Disease Burden

Patients with desmoid tumors may face uncontrollable growth and a high symptom burden.

Even though they do not metastasize, desmoid tumors often interfere with everyday activities and can be life-threatening1,2

Desmoid tumor characteristics:2-5

  • Rare
  • Locally aggressive
  • Unpredictable clinical course
  • Vital organs can be impacted

Desmoid tumors are associated with a potentially high and multifaceted burden of illness:6,*

  • Pain
  • Disfigurement
  • Decreased physical function

In focus groups and interviews, patients reported that pain was the most debilitating symptom of desmoid tumors.1,†

In medical literature and clinical practice, desmoid tumors may also be referred to as aggressive fibromatosis, desmoid-type fibromatosis, or deep fibromatosis

Examples of desmoid tumors and potential symptoms

Back

Large desmoid tumor (15 cm x 10 cm) proximal to the spine7

Image adapted from Cohen S, et al. World J Surg Oncol. 2008;6:28. Reused under Creative Commons License 2.0 (creativecommons.org/licenses/by/2.0). Image background changed to gray.

Hand

Desmoid tumor causing severe restriction in the flexion of the hand8

A hand with a desmoid tumor
Image reproduced from Scaramussa FS, et al. SM J Orthop. 2016;2(3):1036. Reused under Creative Commons License 4.0 (creativecommons.org/licenses/by/4.0).

Knee

MRI scan showing desmoid tumor behind the right knee associated with electric paresthesias and reduced flexion9

MRI image of a desmoid tumor behind the knee
Image adapted from Weschenfelder W, et al. Case Rep Surg. 2015;2015:262654. Reused under Creative Commons License 3.0 (creativecommons.org/licenses/by/3.0). False color added.

Neck

CT scan showing desmoid tumor in the right upper neck involving the brachial plexus associated with pain, numbness, and weakness in the right arm10

CT scan of desmoid tumor in the neck.
Image adapted from Styring E, et al. Am J Med Case Rep. 2019;7(3):36-40. Reused under Creative Commons License 4.0 (creativecommons.org/licenses/by/4.0). False color added.

Data from a Memorial Sloan Kettering/Desmoid Tumor Research Foundation patient-reported outcome (PRO) validation study included patients with desmoid tumors (n=31, age range 20-68, 77% female). Patients participated in 60-minute qualitative phone interviews to provide their perspectives on disease symptoms and impact on their quality of life. The majority of the patients in this study were symptomatic (84%). Tumor site and type varied across patients. The concepts discussed during interviews were used to develop a draft patient-reported outcome scale, which was further refined in cognitive interviews of additional patients with desmoid tumors (n=15).6

Twenty-seven patients with desmoid tumors were interviewed from the Royal Marsden Hospital in the United Kingdom. Two focus groups and 13 interviews explored health-related quality of life issues and experiences of healthcare related to their desmoid tumors.1

CT, computed tomography; MRI, magnetic resonance imaging.

Desmoid tumor management is evolving

Clear margins are often challenging to achieve with surgery and may require extensive resection that can lead to additional pain and functional impairment.11-14

Surgery is no longer recommended as first-line treatment for most clinical situations15,16

  • Up to 77% recurrence rates after surgical resection of desmoid tumors13,17,‡
  • In a retrospective analysis, extremity, chest wall, and intra-abdominal desmoid tumors were associated with higher risk of recurrence than tumors located at other sites18
  • Independent studies have reported desmoid tumor recurrence rates of up to 30% even with clear margins18-21
  • Surgical trauma and growth factors released during wound healing may worsen desmoid tumors and promote recurrence12,13,22

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.15,16,§

Based on retrospective, observational data. Factors associated with local recurrence postsurgery include tumor location, age of the patient, tumor size, margin status, and prior recurrence.18,23

NCCN Guidelines also recommend ablation therapy procedures and definitive radiation therapy as first-line treatment options for progressive, morbid, or symptomatic desmoid tumors for certain patients.15

Identifying the earliest signs of desmoid tumor progression is key for patient management

NCCN Guidelines and DTWG Guideline recommendations for initiating treatment15,16,24

  • Symptoms worsening
  • Tumor growth documented on imaging (eg, MRI or CT)
  • Impairing functioning or daily activities

CT, computed tomography; MRI, magnetic resonance imaging; NCCN, National Comprehensive Cancer Network® (NCCN®).

