Materiały źródłowe

• #1 Desmoid tumors: Treatment – UpToDate

  https://www.uptodate.com/contents/desmoid-tumors-treatment

  Desmoid tumors: Treatment […] Desmoid tumors can follow an unpredictable clinical course, with some tumors undergoing regression in the absence of therapy. For many patients, especially those with asymptomatic or minimally symptomatic tumors, an initial strategy of active surveillance is the preferred approach. […] However, if a desmoid tumor progresses on serial imaging or is associated with significant symptoms that are difficult to manage, a multidisciplinary team should determine the best individualized approach. […] Although surgery and radiation had traditionally been used, systemic therapy is often preferred for many patients, including those with FAP or intra-abdominal/mesenteric tumors, when an intervention is required. […] Initial surveillance — In patients with newly diagnosed desmoid tumors, we pursue active surveillance as the initial management unless patients have moderate or severe symptoms that cannot be controlled with appropriate supportive care (eg, nonsteroidal anti-inflammatory agents [NSAIDs] or acetaminophen) or further progression of the tumor would cause significant morbidity (eg, threatens a limb or vital organ).

• #1 Desmoid Tumor – NCI

  https://www.cancer.gov/pediatric-adult-rare-tumor/rare-tumors/rare-soft-tissue-tumors/desmoid-tumor

  Desmoid tumors can be hard to predict. They can shrink and go away on their own, they can remain the same size, or they can grow quickly. […] Treatment for each patient will be unique so you should go to an expert in sarcoma treatment to decide the best approach for your tumor. […] Treatment options to discuss with your doctor include: […] Surgery has been a standard treatment for desmoid tumors in the past but this may be changing. Given that the tumor often returns to the same location after surgery, doctors are looking for other treatment options. […] Radiation therapy is a treatment option for some desmoid tumors. Given that radiation therapy can cause other cancers in the future, it is important to discuss alternatives with your doctor. […] There is no standard chemotherapy for desmoid tumors but promising new drugs have been shown to shrink these tumors. […] It is important to discuss these different options with your doctor and seek second opinions, if possible.

• #1 Desmoid tumors: Treatment – UpToDate

  https://www.uptodate.com/contents/desmoid-tumors-treatment

  Active surveillance typically involves imaging studies at least every three to six months. […] Support for observation as a treatment strategy is supported in several studies. As an example, in a phase III trial comparing the use of sorafenib with placebo in patients with desmoid fibromatosis, 20 percent of patients in the placebo arm had a partial response.

• #1 Treatment – Desmoid Fundation

  https://www.desmoidfoundation.org/en/treatment/

  Therefore, considering the high number of recurrences, the morbidity related to surgery and the possibility of stability of the disease or even spontaneous regression also in absence of treatments, recently a more conservative treatment has been proposed and an initial surveillance approach has been suggested. […] This ACTIVE SURVEILLANCE program, with clinical and radiological observation only, is defined as Watch and Wait protocol. […] Active surveillance is typically the first approach for most people with a new diagnosis of DF. Its defined as active monitoring or active observation (via FOLLOW UP). This monitoring program can be slowed after 1-2 years. […] If the patient with desmoid fibromatosis is initiated into an active surveillance program, he will follow a regular monitoring program with his oncologist which includes checks every three to four months. At each appointment, the possibility of starting a treatment through a clinical examination and the study of radiological examinations (ultrasound or MRI) will be examined.

• #1 Desmoid Tumor – StatPearls – NCBI Bookshelf

  https://www.ncbi.nlm.nih.gov/books/NBK459231/

  Desmoid tumors pose a significant challenge due to the lack of a standard treatment approach. […] Surgical resection has fallen out of favor due to high rates of recurrence. […] Systemic treatment options have increased with the recent approval of gamma-secretase inhibitor nirogacestat. […] Surgical resection has taken a backseat in the management of desmoid tumor due to high recurrence rates and morbid resections. […] Radiation therapy is primarily used in the adjuvant setting when surgery leaves behind a positive margin, or surgical resection is impossible. […] Systemic therapy is usually reserved for patients who experience rapid growth of a desmoid tumor, or for those patients in whom the tumor is threatening a critical structure. […] Desmoid tumor is not a chemosensitive tumor; however, few regimens may benefit patients.

• #1 Treatment & Monitoring – The Desmoid Tumor Research Foundation

  https://dtrf.org/about-desmoid-tumors/treatment-monitoring/

  The treatment of desmoid tumors can be complex, and what works for one person may not necessarily work for another. Experts are continually working toward standardizing protocols based on tumor location, symptoms, and aggressiveness. In 2020, supported by a grant from the DTRF, an international team of experts working in the field of desmoid tumors published evidence-based, consensus guidelines for medical treatment. Known as the Global Consensus Paper on Desmoid Tumor Management, the paper answers critical questions regarding approach to treatment, types of treatments, and pain management. […] Active surveillance is the well-established primary approach to primary/recurrent sporadic/familial desmoid tumors. […] Surgery is the accepted second-line treatment only for sporadic desmoid tumors located in the abdominal wall failing observation.

• #1 Desmoid tumors – Diagnosis and treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/desmoid-tumors/diagnosis-treatment/drc-20446388

  Treatments for desmoid tumors include: […] Monitoring the growth of the tumor. If your desmoid tumor causes no signs or symptoms, your doctor may recommend monitoring the tumor to see if it grows. You may undergo imaging tests every few months. Some tumors never grow and may never require treatment. Some tumors may shrink on their own without any treatment. […] Surgery. If your desmoid tumor causes signs and symptoms, your doctor may recommend an operation to remove the entire tumor and a small margin of healthy tissue that surrounds it. But sometimes the tumor grows to involve nearby structures and can’t be completely removed. In these cases, surgeons may remove as much of the tumor as possible. […] Radiation therapy. Radiation therapy uses high-powered beams, such as X-rays and protons, to kill tumor cells. Radiation therapy may be recommended instead of surgery if you’re not healthy enough for surgery or if the tumor is located in a place that makes surgery risky. Radiation therapy is sometimes used after surgery if there’s a risk that the tumor might return.

• #1 Extra-Abdominal Desmoid Tumors – OrthoInfo – AAOS

  https://orthoinfo.aaos.org/en/diseases–conditions/extra-abdominal-desmoid-tumors

  Desmoid tumors treated with a combination of surgery and radiation or with radiation therapy alone may return. […] Drugs that block the hormonal stimulation of the tumor can be effective at controlling the tumor growth. […] Medications such as ibuprofen and naproxen, as well as a special kind of NSAID called a COX-II inhibitor, can be effective in treating desmoid tumors. […] Low doses of chemotherapy drugs are effective in management of desmoids. Long-term use of these drugs is difficult, however, due to their cumulative effects in the body. […] In some cases, surgery to remove the tumor may be recommended. However, it can be difficult during surgery to determine the outer extent of the tumor. […] Limited surgery, combined with additional treatments, such as radiation or chemotherapy, may be successful in these cases.

• #1 Radiotherapy in the treatment of aggressive fibromatosis: experience from a single institution | Radiation Oncology | Full Text

  https://ro-journal.biomedcentral.com/articles/10.1186/s13014-020-01565-9

  Desmoid-type fibromatosis is a rare, potentially locally aggressive disease. Herein we present our experience in the treatment with radiotherapy. […] The median prescribed dose was 54Gy/ Gy (RBE) [range 39.666, IQR 5060]. […] Radiotherapy in the treatment of desmoids can lead to long term control. Treatment of patients with abdominopelvine desmoids should be avoided, as the risk of higher-grade complications is substantial. […] The treatment options include surgery, radiotherapy, hormonal therapy, chemotherapy and anti-inflammatory agents. […] When active therapy is necessary, surgery is one option. Wide microscopic margin-negative resection (R0) is the goal, but conservation of cosmesis and function is the major priority. […] Preservation of function is a priority; mutilating surgery has to be avoided and radiotherapy can be beneficial in selected cases esp. in the treatment of head and neck or intrathoracic desmoids.

• #1 Radiotherapy in the treatment of aggressive fibromatosis: experience from a single institution | Radiation Oncology | Full Text

  https://ro-journal.biomedcentral.com/articles/10.1186/s13014-020-01565-9

  In cases of recurrence after previous surgery and when local control is crucial, postoperative radiotherapy can be considered. […] The latest consensus guidelines of the Desmoid Working Group suggest a moderate dose radiotherapy in those cases when active therapy is necessary but the risk of surgery-associated long-term morbidity is high. […] Our data show that radiotherapy although highly effective in the reduction of size cannot prevent and might even promote the formation of these complications. Based on this experience we conclude that RT in patients with FAP-associated desmoid tumors should be only initiated when other options have been exploited.

• #1 Desmoid Tumor Treatment | OGSIVEO®(nirogacestat) HCP Site

  https://www.ogsiveo.com/hcp/desmoid-tumor-treatment/

  Surgery is no longer recommended by guidelines as first-line treatment for most clinical situations. […] Systemic therapies are recommended as a first-line treatment option for progressive, morbid, or symptomatic desmoid tumors, according to the NCCN Guidelines and Desmoid Tumor Working Group (DTWG) Guideline. […] NCCN Guidelines also recommend ablation/embolization and definitive radiation therapy as first-line treatment options for progressive, morbid, or symptomatic desmoid tumors for certain patients.

