Real-World Study Highlights Evolving Treatment Patterns and Petsistent Diagnostic Challenges in Patients With Desmoid Tumors

Clinical Summary:

• Design/Population: This retrospective, single-cohort study assessed clinical course, treatment patterns, symptom burden, and healthcare resource utilization among adults with desmoid tumors. 
• Key Outcomes: Most patients experienced active or symptomatic disease, and first-line treatment patterns shifted away from surgery toward systemic therapies and cryoablation over time. Although surgery remained the most common intervention, recurrence occurred in approximately one-third of patients, and potential misdiagnosis was associated with higher healthcare resource utilization before diagnosis. 
• Clinical Relevance: These findings highlight evolving treatment practices that increasingly favor systemic therapy over surgery while emphasizing the importance of timely diagnosis and improved therapeutic strategies for patients with desmoid tumors. 

Real-world data from a single academic health network demonstrated that management of desmoid tumors has shifted away from surgery and toward systemic therapies and cryoablation, reflecting evolving recommendations favoring nonsurgical treatment approaches. 

 “Because of the difficulty in achieving effective treatment in many cases, the disease course of [desmoid tumors] is typically protracted,” stated Chia Jie Tan, MD, University of Utah, Salt Lake City, Utah, and coauthors. “Surgical resection has historically been the primary intervention for [desmoid tumors]…However, this approach has been associated with high recurrence rates.” 

In this retrospective, single-center cohort study, researchers identified 148 adult patients diagnosed with desmoid tumors between January 2011 and July 2023 within the University of Utah Health network. Investigators characterized patient demographics, symptom burden, treatment patterns, healthcare resource utilization, and the frequency of potential misdiagnosis before definitive diagnosis.

At a median follow-up of 26.9 months, 88.5% of patients experienced at least 1 episode of active disease, including disease progression (36.5%), symptomatic disease (59.5%), or active treatment. Tumor-related pain was the most frequently reported symptom (57.4%), followed by fullness (15.5%), loss of function (14.9%), and paresthesia (11.5%).

Overall, 79.7% of patients received at least 1 line of treatment, while 20.3% of patients underwent active surveillance alone. Surgery remained the most common treatment modality, performed in 52.7% of patients, although recurrence developed in 34.6% of patients following surgical resection, with a median time to recurrence of 18.8 months.

Treatment patterns changed significantly during the study period. First-line surgery declined from 75% of patients treated between 2011 and 2014 to 39.3% between 2021 and 2023 (P < .001), whereas first-line tyrosine kinase inhibitor use increased from 3.1% to 28.6% (P < .001). Cryoablation also became more common after 2018. 

Healthcare resource utilization increased during periods of symptomatic and active disease. Approximately one-third of patients (29.1%) had evidence of a potential misdiagnosis before confirmation of desmoid tumor, most commonly as another benign soft tissue tumor or a low-grade sarcoma. Compared with patients without a potential misdiagnosis, these individuals had significantly greater outpatient visits, emergency department visits, and hospitalizations during the year preceding diagnosis.

“Treatment patterns for [desmoid tumors] have changed in the last decade in line with recommendations to limit the use of surgery…However, the varied treatment approaches used in place of surgery, multiple lines of therapy, and substantial symptom burden among some patients support the use of better therapeutic agents,” concluded Dr Tan et al. “FDA approval of nirogacestat may transform the treatment landscape of [desmoid tumors], and future work is needed to examine how [desmoid tumor] management and patient outcomes may change.” 

Source:

Tan CJ, Willis C, Bell T, et al. Real-world characterization and treatment patterns of patients with desmoid tumors at an academic center in the United States. Cancer Res Commun. Published online: April 9, 2026. doi:10.1158/2767-9764.CRC-25-0581