Management

Overview

Currently there are no approved therapies available in Europe for the treatment of desmoid tumours. There is a high unmet need for treatments that specifically target desmoid tumours and can improve quality of life.1

It is important that patients be evaluated by a multidisciplinary team with expertise and experience in sarcoma to optimise management.1

Recommendations on desmoid tumour management and treatment

Active surveillance
A “wait and see” period and continuous monitoring is recommended, with a first magnetic resonance imaging (MRI) (or computed tomography [CT] if MRI is not possible) within 1 to 2 months and repeated at 3 to 6 months intervals.1,2
Active treatment

The choice of active treatment may depend on tumour location, symptoms, and risk of morbidity.1
Systemic therapy
  • Currently there are no approved therapies available in Europe for the treatment of desmoid tumours. However, systemic therapy is used for patients with rapidly growing and symptomatic unresectable tumours or advanced disease, including low-dose chemotherapy, conventional chemotherapy, tyrosine kinase inhibitors, and γ secretase inhibitor1
Surgery
  • Generally, no longer the preferred primary treatment3
  • Surgery is reasonable in situations where the expected functional outcome is favorable2
Radiotherapy and locoregional therapy
  • Radiotherapy is administered alone or after surgery1
  • Locoregional therapy includes radiofrequency and cryoablation1

Disease progression identification

Identifying desmoid tumour progression can lead to timely initiation of appropriate treatment

  • To help improve patient outcomes, healthcare professionals should assess for progression as early as possible with at least one of the following:
Tumour growth documented by MRI or CT2,4
OR
Worsening of symptoms2,4
OR
Worsening impact on daily living (e.g., functional impairment)2,4
  • Pain can be a prognostic indicator of progression5 and can be associated with worse outcomes6
  • Risk of progression may be higher for larger tumours2,7

Treatment goals should address both clinical markers and patient-relevant endpoints1

Treatment goals should not solely focus on clinical markers, such as progression-free survival. They should also consider patient-relevant endpoints, such as a reduction in desmoid tumour-specific symptom burden (e.g., pain) and its impact on patients’ lives, improvement in functioning with daily activities, and overall quality of life.1

Active surveillance (also known as “watch and wait”)

  • Recommended by both the Desmoid Tumour Working Group (DTWG*) and National Comprehensive Cancer Network® (NCCN®) Guidelines as the preferred approach for managing tumours that are asymptomatic or mildly symptomatic and not progressing or morbid1
  • If there is ongoing progression of the tumour, an increase of symptoms, or higher risk of disease morbidity, the recommended treatment options depend on the anatomic location of the tumour and its potential morbidity1

*The DTWG includes more than 50 sarcoma experts from different disciplines, patients, and patient advocates from Europe, North America, and Japan.7

“Worsening symptoms may be a sign of disease progression, even in the absence of radiographic changes. I try to be vigilant in preserving quality of life for patients affected by desmoid tumors."
Dr. Riedel
MD Medical Oncologist

Active treatment

Knowing the biology and the unpredictable course of the disease, active treatments are to be considered only in case of persistent progression2

A decision towards an active treatment should be postponed until the occurrence of subsequent progression or increase of symptom burden, assessed with at least 2 further assessments and possibly not before one year from diagnosis in the absence of fulfilling Response Evaluation Criteria in Solid Tumors (RECIST) progressive disease. This policy clearly avoids any kind of overtreatment in patients with tumours that could spontaneously regress and discourages treatment for stable and pauci-symptomatic patients.2

Patients with recurrent desmoid tumour should be considered for active treatment in cases of symptoms or continued progression or if located at anatomic sites where further progression could cause significant morbidity or could be life-threatening.2

It is imperative to carefully select the management strategy to optimize tumour control and enhance quality of life. This depends on the clinical scenario and should be discussed in a multidisciplinary team.3
Abdominal wall
First-line treatment includes surgery, local ablative techniques, or medical therapy3
Intra-abdominal / retroperitoneal / pelvic
  • In sporadic mesenteric desmoid tumour cases with complications, such as bleeding, obstruction, or perforation, surgery plays a critical role; however, the goal of surgery in this scenario is not to pursue or achieve an R0 resection because this may be associated with considerable and unnecessary morbidity3
  • For non mesenteric desmoid tumour, in cases of progression on medical treatment or active surveillance, surgery may be discussed in the multidisciplinary team when morbidity of resection is limited (e.g., abdominal wall) or when further progression or residual disease after therapy completion would result in bowel complications in cases of sporadic mesenteric desmoid tumours3
Extremity / girdles / chest wall
  • The first-line treatment should be local ablative tecniques (not including surgery) or medical therapy, depending on the clinical scenario3
  • Surgery, radiotherapy and isolated limb perfusion (ILP) may be part of the further treatment strategy3
Head and neck / intrathoracic
  • Medical therapy is generally considered the first line option3
  • Secondary treatment options include surgery, radiotherapy, local ablative techniques3