• #1 FDA Approves Nirogacestat, the First-Ever Targeted Therapy for Desmoid Tumors | Memorial Sloan Kettering Cancer Center

  https://www.mskcc.org/news/nirogacestat-new-desmoid-tumor-treatment-improves-outcomes-people-sarcoma

  Update: On November 27, 2023, the U.S. Food and Drug Administration approved nirogacestat (Ogsiveo) for adults with desmoid tumors that cannot be treated with surgery alone. The approval was based on research led by investigators at Memorial Sloan Kettering Cancer Center. Nirogacestat is the first targeted therapy ever to be approved for desmoid tumors. […] Nirogacestat, which blocks a protein called Notch, is a new type of targeted drug called a gamma-secretase inhibitor. The paper reported that 41% of patients’ tumors significantly shrank after they took nirogacestat. […] MSK has a multidisciplinary team that specializes in treating desmoid tumors. Dr. Gounder; sarcoma surgeon Aimee Crago, MD, PhD, who also studies desmoid tumors in her lab; and interventional radiologist Joseph Erinjeri, MD, PhD, work together to determine the best, most personalized treatment for each patient. The treatments may include medications like nirogacestat and sorafenib, surgery, an interventional procedure, or a combination of these.

• #1 Nirogacestat Shrinks Desmoid Tumors – NCI

  https://www.cancer.gov/news-events/cancer-currents-blog/2023/nirogacestat-shrinks-desmoid-tumors

  Nirogacestat blocks the activity of an enzyme called gamma secretase, which is involved in driving desmoid tumor growth. […] On November 27, 2023, the Food and Drug Administration (FDA) approved nirogacestat (Ogsiveo) for adults with desmoid tumors that are growing and cannot be removed surgically. This is the first-ever FDA approval of a treatment for people with desmoid tumors. […] The approval was based on results of the DeFi trial, which showed that nirogacestat was effective in shrinking desmoid tumors. […] At 2 years after starting treatment with the investigational drug nirogacestat, three-quarters of trial participants were alive without their disease getting worse, compared with less than half of patients who were given a placebo. […] In addition, treatment with nirogacestat either partially or completely shrank tumors in about 40% of patients, whereas only 8% of patients given a placebo had tumor shrinkage.

• #1 FDA approves nirogacestat for desmoid tumors | FDA

  https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-nirogacestat-desmoid-tumors

  On November 27, 2023, the Food and Drug Administration approved nirogacestat (OGSIVEO, SpringWorks Therapeutics, Inc.) for adult patients with progressing desmoid tumors who require systemic treatment. This is the first approved treatment for desmoid tumors. […] The recommended nirogacestat dose is 150 mg administered orally twice daily with or without food until disease progression or unacceptable toxicity. Each 150 mg dose consists of three 50 mg tablets.

• #1 Side Effects of Desmoid Tumor Treatment: 6 Ways To Feel Better | MyDesmoidTumorTeam

  https://www.mydesmoidtumorteam.com/resources/side-effects-of-desmoid-tumor-treatment-6-ways-to-feel-better

  Nirogacestat is a newly approved treatment for desmoid tumor. It works by blocking gamma secretase an enzyme desmoid tumor cells use. […] TKIs block growth signals in desmoid tumor cells. They help slow desmoid tumor growth and progression. […] Here are six tips for managing your side effects from desmoid tumor treatment. Always talk to your health care team about your side effects, and never stop taking medication without medical guidance. […] If you know you get sick around the time of your desmoid tumor treatment, talk to your doctor. They can prescribe an anti-nausea drug also known as an antiemetic drug to take at a specific time. […] Some treatment options can cause vomiting and diarrhea, meaning you’re losing lots of fluid. It’s important to stay as hydrated as possible to prevent dehydration.

• #1 Desmoid Tumor – StatPearls – NCBI Bookshelf

  https://www.ncbi.nlm.nih.gov/books/NBK459231/

  Doxorubicin-based regimens have had the best success rates. […] Imatinib has been successfully used in patients with desmoid tumor. […] Nirogacestat is the first gamma-secretase inhibitor granted full USFDA approval for patients with DT whose tumors are progressing. […] Hormonal therapy is usually prescribed in combination with nonsteroidal anti-inflammatory drugs (COX-2 inhibitors). However, this approach to the treatment of desmoid tumor has fallen out of favor and is no longer recommended. […] Surgical resections are avoided due to high morbidity and high recurrence rates.

• #1 UNDERSTANDING DESMOID TUMORS: CAUSES AND TREATMENTS | Mya Care

  https://myacare.com/blog/understanding-desmoid-tumors-causes-and-treatments

  Some common medications used for desmoid tumors include non-steroidal anti-inflammatory drugs (NSAIDs), chemotherapy drugs, and hormone therapy. […] Chemotherapy drugs with a high success rate include: Sorafenib is an oral chemotherapy used as a molecular-targeted therapy. […] Ogivri (nirogacestat) is the first FDA-approved therapy for the treatment of desmoid tumors, a rare type of non-cancerous tumor. […] Researchers are constantly exploring new and innovative treatments for desmoid tumors, refining their knowledge of inhibitory mechanisms. […] One of the latest advancements in the treatment of desmoid tumors is the use of proton therapy. […] Treatment options for desmoid tumors include active monitoring, surgery, radiation therapy, and medications.

• #1 Recent Advances in Desmoid Tumor Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7463981/

  The role of surgery in treatment of DT is still under debate. […] Currently, surgery is considered a valid option for the local control of DT after failure of active surveillance. […] Radiation therapy is often considered in selected cases as a salvage therapy when other therapeutic options fail. […] Other locoregional treatments can be considered for patients with advanced DF in which surgical resection would result in significant functional impairment. […] When local treatments fail or are contraindicated, several medical strategies can be considered. […] First-line medical treatment should be represented by the least toxic options (NSAIDs and hormonal therapy), while a more aggressive approach (such as combination chemotherapy) should be reserved for patient with rapidly growing and symptomatic unresectable or advanced diseases.

• #1 Desmoid Tumor Treatment & Management: Systemic Therapies, Excision of Tumor, Long-Term Monitoring

  https://emedicine.medscape.com/article/1060887-treatment

  Systemic therapy is appropriate if a primary complete resection is not feasible or if there is relapse or progression after resection. […] The US Food and Drug Administration (FDA) approved the first drug for desmoid tumor, nirogacestat (Ogsiveo), in November 2023. Nirogacestat, a gamma secretase inhibitor, cleaves multiple transmembrane proteins, including Notch, that are believed to play a role in activating pathways that contribute to growth of desmoid tumors. It is indicated for adults with progressing tumors who require systemic treatment. […] There are a number of novel drug therapy candidates for desmoid tumors. […] Pharmacologic therapy with antiestrogens and prostaglandin inhibitors may also be used. Pharmacologic agents result in objective response rates of approximately 40-50%; the duration of response is variable.

• #1 Treatments | Stanford Health Care

  https://stanfordhealthcare.org/medical-conditions/cancer/desmoid-tumor/treatments.html

  During radiofrequency thermal ablation (RTA), we use heat to destroy tumor cells. We usually recommend RTA as an alternative to surgery. […] We may recommend intravenous (IV) or oral medications to help treat desmoid tumors. You can use certain medications along with other treatments, such as surgery and radiation therapy. Or you can take other medications alone. Medications we use include: Chemotherapy: We may use these powerful medications to treat desmoid tumors that grow quickly. […] Hormone therapy: We may use hormones to slow or stop the growth of tumor cells. For instance, tamoxifen can block your cells ability to use estrogen, which may reduce the growth of desmoid tumors. However, recent data shows that hormonal blockade may not be as effective as once thought. […] Nonsteroidal anti-inflammatory drugs (NSAIDs): NSAIDs such as aspirin and ibuprofen may help shrink the inflammation component of desmoid tumors and lessen side effects like swelling and pain. […] Targeted therapy: Targeted therapies affect genes or proteins that cause desmoid tumors to grow. We may suggest tyrosine kinase inhibitors (TKIs), such as imatinib or sorafenib, to block proteins called kinases. This treatment may prevent desmoid tumors from growing or cause them to shrink.

• #1 Evaluation of management of desmoid tumours associated with familial adenomatous polyposis in Dutch patients | British Journal of Cancer

  https://www.nature.com/articles/6605997

  The present study demonstrates that for intra-abdominal desmoid tumours, similar PFS rates were observed after surgical treatment and a more conservative approach. […] Most patients with abdominal wall and extra-abdominal desmoid tumours were treated surgically. […] Based on this and previous studies, surgery seems to be safe for extra-abdominal and abdominal wall desmoid tumours. […] Commonly used pharmacological agents are NSAIDs and hormonal agents. […] Based on personal experience from our authors, the optimal dose of tamoxifen is 40mg 4 times a day, and for toremifene 60mg 4 times a day. […] Based on these results, the authors advised high-dose tamoxifen and sulindac as the primary treatment for FAP-related desmoid tumours. […] Our findings and those of others suggest that (pegylated liposomal) doxorubicin-based chemotherapeutic regimens are effective for patients with progressive, symptomatic desmoid tumours. […] Cytotoxic chemotherapy may be effective in patients with progressively growing desmoids.

• #1 Treatments | Stanford Health Care

  https://stanfordhealthcare.org/medical-conditions/cancer/desmoid-tumor/treatments.html

  Surgery is only offered for special cases where immediate symptom relief is needed. During surgery, we will remove the entire tumor and an area of healthy tissue around it (margin). Since desmoid tumors often grow again in the same place after surgery, we will monitor you regularly and suggest further treatments if needed. […] During radiation therapy, we use high-energy X-rays to destroy the tumor. We may use radiation therapy when we’re not able to remove the entire tumor through surgery or if surgery isn’t possible. Since radiation therapy increases your cancer risk, we will discuss all risks and side effects with you before using this treatment. […] During cryoablation, we freeze tumor cells to destroy them. We prefer cryoablation instead of surgery as most desmoid tumors recur following removal. We often use MRI to help target the tumors exact location. You may need several rounds of cryoablation to destroy the tumor.