Give your patients with desmoid tumors a chance to respond to a new systemic treatment

Important Safety Information

Indication 

OGSIVEO is indicated for adult patients with progressing desmoid tumors who require systemic treatment.

Important Safety Information

Warnings and Precautions

Diarrhea: Diarrhea, sometimes severe, can occur in patients treated with OGSIVEO. Diarrhea occurred in 84% of patients treated with OGSIVEO, and included Grade 3 events in 16% of patients. Median time to first diarrhea event was 9 days (range: 2 to 434 days). Monitor patients and manage using antidiarrheal medications. Modify dose as recommended.

Ovarian Toxicity: Female reproductive function and fertility may be impaired in patients treated with OGSIVEO. Impact on fertility may depend on factors like duration of therapy and state of gonadal function at time of treatment. Long-term effects of OGSIVEO on fertility have not been established. Advise patients on the potential risks for ovarian toxicity before initiating treatment. Monitor patients for changes in menstrual cycle regularity or the development of symptoms of estrogen deficiency, including hot flashes, night sweats, and vaginal dryness.

Hepatotoxicity: ALT or AST elevations occurred in 30% and 33% of patients, respectively. Grade 3 ALT or AST elevations (>5 x ULN) occurred in 6% and 2.9% of patients. Monitor liver function tests regularly and modify dose as recommended.

Non-Melanoma Skin Cancers: New cutaneous squamous cell carcinoma and basal cell carcinoma occurred in 2.9% and 1.4% of patients, respectively. Perform dermatologic evaluations prior to initiation of OGSIVEO and routinely during treatment.

Electrolyte Abnormalities: Decreased phosphate (65%) and potassium (22%) occurred in OGSIVEO-treated patients. Phosphate <2 mg/dL occurred in 20% of patients. Grade 3 decreased potassium occurred in 1.4% of patients. Monitor phosphate and potassium levels regularly and supplement as necessary. Modify dose as recommended.

Embryo-Fetal Toxicity: OGSIVEO can cause fetal harm when administered to pregnant women. Oral administration of nirogacestat to pregnant rats during the period of organogenesis resulted in embryo-fetal toxicity and death at maternal exposures below human exposure at the recommended dose of 150 mg twice daily. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use effective contraception during treatment with OGSIVEO and for 1 week after the last dose.

Adverse Reactions

The most common (≥15%) adverse reactions were diarrhea (84%), ovarian toxicity (75% in the 36 females of reproductive potential), rash (68%), nausea (54%), fatigue (54%), stomatitis (39%), headache (30%), abdominal pain (22%), cough (20%), alopecia (19%), upper respiratory tract infection (17%), and dyspnea (16%).

Serious adverse reactions occurred in 20% of patients who received OGSIVEO. Serious adverse reactions occurring in ≥2% of patients were ovarian toxicity (4%).

The most common laboratory abnormalities (≥15%) were decreased phosphate, increased urine glucose, increased urine protein, increased AST, increased ALT, and decreased potassium.

Drug Interactions

CYP3A Inhibitors and Inducers: Avoid concomitant use with strong or moderate CYP3A inhibitors (including grapefruit products, Seville oranges, and starfruit) and strong or moderate CYP3A inducers.

Gastric Acid Reducing Agents: Avoid concomitant use with proton pump inhibitors and H2 blockers. If concomitant use cannot be avoided, OGSIVEO can be staggered with antacids (e.g., administer OGSIVEO 2 hours before or 2 hours after antacid use).

Consult the full Prescribing Information prior to and during treatment for important drug interactions.

Use in Specific Populations

Because of the potential for serious adverse reactions in breastfed children, advise women not to breastfeed during treatment with OGSIVEO and for 1 week after the last dose.

Please click here for full Prescribing Information.

Important Safety Information

CT, computed tomography; HCP, healthcare professional; MRI, magnetic resonance imaging; NCCN, National Comprehensive Cancer Network® (NCCN®).

