwine with nearby structures and tissues, the surgeon will find it difficult or rather impossible to identify where the tumor begins and ends. In such cases, radiation therapy may eliminate any remaining tumor cells after surgery. This type of treatment after a surgical procedure is called “adjuvant therapy.”  

Before the surgical procedure, talk with your doctor or healthcare team about the side effects of surgery and the risk of the tumour recurring in the body. Also, inquire about the outcome of the surgery, including the recovery period.

Radiation therapy

Radiation therapy refers to the use of high energy x-rays or other particles to destroy tumour growth. A radiation oncologist is a doctor who specializes in performing this procedure. 

Since desmoid tumors are a rare disease condition, patients must talk with a radiation oncologist who has experience in treating such disease conditions before the treatment. Most sarcoma specialists do not recommend radiation therapy for treating desmoid tumors. This is because radiation therapy can cause new tumors or aggressive sarcomas to develop in the body. Radiation therapy can cause cancers to develop in people, maybe after 7 to 20 years. Hence patients must talk with the healthcare team about the probable side effects of radiation therapy before the treatment begins.

Usually, radiation therapy is recommended after a surgical procedure. And in some cases, radiation therapy alone is used to treat desmoid tumors ^​3​. The treatment procedure is widely used to treat extra-abdominal tumors. A radiation therapy schedule or regimen usually consists of several treatments given over a set period.

Cryoablation or Cryosurgery 

Cryosurgery, also called cryoablation or cryotherapy, uses liquid nitrogen to freeze and eliminate tumor cells. Cryosurgery may be used if surgery is not possible to treat the tumor. The procedure appears to be more effective in controlling smaller extra-abdominal tumors than larger extra-abdominal tumors that have invaded any vital body structures. Typically, several cycles of cryosurgery cryoablation are done as part of treatment. A recent study found that cryosurgery effectively stopped tumor progression in almost 90% of desmoid tumor patients. In other patients, the tumor was eliminated.

Radiofrequency thermal ablation (RFA)

Radiofrequency thermal ablation (RFA) uses heat to kill and eliminate cancer cells. Like cryosurgery/cryoablation, it is a minimally invasive treatment option that may effectively treat desmoid tumors that cannot be treated and removed with surgery.

Therapies using medication

Systemic therapy refers to medication or drugs to eliminate cancer cells. These drugs or medications are administered through the bloodstream to reach tumor growths or cells throughout the body. A medical oncologist usually prescribes such therapies. Systemic therapies are commonly given in two ways:

• Using an intravenous (IV) tube to administer medication into the vein using a needle.
• A capsule or pill that is swallowed or administered orally.

Following are the types of medicated therapies used to treat desmoid tumours:

• Hormone therapy
• Nonsteroidal anti-inflammatory drugs
• Targeted therapy
• Chemotherapy

A patient may receive one of these, or sometimes a combination of all these therapies simultaneously. Systemic therapies can also be given as part of the overall treatment plan, including surgery or radiation. These therapies can also be given as first-line treatment when a surgical procedure is impossible.

Many studies focus on developing efficient medications that can help treat and cure tumor conditions. Patients are encouraged to talk with their doctor about the prescribed medicines and learn and understand more about them. Ask them questions regarding the purpose, possible side effects, and their possible interactions with other treatments and medications. One should also let their doctor know about any additional treatment or medicines they are taking well before cancer treatment. This is because certain drugs, herbs can interact with cancer medications and can cause complications.

Hormone therapy

Many studies show that the hormone estrogen plays an essential role in contributing to desmoid tumor growth ^​4​. Hormone therapy could be used as a treatment procedure in cases where the tumor cannot be entirely removed via surgery. The most commonly used hormone therapy drug for desmoid tumors is Tamoxifen (Nolvadex). The hormone therapy drug Tamoxifen blocks a cell’s ability to use the hormone estrogen, thereby stopping or slowing the growth of tumor cells that depends on the hormone. The drug can cause a range of side effects like vaginal discharge, hot flashes, sexual side effects, mood changes, and patients also have a high risk of developing blood clots, uterine cancer and stroke. Hormone therapy can also be used with Nonsteroidal anti-inflammatory drugs (NSAIDs) or other treatments. Patients diagnosed with desmoid tumors should end estrogen replacement therapy if they start to receive hormone therapy as part of desmoid tumor treatment.