• #1 Cryotherapy in the Treatment of Extra-Abdominal Desmoid Tumors—A Review

  https://www.mdpi.com/1718-7729/32/3/137

  Cryotherapy has emerged as a promising localized treatment for desmoid tumors (DTs). […] The management of desmoid tumors (DTs) often poses challenges due to their variable clinical behavior, with treatment options including active surveillance, systemic therapy, and local therapies including surgery, ablation, and radiation. […] Cryotherapy is an interventional procedure which employs repeated cycles of freezing and thawing with the purpose of causing cell death. […] Cryoablation is utilized as either a first-line therapy or a salvage therapy in DTs following the failure of other treatment modalities, ideally within the context of a multidisciplinary tumor board review. […] Cryotherapy provides a minimally invasive option that complements existing therapies. […] Overall, cryotherapy offers a viable, minimally invasive treatment option for DTs, with favorable outcomes in both symptom relief and tumor control.

• #1 CRYOABLATION OF DESMOID TUMORS

  https://www.interventionaloncology.center/desmoid-tumors

  Classical treatment in desmoid tumors is the extensive removal of the tumor. However, after the surgical operation, up to 70% of the tumors reappear at the same site (local recurrence). […] In such cases and patients who are not suitable for surgery, radiotherapy can be performed. However, the tumor may frequently recur despite recurrent operation and radiotherapy. […] Percutaneous ablation is a common treatment modality in desmoid tumors. Especially successful results were obtained with cryoablation. […] After cryoablation, the tumor loses its viability and becomes smaller and smaller. […] Because of these advantages, cryoablation has become the treatment of choice in all desmoid tumor patients at our Varisson Radiology Centers.

• #1 Treatment & Monitoring – The Desmoid Tumor Research Foundation

  https://dtrf.org/about-desmoid-tumors/treatment-monitoring/

  Medical therapies are the second-line treatments for sporadic desmoid tumors located at all other sites and for all familial desmoid tumors failing observation. […] Local ablative treatments such as cryotherapy or radiotherapy are options that can be considered as an alternative to medical therapies on an individual basis. […] Pain control is paramount to improve quality of life, independently of the use of active treatment against the disease. […] Medical therapies are the recommended second-line treatments for all sporadic desmoid tumors except for abdominal wall tumors and all familial desmoid tumors failing observation. […] Surgery is no longer recommended as a first-line treatment for desmoid tumors due to the high rate of recurrence after surgery (25-60%) […] Scientific research has shown that some types of desmoid tumors have much higher risk of recurrence after surgery than others.

• #1 Treatment – Desmoid Fundation

  https://www.desmoidfoundation.org/en/treatment/

  Patients affected by DF are treated by surgeons and oncologists. DF is a tumor with a borderline behavior between benign and malignant, due to its characteristics of high local aggression, so the team must have the experience and skills necessary to care for patients with DF. Your treatment will be decided by a group of Doctors specialized in various branches of Medicine (multidisciplinary team) and usually you can find them in centers specialized in sarcomas treatment. […] The optimal choice of medical and/or surgical therapy to offer the patient depends on various factors which may include: the localization and size of the tumor, the presence of symptoms, the rate of growth of the tumor, its proximity to important organs such as blood vessels, the patients preference, who plays a role in the choice once informed of the therapeutic options available, the toxicity profiles of the various drugs and their administration.

• #1 Desmoid Tumor Treatment | OGSIVEO®(nirogacestat) HCP Site

  https://www.ogsiveo.com/hcp/desmoid-tumor-treatment/

  NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) recommendations for initiating treatment include symptoms, impairing or threatening in function, and tumor growth documented on imaging (e.g., MRI or CT). […] A course of ongoing observation is an appropriate option even for patients with disease progression, if the patient is minimally symptomatic and the anatomical location of the tumor is not critical. For tumors that are symptomatic, or impairing or threatening in function, patients should be offered therapy with the decision based on the location of the tumor and potential morbidity of the therapeutic option. […] The NCCN Guidelines and Desmoid Tumor Working Group (DTWG) Guideline recommend evaluation and management of patients by a multidisciplinary team with expertise and experience in desmoid tumors prior to the initiation of a treatment plan.

• #1 Balancing Symptoms and Current Therapies in Desmoid Tumor Management

  https://www.targetedonc.com/view/balancing-symptoms-and-current-therapies-in-desmoid-tumor-management

  The treatment algorithm [recommends] diagnosis by core needle biopsy, get pathology, if needed send out to a sarcoma center that has pathology to look at it because they aren’t very common, you need to make sure you’re getting the diagnosis right and next-generation sequencing if needed. The first-line approach is active surveillance for 1 to 2 years. […] I think there is probably a role for upfront treatment in cases of patients who are symptomatic or have some tumor that, if it grows, will cause some sort of problem. […] For abdominal wall [desmoid tumors], they suggest surgery. We’ve had pretty good local control rates with cryoablation. […] We tend to either do medical treatment, surveillance, or ablation-type procedures. […] I definitely have had patients who had undergone surgery, then the tumor comes back pretty frequently or pretty rapidly, and then you are stuck in the same problem, but now, if they have undergone surgery, they potentially have complications from the surgery. I think we’ve moved from the surgical frontline approach to more of an active surveillance type approach for desmoid tumors.

• #1 Recent Advances in Desmoid Tumor Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7463981/

  Given the long life expectancy of most DT patients and the cumulative toxicity of anthracyclines, low-dose chemotherapy regimen based on the association of methotrexate and a vinca alkaloid (vinorelbine or vinblastine) have also been investigated. […] Many targeted drugs have been investigated for treatment of DF, based on the presumed role of soluble factors such as PDGFs and vascular endothelial growth factor (VEGF) in DT initiation and progression. […] In the last decades, the therapy of DT has radically changed, moving from an aggressive surgical approach to a more conservative one. […] The proven efficacy of low-dose chemotherapy and hopefully and even more so TKIs and other targeted agents will further confirm the prominent role of medical therapy in the management of DT.

• #1 Current Management Strategies for Desmoid Tumors Are Marked by Less Surgery, More Systemic Options

  https://www.onclive.com/view/current-management-strategies-for-desmoid-tumors-are-marked-by-less-surgery-more-systemic-options

  Despite their rarity, the management of desmoid tumors has been an area of significant clinical change as the field has shifted away from aggressive surgical interventions to active surveillance and systemic therapies that are less morbid for patients. […] We want to turn this into a disease that is amenable with systemic therapies rather than surgery. […] The experts highlighted key challenges that remain in the management of desmoid tumors, emerging systemic options that are positioned to transform the paradigm further away from surgical interventions, and the importance of getting second opinions in this space. […] The first inclination most people have when they are told they have a desmoid tumor is to cut it out. The problem is that doing surgery on desmoid tumors can trigger recurrences in a fair number of patients, as many as up to 50% of tumors can recur after surgery. Its important not to necessarily jump into surgery and to, instead, learn about different options. Some desmoid tumors can regress on their own. Approximately 20% of desmoid tumors will simply go away by doing nothing.

• #1

  https://link.springer.com/article/10.1007/s11864-024-01177-5

  While a wait and watch strategy is suitable for asymptomatic patients, systemic therapy is appropriate for symptomatic patients, especially those with Gardeners syndrome. Upfront surgical resection and hormonal therapy with or without NSAIDs are not recommended in the current treatment landscape. TKI (like sorafenib) and GSI (like nirogacestat) have shown significant benefits in randomized phase 3 trials, inducing response and improving the symptoms of patients with DT. Several other molecules targeting the gamma-secretase and Wnt pathway are currently being developed. Amongst localized therapies, cryoablation is an attractive option in patients with extra-abdominal DT. However, careful selection of patients is needed for such procedures.

• #1 Current Management Strategies for Desmoid Tumors Are Marked by Less Surgery, More Systemic Options

  https://www.onclive.com/view/current-management-strategies-for-desmoid-tumors-are-marked-by-less-surgery-more-systemic-options

  The challenge right now in the field is determining at what point and in which cases do we decide to go with aggressive treatment and which we continue watchful waiting. We are hoping that some of the new oral or intravenous systemic therapies can make a real difference for these patients in absence of surgical management, which can sometimes be mutilating. […] If we do medical management, adverse events can be reversed and, to our knowledge, we dont see many long-term complications with most of our systemic therapies. Some systemic options can cause long-term complications, but they are much less than that of surgery. Systemic therapy is recommended based on where the tumor is located, how symptomatic the patient is, and whether the desmoid tumor is potentially organ- or life-threatening.

• #1 Approaches to Patient Management of Desmoid Tumors –

  https://www.desmoidtumors.com/hcp/management/

  For patients with progressive and/or symptomatic desmoid tumors who are not candidates for systemic therapy, or whose disease is refractory to systemic therapy, consideration of cryoablation and radiation therapy is appropriate. […] Enrollment in a clinical trial may be considered an option for appropriate patients.

• #1 Side Effects of Desmoid Tumor Treatment: 6 Ways To Feel Better | MyDesmoidTumorTeam

  https://www.mydesmoidtumorteam.com/resources/side-effects-of-desmoid-tumor-treatment-6-ways-to-feel-better

  Desmoid tumor treatments can leave you feeling under the weather. All medications, including chemotherapy and targeted therapies (which target specific parts of cancer cells to stop them from spreading), have side effects. […] Doctors use chemotherapy to treat aggressive desmoid tumors that are near organs or are causing symptoms. Chemotherapy uses toxic chemicals to kill rapidly dividing cells like desmoid tumor cells. […] Chemotherapy drugs are effective for shrinking tumors. However, they also slow your body’s rapidly dividing cells. You may experience hair loss, nausea, vomiting, and mouth sores. This is because chemotherapy affects your hair follicles and the cells lining your intestines and mouth. […] Targeted therapies work by targeting specific proteins made by desmoid tumor cells. Your doctor may prescribe one of several targeted therapies, including: Nirogacestat (Ogsiveo) and Tyrosine kinase inhibitors (TKIs), like sorafenib, imatinib, and pazopanib.