References
  1. Husson O, Younger E, Dunlop A, et al. Desmoid fibromatosis through the patients’ eyes: time to change the focus and organisation of care? Support Care Cancer. 2019;27(3):965-980.
  2. Constantinidou A, Scurr M, Judson I, Litchman C. Clinical presentation of desmoid tumors. In: Litchman C, ed. Desmoid Tumors. Springer; 2012:chap 2. Accessed December 18, 2023. https://www.researchgate.net/publication/226455135.
  3. Kasper B, Baumgarten C, Garcia J, et al. Desmoid Working Group. An update on the management of sporadic desmoid-type fibromatosis: a European Consensus Initiative between Sarcoma Patients EuroNet (SPAEN) and European Organization for Research and Treatment of Cancer (EORTC)/Soft Tissue and Bone Sarcoma Group (STBSG). Ann Oncol. 2017;28(10):2399-2408.
  4. Penel N, Chibon F, Salas S. Adult desmoid tumors: biology, management and ongoing trials. Curr Opin Oncol. 2017;29(4):268-274.
  5. Sbaraglia M, Bellan E, Dei Tos AP. The 2020 WHO Classification of Soft Tissue Tumours: news and perspectives. Pathologica. 2021;113(2):70-84.
  6. Gounder MM, Maddux L, Paty J, Atkinson TM. Prospective development of a patient-reported outcomes instrument for desmoid tumors or aggressive fibromatosis. Cancer. 2020;126(3):531-539.
  7. Cohen S, Ad-El D, Benjaminov O, Gutman H. Post-traumatic soft tissue tumors: case report and review of the literature a propos a post-traumatic paraspinal desmoid tumor. World J Surg Oncol. 2008;6:28.
  8. Scaramussa FS, Castro UB. Desmoid tumor in hand: a case report. SM J Orthop. 2016;2(3):1036.
  9. Weschenfelder W, Lindner R, Spiegel C, et al. Desmoid tumor of the popliteal fossa during pregnancy. Case Rep Surg. 2015;2015:262654.
  10. Styring E, Ahlström M, Rissler P, et al. Desmoid fibromatosis in the brachial plexus mimicking an ulnar nerve entrapment. Am J Med Case Rep. 2019;7(3):36-40.
  11. Lewis JJ, Boland PJ, Leung DHY, et al. The enigma of desmoid tumors. Ann Surg. 1999;229(6):866-872.
  12. Bonvalot S, Desai A, Coppola S, et al. The treatment of desmoid tumors: a stepwise clinical approach. Ann Oncol. 2012;23(suppl 10):x158-x166.
  13. Skubitz KM. Biology and treatment of aggressive fibromatosis or desmoid tumor. Mayo Clin Proc. 2017;92(6):947-964.
  14. Dafford K, Kim D, Nelson A, Kline D. Extraabdominal desmoid tumors. Neurosurg Focus. 2007;22(6):E21.
  15. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Soft Tissue Sarcoma V.3.2023. © National Comprehensive Cancer Network, Inc. 2023. All rights reserved. Accessed December 18, 2023. 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.
  16. 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.
  17. Easter DW, Halasz NA. Recent trends in the management of desmoid tumors. Summary of 19 cases and review of the literature. Ann Surg. 1989;210(6):765-769.
  18. Crago AM, Denton B, Salas S, et al. A prognostic nomogram for prediction of recurrence in desmoid fibromatosis. Ann Surg. 2013;258(2):347-353.
  19. Peng PD, Hyder O, Mavros MN, et al. Management and recurrence patterns of desmoids tumors: a multi-institutional analysis of 211 patients. Ann Surg Oncol. 2012;19(13):4036-4042.
  20. Janssen ML, van Broekhoven DL, Cates JM, et al. Meta-analysis of the influence of surgical margin and adjuvant radiotherapy on local recurrence after resection of sporadic desmoid-type fibromatosis. Br J Surg. 2017;104(4):347-357.
  21. Nuyttens JJ, Rust PF, Thomas CR Jr, Turrisi AT 3rd. Surgery versus radiation therapy for patients with aggressive fibromatosis or desmoid tumors: a comparative review of 22 articles. Cancer. 2000;88(7):1517-1523.
  22. Cheon SS, Cheah AYL, Turley S, et al. Beta-catenin stabilization dysregulates mesenchymal cell proliferation, motility, and invasiveness and causes aggressive fibromatosis and hyperplastic cutaneous wounds. Proc Natl Acad Sci USA. 2002;99(10):6973-6978.
  23. Tsagozis P, Stevenson JD, Grimer R, Carter S. Outcome of surgery for primary and recurrent desmoid-type fibromatosis. A retrospective case series of 174 patients. Ann Med Surg (Lond). 2017;17:14-19.
  24. 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 [supplementary appendix]. Eur J Cancer. 2020;127:96-107.