Nonsteroidal anti-inflammatory drugs (NSAIDs)

Nonsteroidal anti-inflammatory drugs (NSAIDs) can sometimes slowly shrink a desmoid tumor that cannot be removed entirely via surgery ^​5​. These drugs also help in relieving associated pain and swelling. The Nonsteroidal anti-inflammatory drugs include ibuprofen (multiple brand names), celecoxib (Celebrex), naproxen (numerous brand names), indomethacin (Tivorbex), or sulindac. They may be combined with other treatments, such as hormone therapy.

Targeted therapy

Targeted therapy refers to a systemic treatment that targets specific genes, proteins of the cancer cells and the surrounding tissue that contributes to tumour growth and proliferation. This therapy shuns cancer’s development and metastatic capability by causing very little damage to the surrounding healthy cells.

The targets of each tumor differ. The doctor will run various tests or scans to identify the tumor’s proteins, genes, and other characteristic features to choose the suitable treatment option. This helps the doctor to implement the most effective treatment. Many types of research are happening to devise better target therapy options and learn more about specific molecular targets. Tyrosine kinase inhibitors (TKIs) are drugs that target specific proteins called kinases that contribute to the growth and survival of tumors. This type of target therapy drug blocks the growth and spread of tumor cells, limiting the damage caused to the healthy cells. Some Tyrosine kinase inhibitors (TKIs) are seen to be effective in treating progressing or recurrent desmoid tumor growths either by shrinking or stopping its growth.

A recent phase III clinical trial evaluated the effectiveness of TKI called sorafenib (Nexavar) to treat desmoid tumor growths. The drug Sorefenib is currently used to treat thyroid, liver, and kidney cancers. In this trial, patients were randomly assigned to 2 groups and given either sorafenib or a placebo tablet. Out of the people who took sorafenib in the clinical trial, 33% saw their desmoid tumor shrinking, and around 87% experienced the disease being stopped for more than a year. It is equally important to note that about 20% of people who received placebo tablets also had their desmoid tumors shrink. But 18% of patients who took sorafenib had to stop taking the drugs due to the side effects it caused. The general side effects of the drug sorafenib include fatigue, high blood pressure, diarrhoea, decrease in appetite, nausea, vomiting, dry or itchy skin, rash, and mouth ulcers. The drug is usually taken as a pill via the mouth.

Another commonly used Tyrosine kinase inhibitor (TKI) treating in a desmoid tumor is imatinib (Gleevec). This drug is also taken orally in the form of a pill. The drug is approved by the U.S. Food and Drug Administration (FDA) for a type of soft-tissue sarcoma called gastrointestinal stromal tumor or GIST, as well as other disease conditions. The most common side effects of the drug imatinib are nausea, fluid accumulation, rash, diarrhoea, minor muscle aches, and fatigue. The drug’s serious but relatively rare side effects include liver inflammation and bleeding. The side effects caused by TKIs can often be successfully treated, managed, and relieved by your doctor.

In addition, pazopanib (Votrient) is another multitargeted TKI that is sometimes used to treat desmoid tumor growths. In a recently conducted phase II clinical trial, the tumors of patients with advanced desmoid tumors who received pazopanib as a pill by mouth exhibited more shrinkage compared to a combined chemo procedure involving drugs methotrexate and vinblastine. In 2012 the drug pazopanib received FDA approval to treat advanced soft-tissue sarcomas. The most common side effects of pazopanib are fatigue, diarrhoea, high blood pressure, and loss of colouration in the hair and skin.

Chemotherapy

Chemotherapy or chemo procedure refers to drugs or medications to eliminate cancer cells. These drugs operate by preventing the cancer cells from growing, dividing and generating more cells. But normal cells also grow and divide, and chemo procedure can affect these cells, causing a range of side effects.

A chemo regimen generally consists of several cycles given over some time. A patient may receive a single type of drug or a combination of different medications, all given simultaneously. Chemo procedure for desmoid tumors could be given as an outpatient treatment; that is, the patient may not be required to stay overnight at the hospital.

Different chemo drugs are used to treat desmoid tumors. Some types of chemotherapy that might be used alone or in combination for treating desmoid tumors include:

• Vinblastine (Velban)
• Methotrexate (Trexall)
• Doxorubicin (available as a generic drug), used alone or combined with dacarbazine.
• Liposomal doxorubicin (Doxil)
• Vinorelbine (Navelbine)
• Dacarbazine (available as a generic drug), only used in combination with doxorubicin

A Chemo procedure may be used to shrink an aggressive, organ-threatening desmoid tumour or one that is causing a range of signs or symptoms.