• #1 Side Effects of Desmoid Tumor Treatment: 6 Ways To Feel Better | MyDesmoidTumorTeam

  https://www.mydesmoidtumorteam.com/resources/side-effects-of-desmoid-tumor-treatment-6-ways-to-feel-better

  Your dietitian can also help you choose the best foods to stay healthy while undergoing treatment. […] Fatigue is a persistent feeling of exhaustion that doesn’t seem to get better with sleep or rest. This is a common side effect of many treatments, including chemotherapy and targeted therapy. […] Hair loss is a common side effect of chemotherapy. Although there’s no way to completely stop hair loss, there are ways to help prevent it. […] Sometimes, desmoid tumor treatments cause skin rashes that may be itchy or painful. […] Throughout your desmoid tumor treatment, keep track of your side effects. Let your doctor know how you’re doing at your follow-up appointments, and bring up any new side effects. […] If your doctor thinks your desmoid tumors are still growing with treatment, they’ll run additional tests to check. You may need to change your treatment plan to slow the tumor growth.

• #1 Can You Take Supplements During Desmoid Tumor Treatment? | MyDesmoidTumorTeam

  https://www.mydesmoidtumorteam.com/resources/can-you-take-supplements-during-desmoid-tumor-treatment

  If you are undergoing targeted therapies for desmoid tumors, such as nirogacestat (Ogsiveo) the only FDA-approved treatment option for desmoid tumors or tyrosine kinase inhibitors (TKIs), you may wonder whether supplements could interact with these treatment options. […] In addition to targeted therapies and chemotherapy, treatments for desmoid tumors may also include surgery or radiation therapy. […] However, when it comes to desmoid tumors, the use of supplements must be approached with caution. […] The American Cancer Society emphasizes that while dietary supplements can have some benefits, they may also interfere with conventional cancer treatments. […] Taking large doses of antioxidants could make certain treatments, including chemotherapy and radiation, less effective. […] Additionally, herbal supplements can interfere with prescribed medications.

• #1 Can You Take Supplements During Desmoid Tumor Treatment? | MyDesmoidTumorTeam

  https://www.mydesmoidtumorteam.com/resources/can-you-take-supplements-during-desmoid-tumor-treatment

  Always check with your oncologist before using herbal supplements, as some may interact with your treatment. […] For people with desmoid tumors, keeping enough vitamin D may support overall health, especially if treatment makes it hard to spend time outdoors. […] Omega-3 supplements could increase the risk of bleeding, particularly for people having surgery, although this is still debated. […] Taking supplements during desmoid tumor treatment comes with some risks. […] Many supplements, especially herbs, can change how your body processes targeted therapies or chemotherapy. […] Taking high doses of certain supplements can put extra stress on these organs, leading to problems. […] While many supplements can interfere with treatment, a few might be safe or even helpful. […] Always check with your health care team before starting any supplement.

• #1 Find a Physician – The Desmoid Tumor Research Foundation

  https://dtrf.org/patients-caregivers/find-a-physician/

  After being diagnosed with a desmoid tumor, it is important to seek out a doctor who is familiar with desmoid tumors, typically a sarcoma specialist. Desmoid tumors are extremely rare, and are often misdiagnosed or mistreated. Patients with desmoid tumors ideally should be evaluated at a sarcoma center by a multidisciplinary team which includes a medical oncologist, pathologist, surgical oncologist, interventional radiologist and others. […] Sarcoma specialists can typically be found at major academic medical centers and can help support you with a robust team of doctors who are more likely to be knowledgeable in desmoid tumor care. These major medical centers may have designations as a National Comprehensive Cancer Network (NCCN) center or as a Sarcoma Alliance for Research through Collaboration (SARC) center. […] The Global Consensus Paper on Desmoid Tumor Treatment recommends that patients find or be referred to an experienced multidisciplinary team at the time of diagnosis.

• #1 About Desmoid Tumors | OGSIVEO® (nirogacestat) Patient Site Up to 1650 people2 to 3 timesBetween 20 & 44 years of ageUp to 77% of desmoid tumors may recur

  https://www.ogsiveo.com/about-desmoid-tumors/

  OGSIVEO is a prescription medication used to treat adults with progressing desmoid tumors who require a medicine by mouth or injection (systemic therapy). […] Regularly monitoring your symptoms and reporting them to your healthcare providers may help them create a more personalized treatment plan. […] Your care team may include desmoid tumor specialists, such as: Medical oncologist, Surgeon, orthopedic or surgical oncologist, Radiation oncologist, Pathologist. […] A multidisciplinary care team can help you: Decide on your individualized care plan based on the advice of multiple healthcare professionals, including a desmoid tumor specialist or expert, Coordinate your care for a faster time to treatment, Use the team’s shared knowledge to help make informed treatment decisions. […] OGSIVEO can affect fertility in females and males, which may affect your ability to have a child.

• #1 Treatment – Desmoid Fundation

  https://www.desmoidfoundation.org/en/treatment/

  Perfusion (ILP) is a therapeutic option for patients with desmoid tumors of the distal extremities (forearm/hand or leg/foot). […] Cryoablation can be used to treat DF; its based on the use of very low temperatures to freeze the tumor. […] Its a new technique used mostly in US and Australia, for which there is not yet enough long-term data and its practiced only in a few highly specialized centers. […] OBSERVATION represents the first therapeutic approach to this disease. When the disease maintains its evolutionary trend, it requires the initiation of a specific treatment. […] This specific treatment depends on the anatomical localization of the disease, the speed of growth and the choice of the patient. […] In this localization there is the highest percentage of spontaneous regressions.

• #1 Treatment – Desmoid Fundation

  https://www.desmoidfoundation.org/en/treatment/

  THE TREATMENT WILL BE CHOSEN ACCORDING TO THE CHARACTERISTICS OF EACH PATIENT AND HIS DESMOID TUMOR. EACH CASE MUST BE DISCUSSED BY A MULTIDISCIPLINARY TEAM (MADE UP OF ONCOLOGISTS, SURGEONS, PATHOLOGISTS, RADIOLOGISTS AND PAIN THERAPISTS EXPERIENCED IN THE TREATMENT OF THE DESMOID FIBROMATOSIS) TO FIND THE BEST TREATMENT FOR EACH PATIENT. […] Until few years ago, surgery was considered the treatment of choice for patients with desmoid tumor and it involved the removal of the tumor with a large margin, that is associated with the removal of a large area of healthy tissue too. […] This high risk, the need for multiple surgeries in patients with recurrences that is related with functional and aesthetic outcomes as well as the observation of stability in absence of treatment and some spontaneous regressions, led to a change in the therapeutic approach, thus avoiding surgery as an initial treatment.

• #2 Recent Advances in Desmoid Tumor Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7463981/

  The desmoid tumor is a locally aggressive proliferative disease within the family of soft-tissue sarcomas. […] Recently, active surveillance has being regarded as the appropriate strategy at diagnosis, as indolent persistence or spontaneous regressions are not uncommon. Here, we review the most recent advances in desmoid tumor therapy, including low-dose chemotherapy and treatment with tyrosine kinase inhibitors. […] The therapeutic approach in DT patients requires a multidisciplinary evaluation accounting for the large variability in tumor location, extension, intrinsic aggressiveness, as well as patient symptoms and preferences. […] In the last two decades, the validity of active surveillance as a frontline approach in patients with resectable disease has also been shown. […] Currently, a conservative approach can be considered the treatment of choice in asymptomatic or minimally symptomatic patients or those with tumors involving critical sites, as the mesentery.

• #2 Desmoid tumors: Treatment – UpToDate

  https://www.uptodate.com/contents/desmoid-tumors-treatment

  Desmoid tumors: Treatment […] Desmoid tumors can follow an unpredictable clinical course, with some tumors undergoing regression in the absence of therapy. For many patients, especially those with asymptomatic or minimally symptomatic tumors, an initial strategy of active surveillance is the preferred approach. […] However, if a desmoid tumor progresses on serial imaging or is associated with significant symptoms that are difficult to manage, a multidisciplinary team should determine the best individualized approach. […] Although surgery and radiation had traditionally been used, systemic therapy is often preferred for many patients, including those with FAP or intra-abdominal/mesenteric tumors, when an intervention is required. […] Initial surveillance — In patients with newly diagnosed desmoid tumors, we pursue active surveillance as the initial management unless patients have moderate or severe symptoms that cannot be controlled with appropriate supportive care (eg, nonsteroidal anti-inflammatory agents [NSAIDs] or acetaminophen) or further progression of the tumor would cause significant morbidity (eg, threatens a limb or vital organ).

• #2 Desmoid tumors: Treatment – UpToDate

  https://www.uptodate.com/contents/desmoid-tumors-treatment

  Active surveillance typically involves imaging studies at least every three to six months. […] Support for observation as a treatment strategy is supported in several studies. As an example, in a phase III trial comparing the use of sorafenib with placebo in patients with desmoid fibromatosis, 20 percent of patients in the placebo arm had a partial response.

• #2 Treatment – Desmoid Fundation

  https://www.desmoidfoundation.org/en/treatment/

  Therefore, considering the high number of recurrences, the morbidity related to surgery and the possibility of stability of the disease or even spontaneous regression also in absence of treatments, recently a more conservative treatment has been proposed and an initial surveillance approach has been suggested. […] This ACTIVE SURVEILLANCE program, with clinical and radiological observation only, is defined as Watch and Wait protocol. […] Active surveillance is typically the first approach for most people with a new diagnosis of DF. Its defined as active monitoring or active observation (via FOLLOW UP). This monitoring program can be slowed after 1-2 years. […] If the patient with desmoid fibromatosis is initiated into an active surveillance program, he will follow a regular monitoring program with his oncologist which includes checks every three to four months. At each appointment, the possibility of starting a treatment through a clinical examination and the study of radiological examinations (ultrasound or MRI) will be examined.