NCCN Guidelines also recommend ablation/embolisation and definitive radiation therapy as first line treatment options for progressive, morbid, or symptomatic desmoid tumours for certain patients.4

Review the DTWG consensus-based guidelines on the management of desmoid tumours
Systemic Therapy

Currently there are no approved therapies available in Europe for the treatment of desmoid tumours. However, systemic therapy is used for patients with rapidly growing and symptomatic unresectable tumours or advanced disease, including:1

  • Low-dose chemotherapy
  • Conventional chemotherapy
  • Tyrosine kinase inhibitors (sorafenib, imatinib, pazopanib, sunitinib)
  • γ secretase inhibitor

Antihormonal therapies, such as tamoxifen and toremifene, have also been used as treatments for desmoid tumours but are no longer recommended by the DTWG or NCCN Guidelines.1

Surgery

Surgery is generally no longer the standard primary treatment modality.3

There has been a paradigm shift toward active surveillance as the current recommended primary treatment of asymptomatic desmoid tumours.3

Nonetheless, there are a few scenarios in which incorporation of surgical resection into the multidisciplinary management of desmoid tumours can be considered.3

Recurrence is common after surgery for desmoid tumours1

There is a high risk of local recurrence after surgery. Recurrence rates can be exacerbated by trauma, such as trauma from surgery, and range from approximately 25% to 60% at 5 years.1

  • The invasion of major vessels and nerves and quality of surgical margins are key indicators of the high postoperative recurrence rate8
  • Unfortunately, even when surgical margins are clear of tumour, recurrence rates are high9
Learn more about the risk of recurrence
Gross tumour specimen post-resection showing invasive tendrils9
Image adapted with permission from Foo W, Lazar AJ. Pathology of desmoid tumors. In: Litchman C, ed. Desmoid Tumors. Springer; 2012. Accessed April 9, 2024. https://doi.org/10.1007/978-94-007-1685-8_3. Arrows added.
Gross tumour specimen post-resection showing ill-defined borders (arrows)10
Image adapted with permission from Obeidin F, Alexiev B. Fibromatosis-desmoid. PathologyOutlines.com website. https://www.pathologyoutlines.com/topic/softtissuefibromatosisdeep.html. Accessed April 9, 2024. Arrows added.April 9, 2024. https://doi.org/10.1007/978-94-007-1685-8_3.
Radiotherapy and locoregional therapy
  • Radiotherapy can also be considered where recurrence would be technically challenging to resect and would lead to significant morbidity1
  • Adjuvant radiotherapy is considered for surgery with positive margins for abdominal wall and extra-abdominal desmoid tumours1
  • Locoregional therapy modalities, such as cryoablation, are used to treat small- and moderate-sized extra-abdominal desmoid tumours1

CT, Computed Tomography; DTWG, Desmoid Tumor Working Group; ILP, Isolated Limb Perfusion; MRI, Magnetic Resonance Imaging; NCCN, National Comprehensive Cancer Network® (NCCN®); RECIST, Response Evaluation Criteria in Solid Tumors.

  1. Bektas M et al. Desmoid tumors: a comprehensive review. Adv Ther. 2023;40(9):3697-3722.
  2. Kasper B et al. Desmoid Tumor Working Group. Current management of desmoid tumors: a review. [supplementary appendix] JAMA Oncol. 2024;10(8):1121-1128.
  3. Kasper B et al. Desmoid Tumor Working Group. Current management of desmoid tumors: a review. JAMA Oncol. 2024;10(8):1121-1128.
  4. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Soft Tissue Sarcoma V.1.2024. © National Comprehensive Cancer Network, Inc. 2024. All rights reserved. Accessed April 29, 2024. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.
  5. Cuomo P et al. Extra-abdominal desmoid tumor fibromatosis: a multicenter EMSOS study. BMC Cancer. 2021;21(1):437.
  6. Penel N et al. Pain in desmoid-type fibromatosis: prevalence, determinants and prognosis value. Int J Cancer. 2023;153(2):407-416.
  7. Gronchi A et al. Desmoid Tumor Working Group. The management of desmoid tumours: a joint global consensus-based guideline approach for adult and paediatric patients. Eur J Cancer. 2020;127:96-107.
  8. Wang YF et al. Postoperative recurrence of desmoid tumors: clinical and pathological perspectives. World J Surg Oncol. 2015;13:26.
  9. Easter DW et al. Recent trends in the management of desmoid tumors. Summary of 19 cases and review of the literature. Ann Surg. 1989;210(6):765-769.