Chemotherapy can cause many side effects. These side effects depend on the type of drug and the dose used. Some side effects include nausea and vomiting, fatigue, hair loss, risk of infection, loss of appetite, and diarrhoea. These side effects usually go away once the treatment finishes. In some rare cases, chemotherapy can cause long-term problems that can affect the kidneys, heart and can even cause cancer.

Physical, emotional, and social effects of a desmoid tumour

Desmoid tumor and its treatment can cause many physical, emotional and social effects. Tumor care doesn’t end with active treatment. Post-treatment care is as critical as active treatment. Palliative or supportive care focus on managing and relieving symptoms and side effects associated with cancer treatment. Palliative care is carried out along with an active treatment strategy, which helps in improving the treatment efficiency by slowing the cancer growth or destroying it.

Palliative care focuses on improving a patient’s condition by helping them to manage and relieve symptoms. The care extends support not just to the patients but also to their loved ones. It also focuses on the non-medical needs of cancer patients, like managing finances. Palliative care depends on the patient’s age, stage, or tumor type. Palliative care works best when it is begun right after a cancer diagnosis. This helps prevent the cancer condition and its symptoms and side effects from aggravating. Palliative focuses on improving the comfort and quality of a patient’s life. Palliative care enhances the effectiveness of active treatment for desmoid tumor. Sometimes a patient may receive chemotherapy, surgery or radiation therapy as palliative care.  

Patients should talk with their health care team about the aim of each treatment option for desmoid tumor, about the probable side effects and the available palliative care options before the active treatment begins.

The health care team will enquire about your medical history, symptoms and side effects during treatment for desmoid tumor. Be sure to communicate whatever you are feeling to the doctor. This helps them treat any symptoms or side effects that you are experiencing with ease. This will eventually help prevent your condition from becoming worse. 

Remission and the chance of recurrence of desmoid tumour

Remission refers to a stage where there are no signs or symptoms of a tumor in the body. The condition can be called NED or “no evidence of disease” in the body. Remission can be permanent or temporary. Cancer patients in remission are constantly worried about a probable cancer recurrence. Patients must continue tests, scans, physical examinations even after successful treatment of for desmoid tumor to look for recurrence. Patients with desmoid tumors should talk with their doctor about the chance of the tumor condition recurring back in the body. If cancer recurs in the same place as before, it is called local recurrence, and if it happens nearby the origin point, it is called a regional recurrence. If it recurs in some distant place in the body, it is called distant recurrence.

The doctor will check for any signs of tumor recurrence. And if the tumor growth is back in the body, the doctor will list out the possible treatment options. In most cases, the treatment procedures for recurrent for desmoid tumor include surgery, radiation therapy etc., but the same kind of radiation procedure cannot be given more than once. These therapies may be performed in different combinations.

Many clinical trials are testing the viability of various treatment options for dealing with recurrent desmoid tumors. Patients are encouraged to talk with their doctor about any such possibilities. Whatever the treatment plan be, palliative care will always be an essential part that will primarily focus on managing and relieving symptoms and side effects of treatment.

When a tumor recurs back in the body or the disease worsens, it is quite natural and entirely ok for patients and their loved ones to feel upset and be in extreme fear. In such cases, both the patients and their caretakers are encouraged to talk to the healthcare team or counsellors or seek support services or groups to cope with the circumstances. 

What If the treatment does not work?

Desmoid tumor recovery may not be successful at times. The disease condition is advanced or terminal if it cannot be controlled or cured. Any stage of cancer or tumor can be stressful to people. A diagnosis that states an advanced, aggressive tumor can cause even more stress and make people hopeless and uncertain about their lives. Patients should have open, honest communications with their doctor or health care team about their concerns, feelings and preferences. Health care team members are skilled and will support the patients and their caregivers. They will focus on improving the comfort and quality of the patient’s life by providing them physical, mental and financial support.

People diagnosed with advanced tumors and the expected chance of survival is for less than six months can consider hospice care. Hospice care focuses on providing the best possible quality care for patients whose survival chance is low. Patients and their families can talk to the health care team about the available hospice options, including a unique hospice Centre, hospice care at home or any other health care location. People can also avail nursing care options.

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