• #2 Desmoid Tumor Treatment | OGSIVEO®(nirogacestat) HCP Site

  https://www.ogsiveo.com/hcp/desmoid-tumor-treatment/

  NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) recommendations for initiating treatment include symptoms, impairing or threatening in function, and tumor growth documented on imaging (e.g., MRI or CT). […] A course of ongoing observation is an appropriate option even for patients with disease progression, if the patient is minimally symptomatic and the anatomical location of the tumor is not critical. For tumors that are symptomatic, or impairing or threatening in function, patients should be offered therapy with the decision based on the location of the tumor and potential morbidity of the therapeutic option. […] The NCCN Guidelines and Desmoid Tumor Working Group (DTWG) Guideline recommend evaluation and management of patients by a multidisciplinary team with expertise and experience in desmoid tumors prior to the initiation of a treatment plan.

• #2 Treatment & Monitoring – The Desmoid Tumor Research Foundation

  https://dtrf.org/about-desmoid-tumors/treatment-monitoring/

  Medical therapies are the second-line treatments for sporadic desmoid tumors located at all other sites and for all familial desmoid tumors failing observation. […] Local ablative treatments such as cryotherapy or radiotherapy are options that can be considered as an alternative to medical therapies on an individual basis. […] Pain control is paramount to improve quality of life, independently of the use of active treatment against the disease. […] Medical therapies are the recommended second-line treatments for all sporadic desmoid tumors except for abdominal wall tumors and all familial desmoid tumors failing observation. […] Surgery is no longer recommended as a first-line treatment for desmoid tumors due to the high rate of recurrence after surgery (25-60%) […] Scientific research has shown that some types of desmoid tumors have much higher risk of recurrence after surgery than others.

• #2 Desmoid tumors: 8 things to know | MD Anderson Cancer Center

  https://www.mdanderson.org/cancerwise/desmoid-tumors–8-things-to-know.h00-159622590.html

  Desmoid tumors are slow-growing, locally invasive tumors that develop in the body’s connective tissues. Also known as aggressive fibromatosis, they’re treated with many of the same therapies that are used on soft tissue cancers like sarcoma, including surgery and chemotherapy. […] Desmoid tumors tend to wax and wane in terms of size, growing slowly for a while only to turn around and become stable or even shrink. So, we’ll start by watching and waiting first to gauge the tempo of each patient’s disease. There’s no one cookie-cutter approach. […] That said, there are certain cases in which you’d need immediate treatment. If a desmoid tumor is causing severe symptoms, for instance, or growing in a location where it could cause serious complications, such as a bowel obstruction, we’ll look at whether surgery is an option.

• #2 Desmoid tumor: Symptoms, treatment, and prognosis

  https://www.medicalnewstoday.com/articles/319365

  It is best to receive treatment for a desmoid tumor from a medical team that specializes in managing sarcomas. The team should include: medical oncologists, surgeons, radiation oncologists, genetic specialists, nurses. […] In some cases, doctors may simply monitor desmoid tumors, as they can sometimes shrink without intervention. The medical community refers to nondividing cancer cells as being in a quiescent state. […] Depending on the characteristics of the tumor, a wait and watch period is sometimes the best course of action before a person undergoes more intensive treatment. […] Surgery is a standard treatment for a desmoid tumor, but it is only an option if a surgeon can remove the tumor without damaging the surrounding organs. […] The surgeon will also remove a margin of the tissue surrounding the tumor. Desmoid tumors often invade surrounding structures, including blood vessels and organs. Removing the extra tissue reduces the risk of this occurring.

• #2 Radiotherapy in the treatment of aggressive fibromatosis: experience from a single institution | Radiation Oncology | Full Text

  https://ro-journal.biomedcentral.com/articles/10.1186/s13014-020-01565-9

  Desmoid-type fibromatosis is a rare, potentially locally aggressive disease. Herein we present our experience in the treatment with radiotherapy. […] The median prescribed dose was 54Gy/ Gy (RBE) [range 39.666, IQR 5060]. […] Radiotherapy in the treatment of desmoids can lead to long term control. Treatment of patients with abdominopelvine desmoids should be avoided, as the risk of higher-grade complications is substantial. […] The treatment options include surgery, radiotherapy, hormonal therapy, chemotherapy and anti-inflammatory agents. […] When active therapy is necessary, surgery is one option. Wide microscopic margin-negative resection (R0) is the goal, but conservation of cosmesis and function is the major priority. […] Preservation of function is a priority; mutilating surgery has to be avoided and radiotherapy can be beneficial in selected cases esp. in the treatment of head and neck or intrathoracic desmoids.

• #2 Radiotherapy in the treatment of aggressive fibromatosis: experience from a single institution | Radiation Oncology | Full Text

  https://ro-journal.biomedcentral.com/articles/10.1186/s13014-020-01565-9

  In cases of recurrence after previous surgery and when local control is crucial, postoperative radiotherapy can be considered. […] The latest consensus guidelines of the Desmoid Working Group suggest a moderate dose radiotherapy in those cases when active therapy is necessary but the risk of surgery-associated long-term morbidity is high. […] Our data show that radiotherapy although highly effective in the reduction of size cannot prevent and might even promote the formation of these complications. Based on this experience we conclude that RT in patients with FAP-associated desmoid tumors should be only initiated when other options have been exploited.

• #2 Desmoid Tumors | UCSF Radiology

  https://radiology.ucsf.edu/patient-care/services/desmoid-tumors

  More established therapies for desmoid tumors include close observation, surgical resection, radiation therapy, and systemic medical therapy. […] Surgery, even with negative margins, can have recurrence rates as high as 50%, and multiple recurrences following surgery can result in local morbidity. […] Radiation results in tumor control in approximately 75% of adults but can be associated with pain, reduced motion, pathological fractures, and secondary malignancies. […] HIFU has shown promise as a treatment option. In the largest study to date of 15 patients with desmoid tumors (25 total treatments), total tumor volume decreased 63% and pain improved from approximately 6 on a 1-10 scale to 1.3, on average.

• #2 Desmoid Tumors: Symptoms, Causes, Treatment

  https://www.webmd.com/colorectal-cancer/desmoid-tumors-facts

  If you’re diagnosed with a desmoid tumor, your doctor will recommend one of the following: […] Surgery: This is done when possible, though it can be difficult for tumors in the abdomen. As many as 25% to 50% come back to the same area after surgery. You may need other treatments as well. […] Radiation therapy: Used alone or with surgery or medicines or both, radiation is effective for many people. However, you may not be able to have it if the tumor is in your abdomen, because radiation can damage other important areas of your body. […] Radiofrequency ablation: Your doctor will put needles into the tumors and send radio waves through them to blast them with heat. This is a new approach and likely won’t be the first one your doctor recommends. […] Medication: There isn’t a standard drug treatment for desmoid tumors. But different types of medicines, including anticancer drugs, can be used to try to shrink them or stop them from growing. […] Cryoablation: A probe is used to freeze the tumor tissue.

• #2 Approaches to Patient Management of Desmoid Tumors –

  https://www.desmoidtumors.com/hcp/management/

  Guidelines recommend first-line systemic therapy for progressive or symptomatic disease […] For progressive, morbid, or symptomatic desmoid tumors, systemic therapies are recommended as a first-line treatment option according to the NCCN Guidelines and DTWG Guideline. […] There is one FDA-approved treatment option for desmoid tumors. […] NCCN Guidelines also recommend ablation/embolization and definitive radiation therapy as first-line treatment options for progressive, morbid, or symptomatic desmoid tumors for certain patients. […] According to the NCCN Guidelines: In general, surgery is not considered a first-line treatment option for desmoid tumors, except in certain situations if agreed upon by a multidisciplinary tumor board. […] According to the NCCN Guidelines, radiation therapy, cryoablation, and drug-eluting bead chemoembolization are sometimes used to help treat desmoid tumors.

• #2 FDA Approves First Therapy for Rare Type of Non-Cancerous Tumors | FDA

  https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-rare-type-non-cancerous-tumors

  Today, the U.S. Food and Drug Administration approved Ogsiveo (nirogacestat) tablets for adult patients with progressing desmoid tumors who require systemic treatment. Ogsiveo is the first drug to be approved for the treatment of patients with desmoid tumors, a rare subtype of soft tissue sarcomas. […] Although surgical removal has historically been the treatment of choice, there is a high risk that the tumor will return or that other health challenges will occur after removal; therefore, systemic therapies (cancer treatment targeting the entire body) are being increasingly evaluated in clinical trials. […] Todays approval will offer the first approved treatment option for patients beyond surgery and radiation. […] The pivotal clinical trial demonstrated that Ogsiveo provided clinically meaningful and statistically significant improvement in progression-free survival compared to placebo. Additionally, the objective response rate was also statistically different between the two arms with a response rate of 41% in the Ogsiveo arm and 8% in the placebo arm. Ogsiveo also received FDA Fast Track and Breakthrough Therapy designations for the indication noted above, as well as Orphan-Drug designation for treatment of desmoid tumor (aggressive fibromatosis).

• #2 Nirogacestat Shrinks Desmoid Tumors – NCI

  https://www.cancer.gov/news-events/cancer-currents-blog/2023/nirogacestat-shrinks-desmoid-tumors

  Nirogacestat blocks the activity of an enzyme called gamma secretase, which is involved in driving desmoid tumor growth. […] On November 27, 2023, the Food and Drug Administration (FDA) approved nirogacestat (Ogsiveo) for adults with desmoid tumors that are growing and cannot be removed surgically. This is the first-ever FDA approval of a treatment for people with desmoid tumors. […] The approval was based on results of the DeFi trial, which showed that nirogacestat was effective in shrinking desmoid tumors. […] At 2 years after starting treatment with the investigational drug nirogacestat, three-quarters of trial participants were alive without their disease getting worse, compared with less than half of patients who were given a placebo. […] In addition, treatment with nirogacestat either partially or completely shrank tumors in about 40% of patients, whereas only 8% of patients given a placebo had tumor shrinkage.

• #2 Nirogacestat Shrinks Desmoid Tumors – NCI

  https://www.cancer.gov/news-events/cancer-currents-blog/2023/nirogacestat-shrinks-desmoid-tumors

  This is one of the largest studies to date on desmoid tumors, said Mrinal M. Gounder, M.D., of Memorial Sloan Kettering Cancer Center, who led the study. The results can potentially lead to the first new drug approved in this ultra-rare disease. […] The manufacturer of nirogacestat, SpringWorks Therapeutics, has submitted an application to the Food and Drug Administration (FDA) for approval of nirogacestat for the treatment of adults with desmoid tumors. […] Nirogacestat shows effectiveness in shrinking desmoid tumors. […] After a median of 16 months, people treated with nirogacestat were 71% less likely to have died or had their disease worsen than those treated with a placebo. […] After 2 years, there was no evidence of tumors getting worse in 76% of people who received nirogacestat, compared with 44% of people who received a placebo.

• #2 Desmoid tumors: 8 things to know | MD Anderson Cancer Center

  https://www.mdanderson.org/cancerwise/desmoid-tumors–8-things-to-know.h00-159622590.html

  The next thing we’ll consider is local interventions, such as radiation therapy or microwave/radiofrequency ablation. If those are not options, then we’ll move on to systemic drug therapies. There’s a wide spectrum of choices there, including hormonal therapies like tamoxifen, targeted therapies like tyrosine kinase inhibitors, and chemotherapy. […] The odds of a good response to therapy increase the further down that path you go, but so does the chance of developing side effects. And, for a tumor that doesn’t metastasize, doesn’t transform into something more aggressive, and doesn’t have lethal implications, there’s no reason to jump right into chemo. There are lots of choices and options available, so we try to pick the one that’s best for each patient.

• #2 Desmoid Tumor Treatment & Management: Systemic Therapies, Excision of Tumor, Long-Term Monitoring

  https://emedicine.medscape.com/article/1060887-treatment

  In cases of recurrent extra-abdominal desmoid tumors in which surgery is contraindicated or in cases of recurrence, a chemotherapeutic regimen of doxorubicin, dacarbazine, and carboplatin may be effective. […] Radiation therapy may be used as a treatment for recurrent disease or as primary therapy to avoid mutilating surgical resection. It may be used postoperatively, preoperatively, or as the sole treatment. […] Aggressive, wide surgical resection is the treatment of choice. […] Complete surgical excision of desmoid tumors is the most effective method of cure. […] For tumors that are asymptomatic or nonprogressive, some prefer a wait-and-see approach. […] After surgery, MRI may be useful for monitoring desmoid tumor recurrence.

• #2 Evaluation of management of desmoid tumours associated with familial adenomatous polyposis in Dutch patients | British Journal of Cancer

  https://www.nature.com/articles/6605997

  The present study demonstrates that for intra-abdominal desmoid tumours, similar PFS rates were observed after surgical treatment and a more conservative approach. […] Most patients with abdominal wall and extra-abdominal desmoid tumours were treated surgically. […] Based on this and previous studies, surgery seems to be safe for extra-abdominal and abdominal wall desmoid tumours. […] Commonly used pharmacological agents are NSAIDs and hormonal agents. […] Based on personal experience from our authors, the optimal dose of tamoxifen is 40mg 4 times a day, and for toremifene 60mg 4 times a day. […] Based on these results, the authors advised high-dose tamoxifen and sulindac as the primary treatment for FAP-related desmoid tumours. […] Our findings and those of others suggest that (pegylated liposomal) doxorubicin-based chemotherapeutic regimens are effective for patients with progressive, symptomatic desmoid tumours. […] Cytotoxic chemotherapy may be effective in patients with progressively growing desmoids.

• #2 Treatment – Desmoid Fundation

  https://www.desmoidfoundation.org/en/treatment/

  Non-steroidal anti-inflammatory drugs (NSAID) can be used to treat DF. These drugs can have an impact on tumor growth and are also useful for treating the inflammation and pain associated with the tumor. […] Desmoid tumors are often sensitive to hormone therapy, particularly hormone therapy with anti-estrogenic activity. […] Chemotherapy is a treatment that involves the use of drugs to destroy cancer cells. […] The most frequently used chemotherapy drugs are Methotrexate in combination with Vinorelbine/Vinblastine, with low dosage, for long periods. […] In DF several drugs with anti-tyrosine kinase activity have been shown to be active: IMATINIB was the first drug of this category to have demonstrated efficacy in patients affected by DF. […] Radiotherapy uses high-energy beams of radiation to destroy cancer cells. This treatment can be used after surgery or on its own; it is intended to improve symptoms and to attempt to slow tumor growth.

• #2

  https://link.springer.com/article/10.1007/s11864-024-01177-5

  There is no standard treatment approach to these unpredictable tumors that can undergo spontaneous regression in up to 20% of patients. Surgical resection as the first line of treatment has fallen out of favor due to significant morbidity and high recurrence rates. Medical therapy includes anthracycline-based chemotherapy, vinca-alkaloid-based chemotherapy, and targeted therapies, including tyrosine kinase inhibitors. The combination of non-steroidal anti-inflammatory drugs and hormone therapy is discouraged due to the lack of a clear benefit in prospective studies. Newer drugs like gamma-secretase inhibitors have shown promise and are currently pending approval from the USFDA. Cryoablation is a relatively newer technique that is currently under investigation for the treatment of patients with DT. Several new molecules are currently being developed to target the Wnt pathway.

• #2 Cryotherapy in the Treatment of Extra-Abdominal Desmoid Tumors—A Review

  https://www.mdpi.com/1718-7729/32/3/137

  Cryotherapy has emerged as a promising localized treatment for desmoid tumors (DTs). […] The management of desmoid tumors (DTs) often poses challenges due to their variable clinical behavior, with treatment options including active surveillance, systemic therapy, and local therapies including surgery, ablation, and radiation. […] Cryotherapy is an interventional procedure which employs repeated cycles of freezing and thawing with the purpose of causing cell death. […] Cryoablation is utilized as either a first-line therapy or a salvage therapy in DTs following the failure of other treatment modalities, ideally within the context of a multidisciplinary tumor board review. […] Cryotherapy provides a minimally invasive option that complements existing therapies. […] Overall, cryotherapy offers a viable, minimally invasive treatment option for DTs, with favorable outcomes in both symptom relief and tumor control.

• #2 CRYOABLATION OF DESMOID TUMORS

  https://www.interventionaloncology.center/desmoid-tumors

  Classical treatment in desmoid tumors is the extensive removal of the tumor. However, after the surgical operation, up to 70% of the tumors reappear at the same site (local recurrence). […] In such cases and patients who are not suitable for surgery, radiotherapy can be performed. However, the tumor may frequently recur despite recurrent operation and radiotherapy. […] Percutaneous ablation is a common treatment modality in desmoid tumors. Especially successful results were obtained with cryoablation. […] After cryoablation, the tumor loses its viability and becomes smaller and smaller. […] Because of these advantages, cryoablation has become the treatment of choice in all desmoid tumor patients at our Varisson Radiology Centers.

• #2 Treatments | Stanford Health Care

  https://stanfordhealthcare.org/medical-conditions/cancer/desmoid-tumor/treatments.html

  During radiofrequency thermal ablation (RTA), we use heat to destroy tumor cells. We usually recommend RTA as an alternative to surgery. […] We may recommend intravenous (IV) or oral medications to help treat desmoid tumors. You can use certain medications along with other treatments, such as surgery and radiation therapy. Or you can take other medications alone. Medications we use include: Chemotherapy: We may use these powerful medications to treat desmoid tumors that grow quickly. […] Hormone therapy: We may use hormones to slow or stop the growth of tumor cells. For instance, tamoxifen can block your cells ability to use estrogen, which may reduce the growth of desmoid tumors. However, recent data shows that hormonal blockade may not be as effective as once thought. […] Nonsteroidal anti-inflammatory drugs (NSAIDs): NSAIDs such as aspirin and ibuprofen may help shrink the inflammation component of desmoid tumors and lessen side effects like swelling and pain. […] Targeted therapy: Targeted therapies affect genes or proteins that cause desmoid tumors to grow. We may suggest tyrosine kinase inhibitors (TKIs), such as imatinib or sorafenib, to block proteins called kinases. This treatment may prevent desmoid tumors from growing or cause them to shrink.

• #2 Approaches to Patient Management of Desmoid Tumors –

  https://www.desmoidtumors.com/hcp/management/

  For patients with progressive and/or symptomatic desmoid tumors who are not candidates for systemic therapy, or whose disease is refractory to systemic therapy, consideration of cryoablation and radiation therapy is appropriate. […] Enrollment in a clinical trial may be considered an option for appropriate patients.

• #2 Treatments | Stanford Health Care

  https://stanfordhealthcare.org/medical-conditions/cancer/desmoid-tumor/treatments.html

  Ease of access to your care team through our multidisciplinary clinics, where you can see a number of specialists, often during the same visit. […] You may not need treatment if you have a desmoid tumor with no symptoms, but we will monitor it over time. This process is called active surveillance, or watchful waiting. Some desmoid tumors never grow, and they may even shrink on their own. But if you develop symptoms that affect your quality of life or organ function, treatment may benefit you. […] Treatment for a desmoid tumor depends on several factors, including: How quickly the tumor is growing, Location of the tumor, Symptoms caused by the tumor, Your age, other health conditions, and overall health. […] Our doctors will suggest the most appropriate treatment for your situation. Treatments we offer include:

• #2 Find a Physician – The Desmoid Tumor Research Foundation

  https://dtrf.org/patients-caregivers/find-a-physician/

  After being diagnosed with a desmoid tumor, it is important to seek out a doctor who is familiar with desmoid tumors, typically a sarcoma specialist. Desmoid tumors are extremely rare, and are often misdiagnosed or mistreated. Patients with desmoid tumors ideally should be evaluated at a sarcoma center by a multidisciplinary team which includes a medical oncologist, pathologist, surgical oncologist, interventional radiologist and others. […] Sarcoma specialists can typically be found at major academic medical centers and can help support you with a robust team of doctors who are more likely to be knowledgeable in desmoid tumor care. These major medical centers may have designations as a National Comprehensive Cancer Network (NCCN) center or as a Sarcoma Alliance for Research through Collaboration (SARC) center. […] The Global Consensus Paper on Desmoid Tumor Treatment recommends that patients find or be referred to an experienced multidisciplinary team at the time of diagnosis.

• #2 Balancing Symptoms and Current Therapies in Desmoid Tumor Management

  https://www.targetedonc.com/view/balancing-symptoms-and-current-therapies-in-desmoid-tumor-management

  The most recent version [of NCCN for desmoid tumors is from] 2024. Anatomic location where progression would not be morbid so if it grows, it’s not going to cause an immediate problem we recommend observation with imaging with either CT or MRI, and if it’s stable or regressed, you continue observation. […] For patients who need active therapy for progressive, morbid, or symptomatic disease, if it’s intraabdominal or retroperitoneal, options include systemic therapy and surgery if resectable. Options for all other sites include…systemic therapy, ablation/embolization definitive radiation that has been used before in desmoid tumors, and we use it for select patients.

• #2

  https://link.springer.com/article/10.1007/s11864-024-01177-5

  While a wait and watch strategy is suitable for asymptomatic patients, systemic therapy is appropriate for symptomatic patients, especially those with Gardeners syndrome. Upfront surgical resection and hormonal therapy with or without NSAIDs are not recommended in the current treatment landscape. TKI (like sorafenib) and GSI (like nirogacestat) have shown significant benefits in randomized phase 3 trials, inducing response and improving the symptoms of patients with DT. Several other molecules targeting the gamma-secretase and Wnt pathway are currently being developed. Amongst localized therapies, cryoablation is an attractive option in patients with extra-abdominal DT. However, careful selection of patients is needed for such procedures.

• #2 Current Management Strategies for Desmoid Tumors Are Marked by Less Surgery, More Systemic Options

  https://www.onclive.com/view/current-management-strategies-for-desmoid-tumors-are-marked-by-less-surgery-more-systemic-options

  The challenge right now in the field is determining at what point and in which cases do we decide to go with aggressive treatment and which we continue watchful waiting. We are hoping that some of the new oral or intravenous systemic therapies can make a real difference for these patients in absence of surgical management, which can sometimes be mutilating. […] If we do medical management, adverse events can be reversed and, to our knowledge, we dont see many long-term complications with most of our systemic therapies. Some systemic options can cause long-term complications, but they are much less than that of surgery. Systemic therapy is recommended based on where the tumor is located, how symptomatic the patient is, and whether the desmoid tumor is potentially organ- or life-threatening.

• #2 UCSF Desmoid Tumor Clinical Trials for 2025 — San Francisco Bay Area

  https://clinicaltrials.ucsf.edu/desmoid-tumor

  Desmoid tumors are rare, non-cancerous growths found in connective tissues. […] UCSF is researching the effects of a drug called tegavivint on these tumors and other solid tumors. […] Tegavivint for the Treatment of Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors […] This phase I/II trial evaluates the highest safe dose, side effects, and possible benefits of tegavivint in treating patients with solid tumors that has come back (recurrent) or does not respond to treatment (refractory). Tegavivint interferes with the binding of beta-catenin to TBL1, which may help stop the growth of tumor cells by blocking the signals passed from one molecule to another inside a cell that tell a cell to grow. […] New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery

• #2 Side Effects of Desmoid Tumor Treatment: 6 Ways To Feel Better | MyDesmoidTumorTeam

  https://www.mydesmoidtumorteam.com/resources/side-effects-of-desmoid-tumor-treatment-6-ways-to-feel-better

  Nirogacestat is a newly approved treatment for desmoid tumor. It works by blocking gamma secretase an enzyme desmoid tumor cells use. […] TKIs block growth signals in desmoid tumor cells. They help slow desmoid tumor growth and progression. […] Here are six tips for managing your side effects from desmoid tumor treatment. Always talk to your health care team about your side effects, and never stop taking medication without medical guidance. […] If you know you get sick around the time of your desmoid tumor treatment, talk to your doctor. They can prescribe an anti-nausea drug also known as an antiemetic drug to take at a specific time. […] Some treatment options can cause vomiting and diarrhea, meaning you’re losing lots of fluid. It’s important to stay as hydrated as possible to prevent dehydration.

• #2 Side Effects of Desmoid Tumor Treatment: 6 Ways To Feel Better | MyDesmoidTumorTeam

  https://www.mydesmoidtumorteam.com/resources/side-effects-of-desmoid-tumor-treatment-6-ways-to-feel-better

  Your dietitian can also help you choose the best foods to stay healthy while undergoing treatment. […] Fatigue is a persistent feeling of exhaustion that doesn’t seem to get better with sleep or rest. This is a common side effect of many treatments, including chemotherapy and targeted therapy. […] Hair loss is a common side effect of chemotherapy. Although there’s no way to completely stop hair loss, there are ways to help prevent it. […] Sometimes, desmoid tumor treatments cause skin rashes that may be itchy or painful. […] Throughout your desmoid tumor treatment, keep track of your side effects. Let your doctor know how you’re doing at your follow-up appointments, and bring up any new side effects. […] If your doctor thinks your desmoid tumors are still growing with treatment, they’ll run additional tests to check. You may need to change your treatment plan to slow the tumor growth.

• #2 Can You Take Supplements During Desmoid Tumor Treatment? | MyDesmoidTumorTeam

  https://www.mydesmoidtumorteam.com/resources/can-you-take-supplements-during-desmoid-tumor-treatment

  Always check with your oncologist before using herbal supplements, as some may interact with your treatment. […] For people with desmoid tumors, keeping enough vitamin D may support overall health, especially if treatment makes it hard to spend time outdoors. […] Omega-3 supplements could increase the risk of bleeding, particularly for people having surgery, although this is still debated. […] Taking supplements during desmoid tumor treatment comes with some risks. […] Many supplements, especially herbs, can change how your body processes targeted therapies or chemotherapy. […] Taking high doses of certain supplements can put extra stress on these organs, leading to problems. […] While many supplements can interfere with treatment, a few might be safe or even helpful. […] Always check with your health care team before starting any supplement.

• #3

  https://link.springer.com/article/10.1007/s11864-024-01177-5

  Desmoid tumors are rare tumors with a tendency to infiltrate locally. The lack of a standard treatment approach makes choosing the most appropriate treatment for patients challenging. Most experts recommend watchful observation for asymptomatic patients as spontaneous regression of tumor is observed in up to 20% of patients. Upfront resection of the desmoid tumor has fallen out of favor due to high morbidity and high relapse rates associated with the tumor. Systemic therapy has evolved over several decades. Where chemotherapy, hormonal therapy, and non-steroidal anti-inflammatory drugs were used over the last several decades, tyrosine kinase inhibitors came to the forefront within the last decade. Most recently, gamma-secretase inhibitors have shown significant clinical benefit in patients with desmoid tumors, bringing forth an entirely new mechanistic approach. Several Wnt pathway inhibitors are also under development. Invasive approaches like cryoablation have also shown clinical benefit in patients with extra-abdominal desmoid tumors in recent years. The recent approval of nirogacestat has ushered in a new era of treatment for patients diagnosed with desmoid tumors. Several new molecules are expected to be approved over the coming years.

• #3 Treatment – Desmoid Fundation

  https://www.desmoidfoundation.org/en/treatment/

  Therefore, considering the high number of recurrences, the morbidity related to surgery and the possibility of stability of the disease or even spontaneous regression also in absence of treatments, recently a more conservative treatment has been proposed and an initial surveillance approach has been suggested. […] This ACTIVE SURVEILLANCE program, with clinical and radiological observation only, is defined as Watch and Wait protocol. […] Active surveillance is typically the first approach for most people with a new diagnosis of DF. Its defined as active monitoring or active observation (via FOLLOW UP). This monitoring program can be slowed after 1-2 years. […] If the patient with desmoid fibromatosis is initiated into an active surveillance program, he will follow a regular monitoring program with his oncologist which includes checks every three to four months. At each appointment, the possibility of starting a treatment through a clinical examination and the study of radiological examinations (ultrasound or MRI) will be examined.

• #3 Balancing Symptoms and Current Therapies in Desmoid Tumor Management

  https://www.targetedonc.com/view/balancing-symptoms-and-current-therapies-in-desmoid-tumor-management

  The most recent version [of NCCN for desmoid tumors is from] 2024. Anatomic location where progression would not be morbid so if it grows, it’s not going to cause an immediate problem we recommend observation with imaging with either CT or MRI, and if it’s stable or regressed, you continue observation. […] For patients who need active therapy for progressive, morbid, or symptomatic disease, if it’s intraabdominal or retroperitoneal, options include systemic therapy and surgery if resectable. Options for all other sites include…systemic therapy, ablation/embolization definitive radiation that has been used before in desmoid tumors, and we use it for select patients.

• #3 Desmoid Tumor Treatment & Management: Systemic Therapies, Excision of Tumor, Long-Term Monitoring

  https://emedicine.medscape.com/article/1060887-treatment

  In cases of recurrent extra-abdominal desmoid tumors in which surgery is contraindicated or in cases of recurrence, a chemotherapeutic regimen of doxorubicin, dacarbazine, and carboplatin may be effective. […] Radiation therapy may be used as a treatment for recurrent disease or as primary therapy to avoid mutilating surgical resection. It may be used postoperatively, preoperatively, or as the sole treatment. […] Aggressive, wide surgical resection is the treatment of choice. […] Complete surgical excision of desmoid tumors is the most effective method of cure. […] For tumors that are asymptomatic or nonprogressive, some prefer a wait-and-see approach. […] After surgery, MRI may be useful for monitoring desmoid tumor recurrence.

• #3 Desmoid Tumors | UCSF Radiology

  https://radiology.ucsf.edu/patient-care/services/desmoid-tumors

  More established therapies for desmoid tumors include close observation, surgical resection, radiation therapy, and systemic medical therapy. […] Surgery, even with negative margins, can have recurrence rates as high as 50%, and multiple recurrences following surgery can result in local morbidity. […] Radiation results in tumor control in approximately 75% of adults but can be associated with pain, reduced motion, pathological fractures, and secondary malignancies. […] HIFU has shown promise as a treatment option. In the largest study to date of 15 patients with desmoid tumors (25 total treatments), total tumor volume decreased 63% and pain improved from approximately 6 on a 1-10 scale to 1.3, on average.

• #3 FDA approves nirogacestat for desmoid tumors | FDA

  https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-nirogacestat-desmoid-tumors

  On November 27, 2023, the Food and Drug Administration approved nirogacestat (OGSIVEO, SpringWorks Therapeutics, Inc.) for adult patients with progressing desmoid tumors who require systemic treatment. This is the first approved treatment for desmoid tumors. […] The recommended nirogacestat dose is 150 mg administered orally twice daily with or without food until disease progression or unacceptable toxicity. Each 150 mg dose consists of three 50 mg tablets.

• #3 Nirogacestat Shrinks Desmoid Tumors – NCI

  https://www.cancer.gov/news-events/cancer-currents-blog/2023/nirogacestat-shrinks-desmoid-tumors

  In addition, 41% of people treated with nirogacestat had tumor shrinkage, compared with 8% of people treated with a placebo. […] People treated with nirogacestat also reported reduced pain, improved physical functioning, and improved health-related quality of life. […] Dr. Gounder pointed out that, even if nirogacestat is approved and becomes the standard of care for people with desmoid tumors, the drug may not be appropriate for every patient. […] For some whose tumors are stable and not causing pain or other problems, observation may be warranted, he said. […] And other potential treatment options for desmoid tumors that are already in clinical trials may be on the horizon.

• #3 Treatments | Stanford Health Care

  https://stanfordhealthcare.org/medical-conditions/cancer/desmoid-tumor/treatments.html

  During radiofrequency thermal ablation (RTA), we use heat to destroy tumor cells. We usually recommend RTA as an alternative to surgery. […] We may recommend intravenous (IV) or oral medications to help treat desmoid tumors. You can use certain medications along with other treatments, such as surgery and radiation therapy. Or you can take other medications alone. Medications we use include: Chemotherapy: We may use these powerful medications to treat desmoid tumors that grow quickly. […] Hormone therapy: We may use hormones to slow or stop the growth of tumor cells. For instance, tamoxifen can block your cells ability to use estrogen, which may reduce the growth of desmoid tumors. However, recent data shows that hormonal blockade may not be as effective as once thought. […] Nonsteroidal anti-inflammatory drugs (NSAIDs): NSAIDs such as aspirin and ibuprofen may help shrink the inflammation component of desmoid tumors and lessen side effects like swelling and pain. […] Targeted therapy: Targeted therapies affect genes or proteins that cause desmoid tumors to grow. We may suggest tyrosine kinase inhibitors (TKIs), such as imatinib or sorafenib, to block proteins called kinases. This treatment may prevent desmoid tumors from growing or cause them to shrink.

• #3 Recent Advances in Desmoid Tumor Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7463981/

  Given the long life expectancy of most DT patients and the cumulative toxicity of anthracyclines, low-dose chemotherapy regimen based on the association of methotrexate and a vinca alkaloid (vinorelbine or vinblastine) have also been investigated. […] Many targeted drugs have been investigated for treatment of DF, based on the presumed role of soluble factors such as PDGFs and vascular endothelial growth factor (VEGF) in DT initiation and progression. […] In the last decades, the therapy of DT has radically changed, moving from an aggressive surgical approach to a more conservative one. […] The proven efficacy of low-dose chemotherapy and hopefully and even more so TKIs and other targeted agents will further confirm the prominent role of medical therapy in the management of DT.

• #3 Desmoid Tumors: Symptoms, Causes, Treatment

  https://www.webmd.com/colorectal-cancer/desmoid-tumors-facts

  If you’re diagnosed with a desmoid tumor, your doctor will recommend one of the following: […] Surgery: This is done when possible, though it can be difficult for tumors in the abdomen. As many as 25% to 50% come back to the same area after surgery. You may need other treatments as well. […] Radiation therapy: Used alone or with surgery or medicines or both, radiation is effective for many people. However, you may not be able to have it if the tumor is in your abdomen, because radiation can damage other important areas of your body. […] Radiofrequency ablation: Your doctor will put needles into the tumors and send radio waves through them to blast them with heat. This is a new approach and likely won’t be the first one your doctor recommends. […] Medication: There isn’t a standard drug treatment for desmoid tumors. But different types of medicines, including anticancer drugs, can be used to try to shrink them or stop them from growing. […] Cryoablation: A probe is used to freeze the tumor tissue.

• #3 Current Management Strategies for Desmoid Tumors Are Marked by Less Surgery, More Systemic Options

  https://www.onclive.com/view/current-management-strategies-for-desmoid-tumors-are-marked-by-less-surgery-more-systemic-options

  Historically, surgery plus or minus radiation therapy was the best treatment modality for patients with desmoid tumors. Now, surgery plays an important but less broad role in the management of desmoid tumors. Often, someone will come in with a solitary desmoid tumor. The first-line option is to watch it if it isnt bothering them to look for progression. If it looks like it is progressing, there are a variety of noncytotoxic and cytotoxic medical therapies that can be offered. […] Sorafenib was studied in a phase 3 trial for patients with desmoid tumors, which was amazing because this is a rare disease, but we were able to do a phase 3 study. That study was a slam dunk. Clearly, this is an active drug. […] We have some new clinical trials with gamma-secretase inhibitors. We have DeFi, the phase 3 clinical trial comparing nirogacestat with placebo.

• #3 About Desmoid Tumors | OGSIVEO® (nirogacestat) Patient Site Up to 1650 people2 to 3 timesBetween 20 & 44 years of ageUp to 77% of desmoid tumors may recur

  https://www.ogsiveo.com/about-desmoid-tumors/

  OGSIVEO is a prescription medication used to treat adults with progressing desmoid tumors who require a medicine by mouth or injection (systemic therapy). […] Regularly monitoring your symptoms and reporting them to your healthcare providers may help them create a more personalized treatment plan. […] Your care team may include desmoid tumor specialists, such as: Medical oncologist, Surgeon, orthopedic or surgical oncologist, Radiation oncologist, Pathologist. […] A multidisciplinary care team can help you: Decide on your individualized care plan based on the advice of multiple healthcare professionals, including a desmoid tumor specialist or expert, Coordinate your care for a faster time to treatment, Use the team’s shared knowledge to help make informed treatment decisions. […] OGSIVEO can affect fertility in females and males, which may affect your ability to have a child.

• #3

  https://link.springer.com/article/10.1007/s11864-024-01177-5

  While a wait and watch strategy is suitable for asymptomatic patients, systemic therapy is appropriate for symptomatic patients, especially those with Gardeners syndrome. Upfront surgical resection and hormonal therapy with or without NSAIDs are not recommended in the current treatment landscape. TKI (like sorafenib) and GSI (like nirogacestat) have shown significant benefits in randomized phase 3 trials, inducing response and improving the symptoms of patients with DT. Several other molecules targeting the gamma-secretase and Wnt pathway are currently being developed. Amongst localized therapies, cryoablation is an attractive option in patients with extra-abdominal DT. However, careful selection of patients is needed for such procedures.

• #4 Recent Advances in Desmoid Tumor Therapy

  https://pmc.ncbi.nlm.nih.gov/articles/PMC7463981/

  The role of surgery in treatment of DT is still under debate. […] Currently, surgery is considered a valid option for the local control of DT after failure of active surveillance. […] Radiation therapy is often considered in selected cases as a salvage therapy when other therapeutic options fail. […] Other locoregional treatments can be considered for patients with advanced DF in which surgical resection would result in significant functional impairment. […] When local treatments fail or are contraindicated, several medical strategies can be considered. […] First-line medical treatment should be represented by the least toxic options (NSAIDs and hormonal therapy), while a more aggressive approach (such as combination chemotherapy) should be reserved for patient with rapidly growing and symptomatic unresectable or advanced diseases.

• #4 Current Management Strategies for Desmoid Tumors Are Marked by Less Surgery, More Systemic Options

  https://www.onclive.com/view/current-management-strategies-for-desmoid-tumors-are-marked-by-less-surgery-more-systemic-options

  Our hope is that newer drugs will prove to be just as effective in that group of patients, but that is an area where we still have big challenges to help our patients live better lives and control the disease. […] We have a lot more tools for desmoid tumors than we used to. Some of these new systemic therapies have demonstrated efficacy or are currently in clinical trials, which will give us new tools. We now know also that watchful waiting can be an effective way to follow and manage these patients.

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